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New Phase IIb/III Clinical Data Demonstrates Over Three Years of Continuous Treatment with Oral Blarcamesine to Significantly Benefit Early Alzheimer’s Disease Patients

Topline ATTENTION-AD trial: Patients showed improved cognition and function over three years

Delayed-start analysis of treatment with oral blarcamesine was significant reflecting importance of early treatment initiation

Blarcamesine exhibited a favorable safety profile with no treatment-related deaths

NEW YORK, Jan. 13, 2025 (GLOBE NEWSWIRE) — Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company focused on developing innovative treatments for Alzheimer’s disease, Parkinson’s disease, schizophrenia, neurodevelopmental, neurodegenerative, and rare diseases, including Rett syndrome, and other central nervous system (CNS) disorders, today announced that over three years of continuous treatment with blarcamesine (ANAVEX®2-73) demonstrated significantly reduced clinical decline showing continued clinically and meaningful benefit for early Alzheimer’s disease patients.

No new safety findings have been observed with continued blarcamesine treatment over three (3) years with good comparative safety profile and no associated neuroimaging adverse events. There were no deaths related to the study drug.

The ATTENTION-AD (ANAVEX®2-73-AD-EP-004) trial followed the 48-week ANAVEX®2-73-AD-004 double-blind (DB) clinical trial with an open-label extension (OLE) treatment duration of 96 weeks for participants in North America and Europe and up to 144 weeks for participants in Australia1 to evaluate the safety and tolerability of blarcamesine and long-term effects of blarcamesine on cognition and function in participants with early Alzheimer’s disease.2

Blarcamesine-treated patients continue to accrue benefit through up to 4 years, as measured by the clinical endpoints ADAS-Cog13 and ADCS-ADL. Delayed-start analysis of treatment with oral blarcamesine was significant reflecting importance of early treatment initiation.

“Long-term clinical ATTENTION-AD study results support the importance of continued long-term blarcamesine treatment,” said Prof. Dr. Timo Grimmer, MD, member of the Anavex Scientific Advisory Board and National Coordinating Investigator for the blarcamesine Phase IIb/III ANAVEX®2-73-AD-004 study. “Blarcamesine is easily scalable and might be a potential therapeutic solution for Alzheimer’s disease patients to potentially offer hope and relief and equitable and accessible for diverse populations and maintaining sustainability within the global healthcare systems.”


Topline Efficacy Data:

The delayed-start analysis was performed using the prespecified Mixed effect Model Repeat Measure (MMRM) model, to evaluate the effect of early treatment with blarcamesine on all data collected in both DB and OLE phases as the potential indication of disease-modifying characteristics of the treatment. Comparisons were made between ‘Continued blarcamesine’ and ‘Placebo to blarcamesine’ for ADAS-Cog13 and ADCS-ADL at the scheduled visits. ‘Continued blarcamesine’ is the early start group and ‘Placebo to blarcamesine’ is the late start group.

The delayed-start analysis for ADAS-Cog13 showed a significant difference between early start and late start treatment groups at Week 144 (LS mean difference -2.70, P = 0.0348), favoring the early start group. This observed treatment difference continued to increase up to Week 192 (LS mean difference -3.83, P = 0.0165). Together these results suggest that participants who initiated treatment with blarcamesine earlier in their disease progression showed greater stability of cognitive function compared to those who did not initiate blarcamesine until ~1 year later.

In addition, to place these findings in context, an ADAS-Cog13 score LS mean difference between the treatment groups at both Week 144 (OLE Week 96) and Week 192 (OLE Week 144) being larger than 2 points are considered clinically meaningful improvements.3

Similarly, the delayed-start analysis for ADCS-ADL showed numerically favorable results for the early start group over the late start group at Week 144 (LS mean difference +2.32, P = 0.125). The treatment difference continued to increase up to Week 192 and reached statistical significance (LS mean difference +4.30, P = 0.0206).

This suggests that earlier initiation of treatment with blarcamesine may have a significant positive impact on disease progression and may provide continued benefits to patients with early Alzheimer’s disease over the long-term.


Topline Safety Data:

Blarcamesine exhibited a favorable safety profile with the majority of adverse events (AEs) mild to moderate in severity (Grade 1 or 2), were predominantly linked to the initial titration phase, and could be managed with adjusted titration schedules. Importantly, no severe or life-threatening adverse events were attributed to blarcamesine. There were no deaths related to blarcamesine.

The ATTENTION-AD trial demonstrated the manageable nature of the most frequent treatment emergent adverse event (TEAE) of dizziness observed in the preceding ANAVEX®2-73-AD-004 trial, which was generally transient in duration (approx. 7-11 days) and mild or moderate in severity (Grade 1 or 2). The titration schedule was adjusted to a slightly longer titration period in the ATTENTION-AD trial, from previous 2-3 weeks to 10 weeks. A markedly lower frequency of the TEAE of dizziness in the respective maintenance phase was observed: from previously 25.2% in the ANAVEX®2-73-AD-004 trial to 9.6% in the ATTENTION-AD trial, demonstrating the manageable nature of the most frequent TEAE (dizziness).

Furthermore, no notable findings were observed over time in vital sign, clinical laboratory test, physical examination, electrocardiogram (ECG), and no pattern of serious adverse events (SAEs) were reported.


Compassionate Use:

Lastly, there are currently 74 participants receiving blarcamesine within the Compassionate Use Program, who continued treatment with blarcamesine subsequent completion of respective preceding open-label-extension studies from both ANAVEX®2-73-AD-EP-004 in early AD and from ANAVEX®2-73-003 in mild-to-moderate AD. Including the start of the respective preceding studies, some participants are on oral blarcamesine once daily for over 9 years. Importantly, no severe or life-threatening adverse events were attributed to blarcamesine.

“These results demonstrate that diagnosing and treating people earlier in the progression of Alzheimer’s disease may lead to greater clinical benefit,” said Juan Carlos Lopez-Talavera, MD, PhD, Head of Research and Development of Anavex. “Additionally, the comprehensive data from the blarcamesine Alzheimer’s disease program represents a solid foundation for the subsequent strategy of our Phase III and IV development plan.”

“Alzheimer’s disease, like other chronic progressive diseases, requires a long-term therapeutic strategy. Blarcamesine mechanism of action with its convenient once daily oral dosing supports long-term therapy,” said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. “We remain excited about the scalable and convenient features of oral blarcamesine, which could be appealing because of its route of administration and good comparative safety profile. This could reduce crucial barriers within the currently complex healthcare ecosystem for Alzheimer’s disease and potentially provide broader access to a diverse population with early Alzheimer’s disease.”

ATTENTION-AD (ANAVEX®2-73-AD-EP-004) trial result details will be presented at the upcoming AD/PD™ 2025 Conference.

This release discusses investigational uses of an agent in development and is not intended to convey conclusions about efficacy or safety. There is no guarantee that any investigational uses of such product will successfully complete clinical development or gain health authority approval.

About Anavex Life Sciences Corp.

Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company dedicated to the development of novel therapeutics for the treatment of neurodegenerative, neurodevelopmental, and neuropsychiatric disorders, including Alzheimer’s disease, Parkinson’s disease, schizophrenia, Rett syndrome, and other central nervous system (CNS) diseases, pain, and various types of cancer. Anavex’s lead drug candidate, ANAVEX®2-73 (blarcamesine), has successfully completed a Phase 2a and a Phase 2b/3 clinical trial for Alzheimer’s disease, a Phase 2 proof-of-concept study in Parkinson’s disease dementia, and both a Phase 2 and a Phase 3 study in adult patients and one Phase 2/3 study in pediatric patients with Rett syndrome. ANAVEX®2-73 is an orally available drug candidate designed to restore cellular homeostasis by targeting SIGMAR1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer’s disease. ANAVEX®2-73 also exhibited anticonvulsant, anti-amnesic, neuroprotective, and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson’s Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX®2-73 for the treatment of Parkinson’s disease. We believe that ANAVEX®3-71, which targets SIGMAR1 and M1 muscarinic receptors, is a promising clinical stage drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimer’s disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid, and tau pathologies. In preclinical trials, ANAVEX®3-71 has shown beneficial effects on mitochondrial dysfunction and neuroinflammation. Further information is available at www.anavex.com. You can also connect with the Company on Twitter,Facebook, Instagram, and LinkedIn.

Forward-Looking Statements

Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks set forth in the Company’s most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Anavex Life Sciences Corp. undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.

