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Results support potential benefit of efruxifermin (EFX) to elicit fibrosis improvement in patients with compensated cirrhosis (F4 fibrosis) due to MASH 
96-week data from SYMMETRY trial presented at EASL Congress 2025

SOUTH SAN FRANCISCO, Calif., May 09, 2025 (GLOBE NEWSWIRE) — Akero Therapeutics, Inc. (Nasdaq: AKRO), a clinical-stage company developing transformational treatments for patients with serious metabolic diseases marked by high unmet medical need, today announced publication of results from the Phase 2b SYMMETRY trial in the New England Journal of Medicine.

The publication reports results from the 96-week SYMMETRY study evaluating the efficacy and safety of Akero’s lead FGF21 analog efruxifermin (EFX), in participants with biopsy-confirmed compensated cirrhosis (F4), Child-Pugh Class A, caused by metabolic dysfunction-associated steatohepatitis (MASH).

“Publication of the Phase 2b SYMMETRY trial results in the New England Journal of Medicine is a significant milestone that affirms the strength of our clinical data and the potentially transformative nature of EFX in the treatment of patients with compensated cirrhosis due to MASH,” said Kitty Yale, chief development officer of Akero. “We remain encouraged by the potential benefits of treatment with EFX observed in the study, including unprecedented fibrosis improvement in patients with compensated cirrhosis due to MASH after 96 weeks of treatment, and look forward to continuing advancement of our ongoing Phase 3 SYNCHRONY program.”

The primary endpoint was ≥1-stage fibrosis improvement without MASH worsening at 36 weeks of treatment, with secondary outcomes of fibrosis improvement without MASH worsening at week 96 and MASH resolution at weeks 36 and 96. Using an intent-to-treat (ITT) analysis of the 36 week results, for which participants with missing biopsies were included as non-responders, 19% of participants in the EFX 50mg group and 18% of participants in the EFX 28mg group met the primary endpoint, compared to 13% for placebo. At week 96, using the same ITT analysis, 29% of participants in the EFX 50mg group and 21% of participants in the EFX 28mg group had fibrosis improvement without MASH worsening, compared to 11% in the placebo group. Most participants in the EFX groups with a fibrosis improvement at week 36 appeared to maintain their response at week 96, with additional new responders observed at week 96, particularly for the EFX 50mg group.

EFX was also associated with improvements in noninvasive markers of liver injury and fibrosis, as well as markers of insulin sensitivity and lipid metabolism compared with placebo at week 96.

The safety and tolerability of EFX observed in the SYMMETRY trial was consistent with previous trials. Observed adverse events, more common with EFX than placebo, were primarily gastrointestinal (e.g., diarrhea and nausea) or injection site related, with the majority being mild or moderate and transient in nature.

About Akero Therapeutics
Akero Therapeutics is a clinical-stage company developing transformational treatments for patients with serious metabolic diseases marked by high unmet medical need, including metabolic dysfunction-associated steatohepatitis (MASH). Akero’s lead product candidate, efruxifermin (EFX), is currently being evaluated in three ongoing Phase 3 clinical studies: SYNCHRONY Histology in patients with pre-cirrhotic (F2-F3 fibrosis) MASH, SYNCHRONY Outcomes in patients with compensated cirrhosis (F4) due to MASH, and SYNCHRONY Real-World in patients with MASH or MASLD (metabolic dysfunction-associated steatotic liver disease). The Phase 3 SYNCHRONY program builds on the results of two Phase 2b clinical trials, the HARMONY study in patients with pre-cirrhotic MASH and the SYMMETRY study in patients with compensated cirrhosis due to MASH. Akero is headquartered in South San Francisco. Visit us at akerotx.com and follow us on LinkedIn and X for more information.

About MASH
MASH is a serious form of MASLD that is estimated to affect 17 million Americans. MASH is characterized by an excessive accumulation of fat in the liver that causes stress and injury to liver cells, leading to inflammation and fibrosis, which can progress to cirrhosis, liver failure, cancer and eventually death. Approximately 20% of patients with MASH will progress to cirrhosis, which has a higher risk of mortality. There are no approved treatments for the condition and MASH is the fastest growing cause of liver transplants and liver cancer in the US and Europe.

About Cirrhosis Due to MASH 
Cirrhosis due to MASH (metabolic dysfunction-associated steatohepatitis) is a life-threatening disease with high risk of liver failure, cancer, and death. By 2030, an estimated 3 million Americans are projected to have MASH cirrhosis, which is the fastest growing cause of liver transplants and liver cancer in the United States and Europe.