For Further Information:

Anavex Life Sciences Corp.
Research & Business Development
Toll-free: 1-844-689-3939
Email: [email protected]

Investors:

Andrew J. Barwicki
Investor Relations
Tel: 516-662-9461
Email: [email protected]

____________________
1 The preceding double-blind trial (ANAVEX®2-73-AD-004) began in Australia before the other regions (Europe and North America), leaving insufficient time for the other regions to participate in the additional OLE extension beyond the initial 96 Weeks OLE period. The extension, requested by investigators in Australia, extended the initial 96-Week OLE period to 144 Weeks.
2 The scheduled visits were [OLE Week 0 = Combined Week 48], [OLE Week 48 = Combined Week 96], [OLE Week 96 = Combined Week 144] and [OLE Week 144 = Combined Week 192]; Combined = OLE (open-label-extension) + DB (double-blind) trials.
3 Muir RT, Hill MD, Black SE, Smith EE. Minimal clinically important difference in Alzheimer’s disease: Rapid review. Alzheimers Dement. 2024;20(5):3352-3363. doi:10.1002/alz.13770



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Arbutus Provides 2025 Corporate and Financial Update

Initiating Phase 2b clinical trial in first half of 2025 after achieving a meaningful functional cure rate in cHBV patients treated with imdusiran and interferon

WARMINSTER, Pa., Jan. 13, 2025 (GLOBE NEWSWIRE) — Arbutus Biopharma Corporation (Nasdaq: ABUS) (“Arbutus” or the “Company”), a clinical-stage biopharmaceutical company leveraging its extensive virology expertise to develop a functional cure for people with chronic hepatitis B virus (cHBV) infection, today announced its 2025 corporate objectives and provided a financial update.

“We enter 2025 with solid financial footing and strong momentum in achieving our mission of developing a functional cure for cHBV, a disease that affects more than 250 million people worldwide and is a leading cause of liver cancer,” said Michael J. McElhaugh, Interim President and Chief Executive Officer of Arbutus. “The data we reported late last year from our IM-PROVE I Phase 2a clinical trial showed a meaningful functional cure rate and immune activation in cHBV patients that were treated with our RNAi therapeutic, imdusiran, interferon and nucleos(t)ide analogue (NA) therapy. These data support our belief that imdusiran is differentiated from other RNAi therapeutics in development for HBV. Therefore, we plan to initiate a Phase 2b clinical trial combining imdusiran, interferon and NA therapy in the first half of 2025.”     


Imdusiran (RNAi therapeutic)

  • At the American Association for the Study of Liver Diseases (AASLD) – The Liver Meeting® in November 2024, the Company presented new data from its IM-PROVE I Phase 2a clinical trial showing that six doses of imdusiran and 24 weeks of pegylated interferon alfa-2α (IFN), a standard-of-care immunomodulator, added to ongoing NA therapy led to a functional cure rate of 50% (3/6) in HBeAg-negative patients with baseline HBsAg levels less than 1000 IU/mL, and an overall functional cure rate of 25% (3/12). Those patients that achieved a functional cure also seroconverted with high anti-HBs antibody levels. The combination of imdusiran, IFN and NA therapy was generally safe and well-tolerated.
  • Based on this data, the Company is planning to initiate a placebo-controlled Phase 2b clinical trial with this treatment regimen in the first half of 2025. Subject to regulatory approval, the clinical trial is anticipated to enroll approximately 170 HBeAg-negative cHBV patients with baseline HBsAg ≤1000 IU/mL. Additional details will be provided by the Company after regulatory approval.
  • The Company also presented data from its IM-PROVE II Phase 2a clinical trial showing that the addition of low dose nivolumab increased rates of HBsAg loss in cHBV patients that were first treated with imdusiran, ongoing NA therapy and Barinthus Biotherapeutics’ VTP-300. In this clinical trial, 23% (3/13) of patients that received imdusiran, VTP-300, NA therapy and nivolumab achieved HBsAg loss by week 48. The Company is evaluating functional cure in these patients and anticipates reporting data in the first half of 2025.


AB-101 (oral PD-L1 inhibitor)

  • AB-101-001 is a Phase 1a/1b double-blind, randomized, placebo-controlled clinical trial designed to investigate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single- and multiple-ascending doses of AB-101, the Company’s oral PD-L1 inhibitor, in healthy subjects and patients with cHBV.
  • Based on data from Part 2 of this clinical trial reported in November 2024 showing that AB-101 was generally well-tolerated with evidence of dose-dependent receptor occupancy, Arbutus has moved into Part 3 which evaluates repeat doses of AB-101 for 28 days in patients with cHBV. Data from the 10 mg cohort is expected in the first half of 2025. Next steps for AB-101 will be determined after Arbutus evaluates data from Part 3 of this clinical trial.


LNP Litigation

  • Arbutus will continue to protect and defend its intellectual property, which is the subject of the on-going lawsuits against Moderna and Pfizer/BioNTech. The Company is seeking fair compensation for Moderna’s and Pfizer/BioNTech’s use of its patented LNP technology that was developed with great effort and at a great expense, without which Moderna’s and Pfizer/BioNTech’s COVID-19 vaccines would not have been successful.
  • The claim construction hearing for the lawsuit against Pfizer/BioNTech occurred on December 18, 2024. The court is expected to provide its ruling on the claim construction and issue the scheduling order in the first half of 2025.
  • The Moderna trial date is scheduled for September 24, 2025, and is subject to the court’s availability. Expert reports and expert depositions continue in this lawsuit.


Financial


Update:

  • The Company had cash, cash equivalents and investments in marketable securities totaling approximately $123 million as of December 31, 2024 (unaudited).
  • The Company expects to significantly reduce its net cash burn in 2025 when compared to 2024. Net cash burn is expected to range from $47 to $50 million in 2025 versus a 2024 net cash burn of approximately $65 million (unaudited). The Company believes its cash, cash equivalents, investments in marketable securities and anticipated contractual milestones from Qilu Pharmaceutical, its strategic partner in Greater China, are sufficient to fund its operations through the first quarter of 2028. This includes fully funding the imdusiran Phase 2b clinical trial.
  • The preliminary cash, cash equivalents and investments as of December 31, 2024 and the estimated 2024 net cash burn were calculated prior to the completion of an audit by Arbutus’ independent registered public accounting firm and are therefore subject to adjustment.
  • With its current cash balance and anticipated 2025 net cash burn, the Company does not anticipate utilizing its “at-the-market” program (ATM) this year.


About Imdusiran

Imdusiran is an RNAi therapeutic specifically designed to reduce all HBV viral proteins and antigens including hepatitis B surface antigen, which is thought to be a key prerequisite to enable reawakening of a patient’s immune system to respond to the virus. Imdusiran targets hepatocytes using Arbutus’ novel covalently conjugated N-Acetylgalactosamine (GalNAc) delivery technology enabling subcutaneous delivery. In a Phase 2a clinical trial, imdusiran achieved meaningful functional cure rates in patients with cHBV when combined with pegylated interferon (IFN) alfa-2α and nucleos(t)ide analogue (NA) therapy. Clinical data generated thus far has shown imdusiran to be generally safe and well-tolerated, while also providing meaningful reductions in hepatitis B surface antigen and hepatitis B DNA. In the first half of 2025, the Company is planning to initiate a Phase 2b clinical trial of imdusiran combined with IFN and NA therapy.


About AB-101

AB-101 is our oral PD-L1 inhibitor candidate that we believe will allow for controlled checkpoint blockade while minimizing the systemic safety issues typically seen with checkpoint antibody therapies. Immune checkpoints such as PD-1/PD-L1 play an important role in the induction and maintenance of immune tolerance and in T-cell activation. Preclinical data generated thus far indicates that AB-101 mediates re-activation of exhausted HBV-specific T-cells from cHBV patients. We believe AB-101, when used in combination with other approved and investigational agents, could potentially lead to a functional cure in patients chronically infected with HBV. AB-101 is currently being evaluated in a Phase 1a/1b clinical trial.


About HBV

Hepatitis B is a potentially life-threatening liver infection caused by the hepatitis B virus (HBV). HBV can cause chronic infection which leads to a higher risk of death from cirrhosis and liver cancer. Chronic HBV infection represents a significant unmet medical need. The World Health Organization estimates that over 250 million people worldwide suffer from chronic HBV infection, while other estimates indicate that approximately 2 million people in the United States suffer from chronic HBV infection. Approximately 1.1 million people die every year from complications related to chronic HBV infection despite the availability of effective vaccines and current treatment options.


About Arbutus

Arbutus Biopharma Corporation (Nasdaq: ABUS) is a clinical-stage biopharmaceutical company leveraging its extensive virology expertise to develop novel therapeutics with distinct mechanisms of action, which can potentially be combined to provide a functional cure for patients with chronic hepatitis B virus (cHBV). Arbutus believes the key to success in developing a functional cure involves suppressing HBV DNA, reducing surface antigen, and boosting HBV-specific immune responses. Arbutus’ pipeline of internally developed, proprietary compounds includes an RNAi therapeutic, imdusiran (AB-729), and an oral PD-L1 inhibitor, AB-101. Imdusiran has achieved meaningful functional cure rates in patients with cHBV when administered as combination therapy. In the first half of 2025, Arbutus is planning to initiate a Phase 2b clinical trial with imdusiran. AB-101 is currently being evaluated in a Phase 1a/1b clinical trial. For more information, visit www.arbutusbio.com.


Forward-Looking Statements and Information

This press release contains forward-looking statements within the meaning of the Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, and forward-looking information within the meaning of Canadian securities laws (collectively, forward-looking statements). Forward-looking statements in this press release include statements about: the potential to lead to a functional cure for HBV; Arbutus’ future development plans for its product candidates; the expected timing, cost and results of Arbutus’ clinical development plans and clinical trials with respect to its product candidates; Arbutus’ expectations with respect to the cost, initiation and the release of data from its clinical trials and the expected timing thereof; the potential for Arbutus’ product candidates to achieve success in clinical trials; Arbutus’ expectations with respect to utilizing its ATM program; Arbutus’ plans with respect to the ongoing patent litigation matters; and Arbutus’ expected financial condition, including the anticipated duration of its cash runway, its expectations regarding its 2025 cash burn and the timing and need for additional capital.

With respect to the forward-looking statements contained in this press release, Arbutus has made numerous assumptions regarding, among other things: the effectiveness and timeliness of clinical trials, and the usefulness of the data; the timeliness of regulatory approvals; the continued demand for Arbutus’ assets; and the stability of economic and market conditions. While Arbutus considers these assumptions to be reasonable, these assumptions are inherently subject to significant business, economic, competitive, market and social uncertainties and contingencies. Additionally, there are known and unknown risk factors which could cause Arbutus’ actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements contained herein. Known risk factors include, among others: ongoing and anticipated clinical trials may be more costly or take longer to complete than anticipated, and may never be initiated or completed, or may not generate results that warrant future development of the tested product candidate; Arbutus may elect to change its strategy regarding its product candidates and clinical development activities; Arbutus may not receive the necessary regulatory approvals for the clinical development of Arbutus’ products; uncertainties associated with litigation generally and patent litigation specifically; economic and market conditions may worsen; market shifts may require a change in strategic focus; Arbutus and its collaborators may never realize the expected benefits of the collaborations; Arbutus may need to utilize its ATM program based on changes in its business; Arbutus’ plans to reduce its net cash burn may not materially extend the cash runway and may create a distraction or uncertainty that may adversely affect its operating results, business, or investor perceptions; and risks related to the sufficiency of Arbutus’ cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures.

A more complete discussion of the risks and uncertainties facing Arbutus appears in Arbutus’ Annual Report on Form 10-K, Arbutus’ Quarterly Reports on Form 10-Q and Arbutus’ continuous and periodic disclosure filings, which are available at www.sedar.com and at www.sec.gov. All forward-looking statements herein are qualified in their entirety by this cautionary statement, and Arbutus disclaims any obligation to revise or update any such forward-looking statements or to publicly announce the result of any revisions to any of the forward-looking statements contained herein to reflect future results, events or developments, except as required by law. 


Contact Information


Investors and Media
  

Lisa M. Caperelli
Vice President, Investor Relations  
Phone: 215-206-1822  
Email: [email protected]



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22nd Century Stands Ready to Support the Adoption of FDA Proposed Tobacco Product Standard to Mandate Reduced Nicotine Content in Cigarettes with its VLN Cigarettes

FDA Reduced Nicotine Content Proposal Indicated to Have Completed OMB Review as of January 3, 2025

FDA Spokesperson Indicates Policy Could Be Among Most Impactful Population-Level Actions in the History of U.S. Tobacco Product Regulation

MOCKSVILLE, N.C., Jan. 13, 2025 (GLOBE NEWSWIRE) — 22nd Century Group, Inc. (Nasdaq: XXII), a tobacco products company that is leading the fight against nicotine and believes smokers should have a choice about their nicotine consumption, today announced that it stands ready to support the U.S. Food and Drug Administration’s proposed tobacco product standard to mandate reduced nicotine content in cigarettes, which is indicated to have cleared review by the U.S. Office of Management and Budget on January 3, 2025. The proposal is part of a long-standing effort by the FDA to mandate a drastic reduction in the amount of nicotine in cigarettes, a cornerstone goal of public health experts seeking to reduce the harms of smoking and very much in concert with 22nd Century’s VLN branded low nicotine cigarettes.

“OMB clearance marks a major step in moving this policy forward, and we are excited to see further definitive action by the federal government toward making this scientifically backed, non-partisan smoking harm reduction policy a reality,” said Larry Firestone, Chief Executive Officer of 22nd Century Group. “The FDA has openly stated that this could be the most impactful action for the improvement of public health in reducing the widespread and clearly documented harms of smoking.”

“22nd Century has played a pivotal role in the research, development and numerous federally funded clinical studies underpinning this policy, which has been advanced across multiple administrations since 2009, including action under the first Trump administration. We are also the first and only Company to bring an FDA authorized reduced nicotine content cigarette designed specifically to reduce the harms of smoking to market through the commercial launch of our VLN reduced nicotine content cigarettes. We are now working to launch additional products, including additional reduced nicotine content products using our tobacco but under other brand labels and give greater awareness to low nicotine cigarettes as a separate category in the market. This is a key component as part of our drive to expand the distribution of reduced nicotine content cigarettes manufactured by 22nd Century, increase consumer awareness and expand the VLN® footprint.”

About 22nd Century Group, Inc.

22nd Century Group is the pioneering nicotine harm reduction company in the tobacco industry enabling smokers to take control of their nicotine consumption. 

We created our flagship product, the VLN® cigarette, to give traditional cigarette smokers an authentic and familiar alternative that helps them take control of their nicotine consumption. VLN® cigarettes have 95% less nicotine than the traditional cigarette and have been proven to greatly reduce nicotine consumption. Instead of offering new ways of delivering nicotine to addicted smokers, we offer smokers the option to take control of their nicotine consumption and make informed and more productive choices, including the choice to avoid addictive levels of nicotine altogether. 

Our wholly owned subsidiaries include a leading cigarette manufacturer that produces all VLN® products and provides turnkey contract manufacturing for other tobacco brands both domestically and internationally. The 60,000 square foot facility in Mocksville, North Carolina has the capacity to produce more than 45 million cartons of combusted tobacco products annually with additional space for expansion. 

Our proprietary reduced nicotine tobacco blends are made possible by comprehensive and patented technologies that regulate nicotine biosynthesis activities in the tobacco plant, resulting in full flavor and high yield with 95% less nicotine. Our extensive patent portfolio has been developed to ensure we have the only low nicotine combustible cigarette in the United States and critical international markets.

VLN® and Helps You Smoke Less® are registered trademarks of 22nd Century Limited LLC.

Learn more at xxiicentury.com, on X (formerly Twitter), on LinkedIn, and on YouTube.

Learn more about VLN® at tryvln.com.

Cautionary Note Regarding Forward-Looking Statements

Except for historical information, all of the statements, expectations, and assumptions contained in this press release are forward-looking statements, including but not limited to our full year business outlook. Forward-looking statements typically contain terms such as “anticipate,” “believe,” “consider,” “continue,” “could,” “estimate,” “expect,” “explore,” “foresee,” “goal,” “guidance,” “intend,” “likely,” “may,” “plan,” “potential,” “predict,” “preliminary,” “probable,” “project,” “promising,” “seek,” “should,” “will,” “would,” and similar expressions. Forward-looking statements include, but are not limited to, statements regarding (i) our cost reduction initiatives, (ii) our expectations regarding regulatory enforcement, including our ability to receive an exemption from new regulations, (iii) our financial and operating performance and (iv) our expectations for our business interruption insurance claim. Actual results might differ materially from those explicit or implicit in forward-looking statements. Important factors that could cause actual results to differ materially are set forth in “Risk Factors” in the Company’s Annual Report on Form 10-K filed on March 28, 2024, and in the Company’s Quarterly Reports filed on May 15, 2024, August 13, 2024, and November 11, 2024. All information provided in this release is as of the date hereof, and the Company assumes no obligation to and does not intend to update these forward-looking statements, except as required by law.