About the SYMMETRY Trial 
SYMMETRY was a Phase 2b, multicenter, randomized, double-blind, placebo-controlled, dose-ranging trial in adult patients with biopsy-confirmed compensated cirrhosis (F4, Child-Pugh A) due to MASH. The study randomized 182 patients, and 181 received once-weekly subcutaneous EFX 28mg or 50mg, or placebo for 96 weeks. The primary efficacy endpoint was the proportion of patients with ≥1-stage fibrosis improvement without worsening of MASH at Week 36. Secondary efficacy measures at Week 96 included ≥1 stage fibrosis improvement without worsening of MASH, MASH resolution, change from baseline in liver enzymes, noninvasive markers of liver fibrosis, serum markers of glucose and lipid metabolism, as well as safety and tolerability measures.

About EFX

Efruxifermin (EFX), Akero’s lead product candidate for MASH, is currently being evaluated in three ongoing Phase 3 studies. In multiple Phase 2 studies, EFX has been observed to reverse fibrosis (including compensated cirrhosis), resolve MASH, reduce non-invasive markers of fibrosis and liver injury, and improve insulin sensitivity and lipoprotein profile. This holistic profile offers the potential to address the complex, multi-system disease state of all stages of MASH, including improvements in lipoprotein risk factors linked to cardiovascular disease – the leading cause of death among MASH patients. Engineered to mimic the biological activity profile of native FGF21, EFX is designed to offer convenient once-weekly dosing and has been generally well-tolerated in clinical trials to date.

Forward Looking Statements
 

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements, including, but not limited to, statements regarding Akero’s business plans and objectives; the potential transformative nature and therapeutic effects of EFX, as well as the dosing, safety and tolerability of EFX; the future potential and long-term benefits of EFX following the preliminary topline week 96 results of Akero’s Phase 2b SYMMETRY study; and the ongoing SYNCHRONY Phase 3 program,. Any forward-looking statements in this press release are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost, and timing of Akero’s product candidate development activities and planned clinical trials; Akero’s ability to execute on its strategy; positive results from any of its clinical studies may not necessarily be predictive of the results of future or ongoing clinical studies; regulatory developments in the United States and foreign countries; Akero’s ability to fund operations; as well as those risks and uncertainties set forth more fully under the caption “Risk Factors” in Akero’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q, as filed with the Securities and Exchange Commission (SEC) as well as discussions of potential risks, uncertainties and other important factors in Akero’s other filings and reports with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Akero undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Investor Contact:

Christina Tartaglia
Precision AQ
212.362.1200
[email protected]

Media Contact:

Peg Rusconi
Deerfield Group
617.910.6217
[email protected]

ALPHARETTA, Ga., May 09, 2025 (GLOBE NEWSWIRE) — Clearside Biomedical, Inc. (Nasdaq: CLSD) (“Clearside” or the “Company”), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS^®), announced today that six presentations related to the Company’s lead program, CLS-AX (axitinib injectable suspension), and suprachoroidal drug delivery platform were featured at the Association for Research in Vision and Ophthalmology (ARVO) 2025 Meeting.

Victor Chong, MD, MBA, Chief Medical Officer and EVP, Head of Research and Development, commented, “We are very pleased with the reception we received at ARVO this year for both our positive CLS-AX Phase 2b ODYSSEY data and our Phase 3 plans, along with our deep dive into our proficiency with suprachoroidal space (SCS^®) delivery. Preclinical and clinical trial results demonstrate that CLS-AX has the potential to be a safe and long-acting therapy for wet AMD given its durability, intrinsic high potency, pan-VEGF inhibition, and proven ability to re-dose.”

“It is evident that our expertise in SCS delivery, as substantiated by our advances in drug formulation and device optimization utilizing proprietary training models and segmentation algorithms for our SCS Microinjector^®, establishes Clearside as the recognized leader in SCS drug delivery. These data position SCS drug delivery as a transformative approach and promising mainstream option in treating retinal diseases,” concluded Dr. Chong.

In addition to the presentations, Viral Kansara, PhD, Vice President, Preclinical Development moderated an ARVO session entitled “Retina/RPE: New drugs, mechanisms of action, and toxicity” and Dr. Chong was featured on a Wet AMD/DR/RVO panel session at Retina Unplugged, a pre-conference of the Retina World Congress.