Investor Relations & Media Contact

Matt Kreps
Investor Relations
22nd Century Group
[email protected]
214-597-8200



Posted on

Kiniksa Pharmaceuticals Reports Preliminary 2024 Net Product Revenue and Provides 2025 Net Product Revenue Guidance

– ARCALYST

®

(rilonacept) 2024 net product revenue of $416.4 million (unaudited), representing ~79% year-over-year growth –

– ARCALYST 2025 net product revenue expected to be $560 – $580 million –

– Kiniksa expects to remain cash flow positive on an annual basis –

LONDON, Jan. 13, 2025 (GLOBE NEWSWIRE) — Kiniksa Pharmaceuticals International, plc (Nasdaq: KNSA) (Kiniksa), a commercial-stage biopharmaceutical company with a pipeline of immune-modulating assets designed to target a spectrum of cardiovascular and autoimmune diseases, today reported preliminary unaudited fourth quarter and full year 2024 ARCALYST net product revenue and provided 2025 ARCALYST net product revenue guidance.

“ARCALYST uptake in recurrent pericarditis continues to grow. As of the end of 2024, approximately 13% of the multiple-recurrence target population was actively on ARCALYST therapy, driving net product revenue to $416.4 million, representing 79% year-over-year growth. Importantly, there is still substantial opportunity ahead for Kiniksa to drive further ARCALYST revenue as we continue executing on our commercial strategy,” said Sanj K. Patel, Chairman and Chief Executive Officer of Kiniksa. “We are also advancing our clinical portfolio as we work to bring new life-changing therapies to patients with unmet medical needs. Lastly, our robust financial profile provides the ability to pursue additional value-creating opportunities.”


Commercial Execution


ARCALYST (IL-1α and IL-1β cytokine trap)

  • ARCALYST net product revenue was $121.9 million and $416.4 million for the fourth quarter and full year 2024, respectively (unaudited).
  • As of the end of the fourth quarter of 2024, approximately 13% of the target 14,000 multiple-recurrence patients were actively on ARCALYST treatment.
  • Since launch, more than 2,850 prescribers have written ARCALYST prescriptions for recurrent pericarditis.
  • As of the end of the fourth quarter of 2024, average total duration of ARCALYST therapy in recurrent pericarditis was approximately 27 months.        
  • Kiniksa expects 2025 ARCALYST net product revenue of between $560 million and $580 million.


Corporate Update

  • As of December 31, 2024, Kiniksa had $243.6 million of cash, cash equivalents, and short-term investments (unaudited).
  • Kiniksa expects to remain cash flow positive on an annual basis.


43



rd



Annual J.P. Morgan Healthcare Conference

  • Sanj K. Patel, Chairman and Chief Executive Officer of Kiniksa, and Ross Moat, Chief Commercial Officer, will provide a corporate presentation at the 43rd Annual J.P. Morgan Healthcare Conference on January 13, 2025, at 1:30 p.m. Pacific Time (4:30 p.m. Eastern Time). A live webcast of Kiniksa’s presentation will be accessible through the Investors & Media section of the company’s website at www.kiniksa.com. A replay of the webcast will also be available on Kiniksa’s website within approximately 48 hours after the event.

About Kiniksa

Kiniksa is a commercial-stage biopharmaceutical company focused on discovering, acquiring, developing, and commercializing therapeutic medicines for patients suffering from debilitating diseases with significant unmet medical need. Kiniksa’s immune-modulating assets are based on strong biologic rationale or validated mechanisms, target a spectrum of underserved cardiovascular and autoimmune conditions, and offer the potential for differentiation. For more information, please visit www.kiniksa.com.

About ARCALYST

ARCALYST is a weekly, subcutaneously injected recombinant dimeric fusion protein that blocks interleukin-1 alpha (IL-1α) and interleukin-1 beta (IL-1β) signaling. ARCALYST was discovered by Regeneron Pharmaceuticals, Inc. (Regeneron) and is approved by the U.S. Food and Drug Administration (FDA) for the treatment of recurrent pericarditis (RP) and reduction in risk of recurrence in adults and children 12 years and older. ARCALYST is also approved by the FDA for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Autoinflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS) in adults and children 12 years and older, and the maintenance of remission of Deficiency of Interleukin-1 Receptor Antagonist (DIRA) in adults and pediatric patients weighing 10 kg or more. The FDA granted Orphan Drug Exclusivity to ARCALYST upon its approval for recurrent pericarditis in 2021. The European Commission granted Orphan Drug Designation to ARCALYST for the treatment of idiopathic pericarditis in 2021.

IMPORTANT SAFETY INFORMATION ABOUT ARCALYST

  • ARCALYST may affect your immune system and can lower the ability of your immune system to fight infections. Serious infections, including life-threatening infections and death, have happened in patients taking ARCALYST. If you have any signs of an infection, call your doctor right away. Treatment with ARCALYST should be stopped if you get a serious infection. You should not begin treatment with ARCALYST if you have an infection or have infections that keep coming back (chronic infection).
  • While taking ARCALYST, do not take other medicines that block interleukin-1, such as Kineret® (anakinra), or medicines that block tumor necrosis factor, such as Enbrel® (etanercept), Humira® (adalimumab), or Remicade® (infliximab), as this may increase your risk of getting a serious infection.
  • Talk with your doctor about your vaccine history. Ask your doctor whether you should receive any vaccines before you begin treatment with ARCALYST.
  • Medicines that affect the immune system may increase the risk of getting cancer.
  • Stop taking ARCALYST and call your doctor or get emergency care right away if you have any symptoms of an allergic reaction.
  • Your doctor will do blood tests to check for changes in your blood cholesterol and triglycerides.
  • Common side effects include injection-site reactions (which may include pain, redness, swelling, itching, bruising, lumps, inflammation, skin rash, blisters, warmth, and bleeding at the injection site), upper respiratory tract infections, joint and muscle aches, rash, ear infection, sore throat, and runny nose.

For more information about ARCALYST, talk to your doctor and see the

Product Information

.

Forward-Looking Statements

This press release contains forward-looking statements. In some cases, you can identify forward looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions, although not all forward-looking statements contain these identifying words. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation, statements regarding: our expectation that ARCALYST 2025 net product revenue will be between $560 million and $580 million; our expectation to remain cash flow positive on an annual basis; our belief that there is still substantial opportunity ahead for Kiniksa to drive further ARCALYST revenue; our belief that our financial profile provides the ability to pursue additional value-creating opportunities; our beliefs about the mechanisms of our assets and potential impact of their approach; and our belief that all of our product candidates offer the potential for differentiation.

These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including without limitation, the following: delays or difficulty in enrollment of patients in, and activation or continuation of sites for, our clinical trials; delays or difficulty in completing our clinical trials as originally designed; potential for changes between final data and any preliminary, interim, top-line or other data from clinical trials; our inability to replicate results from our earlier clinical trials or studies; impact of additional data from us or other companies, including the potential for our data to produce negative, inconclusive or commercially uncompetitive results; potential undesirable side effects caused by our products and product candidates; our inability to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities; potential for applicable regulatory authorities to not accept our filings, delay or deny approval of any of our product candidates or require additional data or trials to support approval; our reliance on third parties as the sole source of supply of the drug substance and drug product used in our products and product candidates; raw material, important ancillary product and drug substance and/or drug product shortages; our reliance on third parties to conduct research, clinical trials, and/or certain regulatory activities for our product candidates; complications in coordinating requirements, regulations and guidelines of regulatory authorities across jurisdictions for our clinical trials; changes in our operating plan, business development strategy or funding requirements; and existing or new competition.

These and other important factors discussed in our filings with the U.S. Securities and Exchange Commission, including under the caption “Risk Factors” contained therein, could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. Except as required by law, we disclaim any intention or obligation to update or revise any forward-looking statements. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

ARCALYST® is a registered trademark of Regeneron Pharmaceuticals, Inc.


Every Second Counts!



®

Kiniksa Investor Contact

Jonathan Kirshenbaum
(781) 829-3949
[email protected]

Kiniksa Media Contact

Tyler Gagnon
(781) 431-9100
[email protected]



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Verastem Oncology Announces Debt Refinancing with Oberland Capital and Strategic Commercialization Partnership with IQVIA to Support Potential Launch in Recurrent KRAS Mutant Low-Grade Serous Ovarian Cancer in Mid-2025

Verastem Oncology Announces Debt Refinancing with Oberland Capital and Strategic Commercialization Partnership with IQVIA to Support Potential Launch in Recurrent KRAS Mutant Low-Grade Serous Ovarian Cancer in Mid-2025

Company cash, cash equivalents, and investments of $88.8 million as of December 31, 2024; pro forma cash position of $128.6 million including debt refinancing and equity issuance provides cash runway beyond FDA approval

BOSTON–(BUSINESS WIRE)–
Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today announced a new credit facility for up to $150 million along with an equity investment of $7.5 million with Oberland Capital Management LLC (Oberland Capital). In addition, the Company announced a strategic collaboration with IQVIA Inc. (IQVIA) to leverage IQVIA’s world-class infrastructure and established commercialization solutions to complement its launch strategy for the investigational combination of avutometinib plus defactinib for the treatment of recurrent KRAS mutant low-grade serous ovarian cancer (LGSOC) planned for mid-2025.

“The Oberland Capital transaction, coupled with our strategic partnership with IQVIA, enables us to launch avutometinib plus defactinib for recurrent LGSOC from a position of financial strength and with commercialization solutions to accelerate our launch. The additional capital will help us create a commercial revenue stream to support our pipeline with new approaches for patients needing treatments for complex and rare cancers,” said Dan Paterson, president and chief executive officer of Verastem Oncology.

Verastem’s commercialization efforts are in anticipation of potential U.S. Food and Drug Administration (FDA) approval of avutometinib plus defactinib in recurrent KRAS mutant LGSOC. The Company announced on December 30, 2024, that the FDA set a Prescription Drug User Fee Act (PDUFA) action date for its NDA submission of June 30, 2025.

Under the terms of the note purchase agreement with Oberland Capital, Verastem will issue an initial $75 million of notes at closing, which is expected to occur on January 13, 2025. The Company then has the ability to access up to an additional $75 million in notes upon achievement of certain pre-determined milestones related to the potential regulatory approval and commercialization of avutometinib plus defactinib for the treatment of LGSOC. The notes carry an interest-only period of six years and will bear interest at a floating rate, which is subject to both a floor and a cap. The note purchase agreement also provides for revenue participation pursuant to which Oberland Capital will initially be entitled to 1.0% of the first $100 million of net sales in each calendar year of certain of the Company’s products, subject to pro-rata increase upon potential future draw downs.

In addition, the Company has entered into a stock purchase agreement with affiliates of Oberland Capital for the private placement of 1,416,939 shares of the Company’s common stock issued at closing, representing $7.5 million of gross proceeds based on the trailing 30-trading days volume-weighted average price or VWAP of $5.2931 per share. Additionally, Oberland has the option to participate in certain future equity offerings that may be consummated by the Company within the three years from closing, for up to $2.5 million at the same price per share in such offering. Closing of the stock purchase agreement is expected to occur concurrently with the closing of the note purchase agreement on January 13, 2025. A portion of the proceeds from the notes and equity investment will be used to fully repay amounts owed under the Company’s existing loan with Oxford Finance ($42.7 million), which has been terminated.

The Company had a preliminary unaudited cash, cash equivalents, and short term investment balance of $88.8 million as of December 31, 2024. Taking into account the initial $75.0 million of notes and $7.5 million of equity to be purchased by Oberland Capital at closing, and the repayment of amounts owed under the Company’s existing loan with Oxford Finance, the Company would have had pro-forma cash, cash equivalents, and short-term investment balance of $128.6 million as of December 31, 2024. Both the actual and pro forma December 31, 2024 balances stated herein are preliminary, unaudited estimates and subject to revision upon completion of the Company’s closing and audit processes and do not present all information necessary for an understanding of the Company’s financial condition as of, and its results of operations for the fiscal year ended December 31, 2024. Additional details regarding this financing will be available in a Current Report on Form 8-K to be filed by the Company with the Securities and Exchange Commission.

The agreement between Verastem and IQVIA allows Verastem to tap into IQVIA’s industry-leading expertise and resources while maintaining strategic oversight through the commercialization process and launch. IQVIA will help accelerate key launch capabilities resulting in significant savings while delivering a world-class product launch.

About the Avutometinib and Defactinib ​​Combination

Avutometinib is an oral RAF/MEK clamp that potentially inhibits MEK1/2 kinase activities and induces inactive complexes of MEK with ARAF, BRAF, and CRAF, potentially creating a more complete and durable anti-tumor response through maximal RAS/MAPK pathway inhibition. In contrast to currently available MEK-only inhibitors, avutometinib blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows avutometinib to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of the MEK-only inhibitors.

Defactinib is an oral, selective inhibitor of focal adhesion kinase (FAK) and proline-rich tyrosine kinase-2 (Pyk2), the two members of the focal adhesion kinase family of non-receptor protein tyrosine kinases. FAK and Pyk2 integrate signals from integrin and growth factor receptors to regulate cell proliferation, survival, migration, and invasion. FAK activation has been shown to mediate resistance to multiple anti-cancer agents, including RAF and MEK inhibitors.

Verastem Oncology is currently conducting clinical trials with avutometinib with and without defactinib in RAS/MAPK-driven tumors as part of its Raf And Mek Program or RAMP. Verastem is currently enrolling patients and activating sites for RAMP 301 (GOG-3097/ENGOT-ov81/NCRI) (NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC).

Verastem was granted Priority Review and a Prescription Drug User Fee Act (PDUFA) date of June 30, 2025, for its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA), for the investigational combination of avutometinib and defactinib in adults with recurrent KRAS mutant LGSOC who received at least one prior systemic therapy. Verastem initiated a rolling NDA in May 2024 to the FDA and completed its NDA submission in October 2024. The FDA granted Breakthrough Therapy Designation for the treatment of patients with recurrent LGSOC after one or more prior lines of therapy, including platinum-based chemotherapy, in May 2021. Avutometinib alone or in combination with defactinib was also granted Orphan Drug Designation by the FDA for the treatment of LGSOC.

Verastem Oncology has established a clinical collaboration with Amgen to evaluate LUMAKRAS™ (sotorasib) in combination with avutometinib and defactinib in both treatment-naïve patients and in patients whose KRAS G12C mutant non-small cell lung cancer progressed on a G12C inhibitor as part of the RAMP 203 trial (NCT05074810). Verastem has received Fast Track Designation from the FDA for the triplet combination in April 2024. RAMP 205 (NCT05669482), a Phase 1b/2 clinical trial evaluating avutometinib and defactinib with gemcitabine/nab-paclitaxel in patients with front-line metastatic pancreatic cancer, is supported by the PanCAN Therapeutic Accelerator Award. FDA granted Orphan Drug Designation to the avutometinib and defactinib combination for the treatment of pancreatic cancer.

About Verastem Oncology

Verastem Oncology (Nasdaq: VSTM) is a late-stage development biopharmaceutical company committed to the development and commercialization of new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition and FAK inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn.

About Oberland Capital

Oberland Capital is a private investment firm formed in 2013 with assets under management in excess of $3.0 billion. The firm is focused exclusively on investing in the global healthcare industry and specializing in flexible investment structures customized to meet the specific needs of its transaction partners. Oberland Capital’s broad suite of financing solutions includes monetization of royalty streams, acquisition of future product revenues, creation of project-based financing structures, and investments in traditional debt and equity. With a combination of deep industry knowledge and extensive structured finance experience, the Oberland Capital team has a history of creating value for its transaction partners. For more information, please visit www.oberlandcapital.com or contact Johnna Schifilliti at (212) 257-5850.