Presentation Key Highlights

• Positive data presented from the CLS-AX Phase 2b ODYSSEY trial, which achieved the primary outcome of maintaining stable best corrected visual acuity (BCVA) with repeat dosing while meaningfully reducing the frequency of injections.
• Clearside’s Phase 3 trial design to feature the flexible dosing of an anti-VEGF biologic with the duration of a pan-VEGF receptor tyrosine kinase inhibitor (TKI).
• Poster featuring data on CLS-AX, XIPERE^®, and viral and non-viral gene therapy data showed that suprachoroidal drug and gene therapy delivery with Clearside’s SCS Microinjector^® holds a promising future for treating chorioretinal diseases.
• Clearside has developed the first ever machine learning algorithm for imaging the opening of the SCS after drug delivery for evaluating patient data.
• Clearside has developed a novel force analysis method designed to provide real-time, formulation-specific feedback that goes beyond ISO compliance, evaluate the injectability of suspensions and allow for better drug-device co-optimization. Clearside leverages these test methods to design internal drug candidate formulations and assist partners with designs specifically for suprachoroidal injection using the SCS Microinjector.
• A decade-long literature review of suprachoroidal delivery across preclinical studies and clinical trials provides key evidence to support the suprachoroidal platform as an opportunity to expand the standard of care in the treatment of macular diseases.
• The suprachoroidal injection training program with the SCS Microinjector is the only clinically validated suprachoroidal training program and over 15,000 injections have been completed to date.

Presentation Details

Title: Top Line Results from ODYSSEY: A Phase 2b Study of Suprachoroidally Administered CLS-AX in Participants with Neovascular Age-related Macular Degeneration

Lead Author: Robert Wang, MD, Texas Retina Associates

Title: Suprachoroidal CLS-AX (Axitinib Injectable Suspension) Offers Durability, Safety, And Therapeutic Potential for Neovascular Age-Related Macular Degeneration (nAMD) Patients: Preclinical and Clinical Corroboration

Lead Author: Viral Kansara, PhD, Clearside Biomedical

Title: The Evolving Role of Suprachoroidal Drug Delivery in Macular Diseases: A Decade-Long Literature Review

Lead Author: Victor Chong, MD, MBA, Clearside Biomedical

Title: Validation of Suprachoroidal Injection Training Program with a Synthetic Eye Model

Lead Author: Chen-rei Wan, PhD, Clearside Biomedical

Title: Dispensability Analysis of Suspension Formulations

Lead Author: Darrin Rountree, MS, Clearside Biomedical

Title: Novel Deep Learning Algorithm for Suprachoroidal Space Segmentation and Measurement in Optical Coherence Tomography

Lead Author: Oluwagbemisola Aderibigbe, Georgia Institute of Technology

The presentations will be available on Clearside’s website here.

About CLS-AX (axitinib injectable suspension)

Clearside is developing CLS-AX as a longer-acting therapy for the treatment of retinal diseases. CLS-AX (axitinib injectable suspension) is a proprietary suspension of axitinib for suprachoroidal injection. Axitinib is a tyrosine kinase inhibitor (TKI), currently approved as an oral tablet formulation to treat advanced renal cell carcinoma, that achieves pan-VEGF blockade, directly inhibiting VEGF receptors-1, -2, and -3 with high potency and specificity. Clearside believes this broad VEGF blockade may have efficacy advantages over existing retinal therapies by acting at a different level of the angiogenesis cascade and may benefit patients who sub-optimally respond to current, more narrowly focused anti-VEGF therapies. Suprachoroidal injection of this proprietary suspension of axitinib has demonstrated meaningful potential in Phase 1/2a and Phase 2b wet AMD clinical trials in which CLS-AX was well tolerated and demonstrated a positive safety profile. With suprachoroidal administration of axitinib, there is the potential to achieve prolonged duration and targeted delivery to affected tissue layers by compartmentalizing axitinib behind the retina, thereby limiting drug exposure to the front of the eye.

About Clearside’s Suprachoroidal Space (SCS
^®
) Injection Platform and SCS Microinjector
^®

Clearside’s patent protected, proprietary suprachoroidal space (SCS^®) injection treatment approach offers unprecedented access to the back of the eye, where sight-threatening disease often occurs. The Company’s unique platform is inherently flexible and intended to work with established and new formulations of medications. Clearside’s patented SCS Microinjector^® can deliver a wide variety of drug candidates into the suprachoroidal space, providing targeted delivery to potentially improve efficacy and compartmentalization of medication to reduce or eliminate toxic effects on non-diseased cells. The SCS Microinjector is comprised of a syringe with a custom-designed hub and two 30-gauge hollow microneedles of varying lengths, each approximately one millimeter, optimizing insertion and suprachoroidal administration of drugs.

About Clearside Biomedical, Inc.