Forward-Looking Statements

This press release includes forward-looking statements about, among other things, Verastem Oncology’s programs and product candidates, strategy, future plans and prospects, including statements related to the anticipated timing of closing an funding of the transactions with Oberland Capital, the expected outcome and benefits of the collaboration and the agreement between Verastem and IQVIA, the expected timing of further FDA action on the New Drug Application (NDA) for the avutometinib and defactinib combination product in KRAS-mutant and recurrent low-grade serous ovarian cancer, the potential clinical value of various of the Company’s clinical trials, interactions with regulators, the potential for and timing of commercialization of product candidates and potential for additional development programs involving Verastem Oncology’s lead compound. The words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” “can,” “promising” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause our actual results to differ materially from those expressed or implied in such statement. Applicable risks and uncertainties include the risks and uncertainties, among other things, regarding: the success in the development and potential commercialization of our product candidates, including avutometinib in combination with other compounds, including defactinib, LUMAKRAS™ and others; the uncertainties inherent in research and development, such as negative or unexpected results of clinical trials, the occurrence or timing of applications for our product candidates that may be filed with regulatory authorities in any jurisdictions; whether and when regulatory authorities in any jurisdictions may approve or reject any such applications that may be filed for our product candidates, and, if approved, whether our product candidates will be commercially successful in such jurisdictions; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the scope, timing, and outcome of any legal proceedings; decisions by regulatory authorities regarding trial design, labeling and other matters that could affect the timing, availability or commercial potential of our product candidates; whether preclinical testing of our product candidates and preliminary or interim data from clinical trials will be predictive of the results or success of ongoing or later clinical trials; the uncertainty around the timing, scope and rate of reimbursement for our product candidates; internal and third-party estimates about the market opportunities of our drug candidates may prove to be incorrect; third-party payors (including government agencies) may not reimburse; there may be competitive developments affecting our product candidates; data may not be available when expected; that enrollment of clinical trials may take longer than expected, which may delay our development programs, including delays in review by the FDA of our NDA submission in recurrent KRAS mutant LGSOC if enrollment in our confirmatory trial is not well underway at the time of submission, or that the FDA may require the Company to have completed enrollment or to enroll additional patients in the Company’s ongoing RAMP-301 confirmatory Phase 3 clinical trial prior to the FDA taking action on our NDA seeking accelerated approval; risks associated with preliminary and interim data, which may not be representative of more mature data, including with respect to interim duration of therapy data; our product candidates may cause adverse safety events and/or unexpected concerns may arise from additional data or analysis, or result in unmanageable safety profiles as compared to their levels of efficacy; we may be unable to successfully validate, develop and obtain regulatory approval for companion diagnostic tests for our product candidates that require or would commercially benefit from such tests, or experience significant delays in doing so; the mature RAMP 201 data and associated discussions with the FDA may not support the scope of our NDA submission for the avutometinib and defactinib combination in LGSOC, including with respect to both recurrent KRAS mutant and recurrent KRAS wild type LGSOC; our product candidates may experience manufacturing or supply interruptions or failures; any of our third party contract research organizations, contract manufacturing organizations, clinical sites, or contractors, among others, who we rely on may fail to fully perform; we face substantial competition, which may result in others developing or commercializing products before or more successfully than we do which could result in reduced market share or market potential for our product candidates; we may be unable to successfully initiate or complete the clinical development and eventual commercialization of our product candidates; the development and commercialization of our product candidates may take longer or cost more than planned, including as a result of conducting additional studies or our decisions regarding execution of such commercialization; we may not have sufficient cash to fund our contemplated operations, including certain of our product development programs; we may not attract and retain high quality personnel; we or Chugai Pharmaceutical Co., Ltd. may fail to fully perform under the avutometinib license agreement; the total addressable and target markets for our product candidates might be smaller than we are presently estimating; we or Secura Bio, Inc. (Secura) may fail to fully perform under the asset purchase agreement with Secura, including in relation to milestone payments; we may not see a return on investment on the payments we have and may continue to make pursuant to the collaboration and option agreement with GenFleet Therapeutics (Shanghai), Inc. (GenFleet), or that GenFleet may fail to fully perform under the agreement; we may not be able to establish new or expand on existing collaborations or partnerships, including with respect to in-licensing of our product candidates, on favorable terms, or at all; we may be unable to obtain adequate financing in the future through product licensing, co-promotional arrangements, public or private equity, debt financing or otherwise; we may not pursue or submit regulatory filings for our product candidates; our product candidates may not receive regulatory approval, become commercially successful products, or result in new treatment options being offered to patients; and that our final audited cash, cash equivalents, and short-term investments for the year ended December 31, 2024 may differ materially from the preliminary and unaudited amount reported herein.

Other risks and uncertainties include those identified under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission (SEC) on March 14, 2024 and in any subsequent filings with the SEC, which are available at www.sec.gov. As a result of these and other factors, we may not achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. The forward-looking statements contained in this press release reflect Verastem Oncology’s views as of the date hereof, and the Company does not assume and specifically disclaims any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by law.

For Investor and Media Inquiries:

Julissa Viana

Vice President, Corporate Communications and Investor Relations

[email protected] or

[email protected]

KEYWORDS: United States North America Massachusetts

INDUSTRY KEYWORDS: Oncology Health FDA Clinical Trials Pharmaceutical Biotechnology

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MakeMyTrip Limited to report Fiscal 2025 Third Quarter Financial & Operating Results on January 23, 2025

MakeMyTrip Limited to report Fiscal 2025 Third Quarter Financial & Operating Results on January 23, 2025

GURUGRAM, India & NEW YORK,–(BUSINESS WIRE)–
MakeMyTrip Limited (Nasdaq: MMYT) plans to report its fiscal 2025 third quarter financial and operating results before markets open on Thursday, January 23, 2025. The company will post the release on its Investor Relations website https://investors.makemytrip.com/and will not distribute over newswires.

A live Zoom Webinar with the senior management team will also be hosted on January 23, 2025, at 7:30 am EDT or 6:00 pm IST through the company’s Investor Relations website at https://investors.makemytrip.com/.

To register for the webinar, please follow this link: https://makemytrip.zoom.us/webinar/register/WN_1XMmOkMtROezj0BmKN2cYg

Registered participants will receive a confirmation email containing the Zoom access link and alternative phone dial-in details.

A replay of the event will be available on the company’s Investor Relations website approximately two hours after the conclusion of the live event.

About MakeMyTrip Limited

We own and operate well-recognized online travel brands, including MakeMyTrip, Goibibo and redBus. Through our primary websites, www.makemytrip.com, www.goibibo.com, www.redbus.in, and mobile platforms, travelers can research, plan and book a wide range of travel services and products in India and overseas. Our services and products include air ticketing, hotel and alternative accommodations bookings, holiday planning and packaging, bus ticketing, rail ticketing, car hire and ancillary travel requirements such as facilitating access to third-party travel insurance, forex services, and visa processing. We provide our customers with access to all major domestic full-service and low-cost airlines operating in India and all major airlines operating to and from India, a comprehensive set of domestic accommodation properties in India and a wide selection of properties outside of India, Indian Railways, and all major Indian bus operators.

For more details, please contact:

Vipul Garg

Vice President – Investor Relations: MakeMyTrip Limited

[email protected]

KEYWORDS: United States India North America Asia Pacific New York

INDUSTRY KEYWORDS: Apps/Applications Technology Mobile/Wireless Transportation Lodging Tourist Attractions Destinations Vacation Travel

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Cybin Highlights 2024 Accomplishments and Upcoming Milestones for 2025

Cybin Highlights 2024 Accomplishments and Upcoming Milestones for 2025

– Announced positive Phase 2 Data for CYB003, Demonstrating Breakthrough 12-Month Efficacy in Treating Major Depressive Disorder (“MDD”) –

– Initiated PARADIGM: A Multinational Pivotal Phase 3 Program Evaluating CYB003 for the Adjunctive Treatment of MDD –

– Received U.S. Food and Drug Administration (“FDA”) Breakthrough Therapy Designation (“BTD”) for CYB003 for the Adjunctive Treatment of MDD –

– Topline efficacy data from Phase 2 Proof-of-Concept Study of CYB004 in Generalized Anxiety Disorder (“GAD”) expected in Q1 2025 –

– Expanded clinical expertise in key roles to support clinical advancement and pipeline development –

TORONTO–(BUSINESS WIRE)–Cybin Inc. (NYSE American:CYBN) (Cboe CA:CYBN) (“Cybin” or the “Company”), a clinical-stage breakthrough neuropsychiatry company committed to revolutionizing mental healthcare by developing new and innovative next-generation treatment options, is pleased to provide a year-end summary of its key accomplishments in 2024 and upcoming milestones for 2025.

“In 2024, we made significant progress toward our goal of transforming the mental health treatment landscape,” said Doug Drysdale, Chief Executive Officer of Cybin. “Our clinical pipeline of tryptamine serotonin receptor agonists – notably CYB003, our proprietary deuterated psilocin program in development for the adjunctive treatment of MDD, and CYB004, our deuterated dimethyltryptamine, in intramuscular (“IM”) form being developed for the treatment of GAD – achieved several critical benchmarks.”

“It’s noteworthy that in March 2024, CYB003 received FDA Breakthrough Therapy Designation. Among its many benefits, this designation provides an expedited review pathway and the potential to accelerate drug development timelines. It also includes all “fast track” program features and acknowledges the significant unmet medical need for more effective treatments of MDD. This is especially gratifying in light of our Phase 2 study results of CYB003 in MDD, which showed remarkable efficacy at 12 months, including a 100% response rate and a 71% remission rate among participants who received two 16 mg doses. With these outstanding findings in hand, we initiated PARADIGM™, our multinational pivotal Phase 3 program, evaluating CYB003 in MDD patients. We also commenced our Phase 2 proof-of-concept study of CYB004 in GAD. I’m proud of our Research and Development team and the tremendous progress made in a relatively short amount of time.”

“Looking ahead, we are excited about Cybin’s next phase of investigation and growth and for the upcoming milestones, including a readout of topline efficacy data from our CYB004 Phase 2 GAD study in the first quarter of 2025 and the initiation of the EXTEND and EMBRACE components of our Phase 3 PARADIGM program,” concluded Drysdale.

2024 Highlights:

CYB003

  • Announced unprecedented 12-month efficacy data from the completed Phase 2 study of CYB003
  • 100% of participants receiving two doses of 16 mg were responders

  • 71% of participants receiving two doses of 16 mg were in remission

  • Mean change from baseline in MADRS was ~23 points after two doses of 16 mg

  • Initiated PARADIGM, a multinational pivotal Phase 3 program evaluating CYB003 for the adjunctive treatment of MDD
  • PARADIGM comprises two 12-week randomized, placebo-controlled studies (APPROACH™ and EMBRACE™) and a long-term extension study (EXTEND)

    • APPROACH has been initiated and will enroll 220 participants at 36 clinical sites across the U.S. and Europe; topline results expected in 2026

    • EMBRACE will enroll 330 participants at sites across the U.S. and Europe and is expected to commence by mid 2025

    • EXTEND is expected to begin 12 weeks after commencement of APPROACH and EMBRACE, respectively

  • Appointed senior clinical team leaders and expanded its clinical operations team in support of its PARADIGM program. The new team members provide significant expertise and decades of experience across critical domains that are integral to the pivotal Phase 3 program.
  • Received additional U.S. patent in support of its CYB003 Breakthrough Therapy Program. The patent is expected to provide exclusivity until at least 2041 and includes claims to pharmaceutical compositions within the Company’s proprietary deuterated psilocin analog program. This brings the total number of granted patents to over 70, with more than 220 patents currently pending.

CYB004

  • Initiated Phase 2 proof-of-concept study of CYB004 in GAD.
  • The Phase 2 study is a randomized, double-blind study evaluating the safety and efficacy of CYB004 in participants with GAD, with concomitant antidepressant/anxiolytic treatment and co-morbid depression allowed

  • The Phase 2 study is being conducted at sites in the U.S., with topline safety and efficacy results expected in Q1 2025

Presented two posters at the American College of Neuropsychopharmacology (“ACNP”) Meeting. The data presented include 12-month efficacy results from the Company’s Phase 2 study of CYB003, its deuterated psilocin program, in MDD, which showed durable response and remission rates at 12 months, in addition to results from a completed Phase 1b study exploring drug-drug interactions between N,N-dimethyltryptamine (“DMT”) and selective serotonin reuptake inhibitors (“SSRIs”), in MDD patients.

CYB005

  • Announced grant of first U.S. Composition of Matter patent in support of its CYB005 phenethylamines program. Cybin is investigating novel molecules within the CYB005 program at non-hallucinogenic doses for a range of Central Nervous System disorders and continues to explore non-hallucinogenic neuroplastogens within its broader discovery pipeline.

First-Half 2025 Expected Milestones

  • Topline efficacy data readout from CYB004 Phase 2 GAD study in Q1 2025

  • Initiate EXTEND and EMBRACE pivotal studies of CYB003

About Cybin

Cybin is a late-stage breakthrough neuropsychiatry company committed to revolutionizing mental healthcare by developing new and innovative next-generation treatment options to address the large unmet need for people who suffer from mental health conditions.

With industry leading proof-of-concept data, Cybin is working to change the mental health treatment landscape through the introduction of intermittent treatments that provide long lasting results. The Company is currently developing CYB003, a proprietary deuterated psilocin analog, in Phase 3 studies for the adjunctive treatment of major depressive disorder and CYB004, a proprietary deuterated N, N-dimethyltryptamine molecule in a Phase 2 study for generalized anxiety disorder. The Company also has a research pipeline of investigational, 5-HT-receptor focused compounds.

Founded in 2019, Cybin is operational in Canada, the United States, the United Kingdom, the Netherlands and Ireland. For Company updates and to learn more about Cybin, visit www.cybin.com or follow the team on X, LinkedIn, YouTube and Instagram.

Cautionary Notes and Forward-Looking Statements

Certain statements in this news release relating to the Company are forward-looking statements and are prospective in nature. Forward-looking statements are not based on historical facts, but rather on current expectations and projections about future events and are therefore subject to risks and uncertainties which could cause actual results to differ materially from the future results expressed or implied by the forward-looking statements. These statements generally can be identified by the use of forward-looking words such as “may”, “should”, “could”, “potential”, “possible”, “intend”, “estimate”, “plan”, “anticipate”, “expect”, “believe” or “continue”, or the negative thereof or similar variations. Forward-looking statements in this news release include statements regarding the Company’s plans to report Phase 2 topline results for CYB004 in Q1 2025; timing of the initiation and ability of the Company to enroll participants for the PARADIGM program; the potential for BTD to accelerate drug development timelines; and the Company’s plans to engineer proprietary drug discovery platforms, innovative drug delivery systems, novel formulation approaches and treatment regimens for mental health conditions.

These forward-looking statements are based on reasonable assumptions and estimates of management of the Company at the time such statements were made. Actual future results may differ materially as forward-looking statements involve known and unknown risks, uncertainties, and other factors which may cause the actual results, performance, or achievements of the Company to materially differ from any future results, performance, or achievements expressed or implied by such forward-looking statements. Such factors, among other things, include: fluctuations in general macroeconomic conditions; fluctuations in securities markets; expectations regarding the size of the psychedelics market; the ability of the Company to successfully achieve its business objectives; plans for growth; political, social and environmental uncertainties; employee relations; the presence of laws and regulations that may impose restrictions in the markets where the Company operates; implications of disease outbreaks on the Company’s operations; and the risk factors set out in each of the Company’s management’s discussion and analysis for the three and six month ended September 30, 2024 and the Company’s annual information form for the year ended March 31, 2024, which are available under the Company’s profile on SEDAR+ at www.sedarplus.ca and with the U.S. Securities and Exchange Commission on EDGAR at www.sec.gov. Although the forward-looking statements contained in this news release are based upon what management of the Company believes, or believed at the time, to be reasonable assumptions, the Company cannot assure shareholders that actual results will be consistent with such forward-looking statements, as there may be other factors that cause results not to be as anticipated, estimated or intended. Readers should not place undue reliance on the forward-looking statements contained in this news release. The Company assumes no obligation to update the forward-looking statements of beliefs, opinions, projections, or other factors, should they change, except as required by law.

Cybin makes no medical, treatment or health benefit claims about Cybin’s proposed products. The U.S. Food and Drug Administration, Health Canada or other similar regulatory authorities have not evaluated claims regarding psilocin, psychedelic tryptamine, tryptamine derivatives or other psychedelic compounds. The efficacy of such products has not been confirmed by approved research. There is no assurance that the use of psilocin, psychedelic tryptamine, tryptamine derivatives or other psychedelic compounds can diagnose, treat, cure or prevent any disease or condition. Rigorous scientific research and clinical trials are needed. If Cybin cannot obtain the approvals or research necessary to commercialize its business, it may have a material adverse effect on Cybin’s performance and operations.

Neither Cboe Canada, nor the NYSE American LLC stock exchange have approved or disapproved the contents of this news release and are not responsible for the adequacy and accuracy of the contents herein.

Investor & Media Contact:

Gabriel Fahel

Chief Legal Officer

Cybin Inc.

1-866-292-4601

[email protected] – or – [email protected]

KEYWORDS: North America Canada

INDUSTRY KEYWORDS: Mental Health Health Neurology Clinical Trials Pharmaceutical Biotechnology

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Dexcom Reports Preliminary, Unaudited Results for the Fourth Quarter and Fiscal Year 2024 and Initial 2025 Outlook

Dexcom Reports Preliminary, Unaudited Results for the Fourth Quarter and Fiscal Year 2024 and Initial 2025 Outlook

SAN DIEGO–(BUSINESS WIRE)–
DexCom, Inc. (Nasdaq: DXCM) today reported preliminary, unaudited results for the fourth quarter ended December 31, 2024 with total revenue of at least $1.113 billion, an increase of 8% over the fourth quarter of 2023. U.S. revenue is expected to be approximately $803 million, representing growth of 4% over the fourth quarter of 2023. International revenue is expected to be approximately $310 million, an increase of 17% over the fourth quarter of 2023.

For fiscal 2024, total preliminary, unaudited revenue is approximately $4.032 billion, an increase of 11% over 2023. In conjunction with the preliminary fourth quarter results, the company is also updating 2024 non-GAAP gross profit margin and non-GAAP operating margin guidance to 62% and 19% respectively. Fourth quarter gross margin is expected to be adversely impacted by a non-cash charge primarily related to inventory damaged in transit and certain build configurations that lowered production yield for the quarter.

“Dexcom made key strategic investments in 2024 that steadily progressed throughout the year, leaving us well positioned to capitalize on our growth opportunity ahead,” said Kevin Sayer, Dexcom’s chairman, president and CEO. “We plan to build on these investments in 2025 by further enhancing our differentiated product portfolio and advocating for greater CGM access globally.”

2025 Outlook

For 2025, Dexcom currently anticipates total revenue of $4.60 billion, representing expected growth of approximately 14% over 2024. This outlook considers sensor volume growth driven by increasing CGM access and awareness for people with diabetes, the continued rollout of Stelo, further international expansion, and overall market dynamics.