Clearside Biomedical, Inc. is a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS^®) to improve patient outcomes. Clearside’s SCS injection platform, utilizing the Company’s patented SCS Microinjector^®, enables an in-office, repeatable, non-surgical procedure for the targeted and compartmentalized delivery of a wide variety of therapies to the macula, retina, or choroid to potentially preserve and improve vision in patients with sight-threatening eye diseases. Clearside is developing its own pipeline of small molecule product candidates for administration via its SCS Microinjector. The Company’s lead program, CLS-AX (axitinib injectable suspension), is in development for the treatment of neovascular age-related macular degeneration (wet AMD). Planning for a Phase 3 program is underway. In addition, Clearside is evaluating various small molecules for the potential long-acting treatment of geographic atrophy (GA). Clearside developed and gained approval for its first product, XIPERE^® (triamcinolone acetonide injectable suspension) for suprachoroidal use, which is available in the U.S. through a commercial partner. Clearside also strategically partners its SCS injection platform with companies utilizing other ophthalmic therapeutic innovations. For more information, please visit clearsidebio.com or follow us on LinkedIn and X.

Cautionary Note Regarding Forward-Looking Statements

Any statements contained in this press release that do not describe historical facts may constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as “believe”, “expect”, “may”, “plan”, “potential”, “will”, and similar expressions, and are based on Clearside’s current beliefs and expectations. These forward-looking statements include statements regarding the clinical development of Clearside’s product candidates, including the Phase 3 design, the potential benefits of novel analysis methods and machine learning algorithms, and the potential benefits of CLS-AX and XIPERE^®, Clearside’s suprachoroidal delivery technology and Clearside’s SCS Microinjector^®. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Risks and uncertainties that may cause actual results to differ materially include uncertainties inherent in the conduct of clinical trials, Clearside’s reliance on third parties over which it may not always have full control, Clearside’s ability to raise additional capital, and other risks and uncertainties that are described in Clearside’s Annual Report on Form 10-K for the year ended December 31, 2024, filed with the U.S. Securities and Exchange Commission (SEC) on March 27, 2025, and Clearside’s other periodic reports filed with the SEC. Any forward-looking statements speak only as of the date of this press release and are based on information available to Clearside as of the date of this release, and Clearside assumes no obligation to, and does not intend to, update any forward-looking statements, whether as a result of new information, future events or otherwise.

Investor and Media Contacts:

Jenny Kobin
Remy Bernarda
[email protected]

Source: Clearside Biomedical, Inc.

IRF5 program strengthens Kymera’s oral immunology pipeline with a complementary mechanism to expand into rheumatic and other autoimmune diseases with a potential best-in-class oral drug

IRF5, a historically undrugged transcription factor and master regulator of immunity, has strong genetic and clinical pathway validation across multiple diseases including RA, SLE, IBD and others

KT-579, a potent, selective, oral degrader of IRF5 with an excellent profile in preclinical safety studies, has demonstrated activity comparable or superior to approved and clinically active drugs in multiple efficacy animal models of lupus and RA

IND-enabling studies are ongoing with Phase 1 testing expected to begin in early 2026

Company to hold video webcast today at 10:00 a.m. ET as part of the release of first quarter 2025 results

WATERTOWN, Mass., May 09, 2025 (GLOBE NEWSWIRE) — Kymera Therapeutics, Inc. (NASDAQ: KYMR), a clinical-stage biopharmaceutical company advancing a new class of oral small molecule degrader medicines for immunological diseases, today unveiled a new wholly-owned program within its industry-leading oral immunology pipeline. KT-579, a highly potent, selective, first-in-class development candidate, targets IRF5, an essential signaling node in genetically and clinically validated immune pathways driving inflammation in many diseases with no or suboptimal oral options. The new program serves as a valuable addition to the Company’s current portfolio, positioned to target multiple common immuno-inflammatory diseases with the potential to expand access to oral systemic advanced therapies for broader patient populations. Kymera will share preclinical data and outline upcoming milestones for KT-579 during a video webcast this morning.

“We’re excited to unveil KT-579 as the latest addition to our paradigm-shifting oral immunology portfolio, providing a complementary immunoregulatory mechanism to our existing pipeline. IRF5 is a master regulator of immunity, and we believe blocking its activity with our degrader has the potential to deliver a transformative oral option in multiple chronic, debilitating rheumatic and autoimmune diseases,” said Nello Mainolfi, PhD, Founder, President and CEO, Kymera Therapeutics. “The compelling data we have generated demonstrating activity in human primary cells, patient cell samples, and preclinical animal models showcases, for the first time in industry, that targeting IRF5 can lead to correcting immune dysregulation in a disease specific way while generally sparing normal cells.”