In addition, Dexcom currently estimates 2025 Non-GAAP Gross Profit Margin and Non-GAAP Operating Margin to be at the following levels:

  • Non-GAAP Gross Profit Margin of approximately 64 – 65%

  • Non-GAAP Operating Margin of approximately 21%

Fourth Quarter 2024 Financial Results Conference Call

Dexcom will report its audited full fourth quarter and fiscal 2024 financial results on Thursday, February 13, 2025 after the close of market. Management is currently scheduled to host a conference call at 4:30 p.m. (Eastern Time) that day. More details will be provided later.

About DexCom, Inc.

Dexcom empowers people to take control of health through innovative biosensing technology. Founded in 1999, Dexcom has pioneered and set the standard in glucose biosensing for more than 25 years. Its technology has transformed how people manage diabetes and track their glucose, helping them feel more in control and live more confidently.

Dexcom. Discover what you’re made of. For more information, visit www.dexcom.com.

Category: IR

Statement Regarding Use of Non-GAAP Financial Measures

This press release includes non-GAAP financial measures. We have not reconciled our Non-GAAP Gross Profit Margin and Non-GAAP Operating Margin guidance for fiscal years 2024 and 2025 because certain items that impact these figures are uncertain or out of our control and cannot be reasonably predicted. Accordingly, a reconciliation of Non-GAAP Gross Profit Margin and Non-GAAP Operating Margin is not available without unreasonable effort.

Cautionary Statement Regarding Forward Looking Statements

This press release contains forward-looking statements that are not purely historical regarding Dexcom’s or its management’s intentions, beliefs, expectations and strategies for the future, including those related to Dexcom’s preliminary, unaudited revenue for the fourth quarter of and the full fiscal year 2024, including growth rates; total revenue guidance for fiscal 2025; Non-GAAP Gross Profit Margin and Non-GAAP Operating Margin guidance for fiscal 2024 and 2025, including expected growth rates; and potential strategic and business opportunities. All forward-looking statements included in this press release are made as of the date of this release, based on information currently available to Dexcom as of the date hereof. Forward-looking statements deal with future events and therefore are subject to various risks and uncertainties. Actual results could differ materially from those anticipated in these forward-looking statements. The risks and uncertainties that may cause actual results to differ materially from Dexcom’s current expectations are more fully described in the sections titled “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in Dexcom’s most recently filed periodic reports on Form 10-K and Form 10-Q and subsequent filings. Except as required by law, Dexcom assumes no obligation to update any such forward-looking statement after the date of this communication or to conform these forward-looking statements to actual results.

Dexcom, Dexcom Clarity, Dexcom Follow, Dexcom One, Dexcom Share, Stelo, and any related logos and design marks are either registered trademarks or trademarks of Dexcom, Inc. in the United States and/or other countries.

FOR MORE INFORMATION:

Sean Christensen

Vice President, Finance and Investor Relations

(858) 203-6657

www.dexcom.com

KEYWORDS: California United States North America

INDUSTRY KEYWORDS: Medical Devices Health Diabetes Health Technology General Health Biotechnology

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Redwire Successfully Delivers Fourth Pair of Roll-Out Solar Array Wings for ISS Power

Redwire Successfully Delivers Fourth Pair of Roll-Out Solar Array Wings for ISS Power

JACKSONVILLE, Fla.–(BUSINESS WIRE)–
Redwire Corporation (NYSE: RDW), a leader in space infrastructure for the next generation space economy, announced today the successful delivery of the fourth pair of Roll-Out Solar Array (ROSA) wings for the International Space Station (ISS), also known as the IROSA program. The wings were developed and delivered through a follow-on contract with Boeing, NASA’s prime contractor for ISS operations.

The IROSA wings enhance the space station’s power supply to support critical research and space operations. The fourth pair of wings were developed in partnership with Boeing’s Spectrolab and delivered through a follow-on contract awarded in June 2022. Since 2021, a total of six Redwire-built arrays have been deployed on the ISS, modernizing its critical power supply.

“With eight IROSA wings produced, and six currently deployed and powering the ISS to date, IROSA is a proven technology for powering sustained human activities in space,” said Peter Cannito, Redwire Chairman and CEO. “The technical success of the program paves the way for new opportunities for commercial space stations and sustained power infrastructure on the Moon and beyond.”

The delivery of the most recent set of IROSA wings follows acceptance testing consisting of multiple ambient functional deployments, vibration, and cold and hot temperature deployments. The wings are undergoing flight package integration in the Space Station Processing Facility at NASA’s Kennedy Space Center in Florida and are slated to launch on an upcoming commercial resupply mission to the ISS.

Each wing provides an additional 20+ kW of power for over 10 years once deployed, and all eight IROSA wings combined will provide more than 160 kW. Since installation on the ISS, the six currently deployed wings have operated nominally, enabling extended operation of ISS to 2030 and beyond.

Redwire has continued to advance ROSA technology to power other spaceflight platforms and ambitious missions with reliable and stable power solutions for civil, DoD, and commercial customers. Redwire is currently building ROSA systems for the Power and Propulsion Element for NASA’s Gateway program, a part of the agency’s Artemis program, and Astrobotic’s Lunar Vertical Solar Array program, which aims to provide sustainable power on the lunar surface. Along with its previous success on the ISS, Redwire’s ROSA technology also powered NASA’s DART spacecraft to impact the asteroid Dimorphos, successfully altering the asteroid’s orbit in September 2022.

About Redwire

Redwire Corporation (NYSE:RDW) is a global space infrastructure and innovation company enabling civil, commercial, and national security programs. Redwire’s proven and reliable capabilities include avionics, sensors, power solutions, critical structures, mechanisms, radio frequency systems, platforms, missions, and microgravity payloads. Redwire combines decades of flight heritage and proven experience with an agile and innovative culture. Redwire’s approximately 700 employees working from 17 facilities located throughout the United States and Europe are committed to building a bold future in space for humanity, pushing the envelope of discovery and science while creating a better world on Earth. For more information, please visit redwirespace.com.

Media Contact:

Emily Devine

[email protected]

+1 305-632-9137

OR

Investors:

[email protected]

+1 904-425-1431

KEYWORDS: Florida United States North America

INDUSTRY KEYWORDS: Satellite Technology Aerospace Batteries Manufacturing Other Technology Hardware

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Volato to Take Delivery of Second Gulfstream G280 in January 2025, Leveraging Strong Market Demand for Premium Jets

Volato to Take Delivery of Second Gulfstream G280 in January 2025, Leveraging Strong Market Demand for Premium Jets

ATLANTA–(BUSINESS WIRE)–
Volato Group, Inc. (the “Company” or “Volato”) (NYSE American: SOAR), a leader in private aviation, today announced the upcoming delivery of its second Gulfstream G280 in January 2025. This milestone is part of a four-aircraft order and reflects Volato’s strategy to capitalize on strong market demand for premium super-midsize jets.

The Gulfstream G280 remains one of the most desirable aircraft in its class, with a range of up to 3,600 nautical miles, advanced performance, and a spacious, luxurious cabin. As the private aviation market faces constrained supply and significant backlogs, Volato’s ability to secure timely deliveries creates a unique opportunity to meet rising customer demand through aircraft sales.

“This delivery marks the second Gulfstream G280 from our four-aircraft order, and we are well-positioned to realize value from these transactions,” said Matt Liotta, CEO of Volato. “The demand for high-quality jets like the G280 remains incredibly strong, and these deliveries allow us to efficiently meet market needs while continuing to drive value for our stakeholders.”

The Gulfstream G280 Advantage:

  • Incredible Performance: A range of 3,600 nautical miles and top cruising speed of Mach 0.85, enabling non-stop coast-to-coast and transcontinental flights.
  • Luxurious Cabin: A spacious, quiet environment with advanced features for work and relaxation.
  • Market Opportunity: Delays across the private aviation industry have created a strong appetite for immediate deliveries of premium jets, allowing Volato to maximize the value of its four-aircraft order.

The delivery of Volato’s second G280 reinforces the company’s focus on strategically aligning its aircraft portfolio with market opportunities. With two additional G280s scheduled for later in 2025, Volato is positioned to benefit from ongoing demand while continuing to deliver value to its customers and shareholders.

About Volato

Volato (NYSE American: SOAR) is a private aviation company advancing the industry with innovative solutions in aviation software and on-demand flight access. Volato’s proprietary Mission Control software drives efficiency across operations and supports operators in managing fractional ownership, charter, and other services. Volato’s Vaunt platform connects travelers with available private flights, offering a flexible option for on-demand travel. With a commitment to advanced technology and customer-focused solutions, Volato is building scalable tools to elevate service quality and operational effectiveness in private aviation.

For more information about Volato, please visit www.flyvolato.com.

For Media:

[email protected]

For Investors:

[email protected]

KEYWORDS: Georgia United States North America

INDUSTRY KEYWORDS: Air Transport Aerospace Manufacturing Transportation Travel

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