Historically an undrugged transcription factor, IRF5 is a master regulator of innate and adaptive immune response pathways involving pro-inflammatory cytokines (TNFα, IL-6, IL-12, IL-23), B cell activation (autoantibody production), and Type I Interferon (IFN). IRF5 is selectively expressed and activated in specific cell types such as dendritic cells, monocytes, macrophages, and B cells. Its cell- and disease activation-specific profile has the potential to block cell-specific immune dysregulation while sparing normal cell function. IRF5 has been challenging to drug using traditional small molecule inhibitors due to multiple complex activation steps and the high degree of IRF family member homology.

KT-579, a potent, selective and oral degrader has the potential to be the first IRF5-targeted therapy to deliver a completely novel and potentially transformative treatment option, in many cases superior to pathway biologics, in rheumatic and autoimmune diseases such as lupus, Sjögren’s, RA, IBD, among others. Currently in IND-enabling studies, the Company intends to advance KT-579 into Phase 1 clinical testing in early 2026.

In preclinical studies, KT-579 demonstrated an encouraging profile in human primary cells, patient derived cells, and in vivo disease models generally superior to existing standards of care:

• Selectivity and Potency: KT-579 was highly selective for IRF5 over all other proteins in the detectable proteome including other IRF family proteins. KT-579 also demonstrated picomolar to nanomolar potencies across all relevant human cell types evaluated, including B cells, dendritic cells, macrophages, and monocytes. KT-579 demonstrated potent inhibition of proinflammatory cytokines downstream of TLR4, TLR7, TLR8 and TLR9 activation in cellular assays and blocked Type I IFN production and response.
• 
  In Vivo
  
  Profile: KT-579 achieved robust degradation (>90%) across multiple preclinical species in vivo and in all disease-relevant tissues with low oral doses. In preclinical safety studies, KT-579 did not show any adverse effects at concentrations up to 200-fold the projected human efficacious levels, demonstrating a favorable safety profile.
• Efficacy Models: In several preclinical efficacy models of lupus and RA, KT-579 was generally more efficacious than clinically active or marketed small molecule inhibitors and injectable biologics, phenocopying IRF5 knockout studies. In a lupus model, KT-579 demonstrated sustained and near complete reduction of proteinuria and circulating autoantibodies superior to the current standard of care. Additionally, in lupus patient PBMC samples, KT-579 effectively blocked TLR7- and TLR8-induced pro-inflammatory cytokines and IFNβ production and TLR9-induced IgG levels. In a mouse RA model, treatment with KT-579 led to significant reduction in joint swelling.

Event Details

Kymera will host a video webcast today, May 9, 2025, at 10:00 a.m. ET. To join the video call or view the livestreamed webcast please register via this link or visit “News and Events” in the Investors section of the Company’s website at www.kymeratx.com. A replay of the webcast and the presentation will be available following the event.

About Kymera Therapeutics

Kymera is a clinical-stage biotechnology company pioneering the field of targeted protein degradation (TPD) to develop medicines that address critical health problems and have the potential to dramatically improve patients’ lives. Kymera is deploying TPD to address disease targets and pathways inaccessible with conventional therapeutics. Having advanced the first degrader into the clinic for immunological diseases, Kymera is focused on building an industry-leading pipeline of oral small molecule degraders to provide a new generation of convenient, highly effective therapies for patients with these conditions. Founded in 2016, Kymera has been recognized as one of Boston’s top workplaces for the past several years. For more information about our science, pipeline and people, please visit www.kymeratx.com or follow us on X or LinkedIn.

Availability of Other Information About Kymera Therapeutics

For more information, please visit the Kymera website at https://www.kymeratx.com/ or follow Kymera on X (@KymeraTx) and LinkedIn (Kymera Therapeutics). Investors and others should note that Kymera communicates with its investors and the public using the Company website, including, but not limited to, corporate disclosures, investor presentations, FAQs, Securities and Exchange Commission (SEC) filings, and press releases, as well as on X and LinkedIn. The information that Kymera posts on its website or on X or LinkedIn could be deemed to be material information. As a result, the Company encourages investors, the media and others interested to review the information that Kymera posts there on a regular basis. The contents of Kymera’s website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements about our expectations regarding strategy, business plans and objectives on the development of our clinical and preclinical pipeline, including the therapeutic potential, clinical benefits and safety thereof, and the advancement of KT-579 into Phase 1 clinical testing in early 2026. The words “may,” “might,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “expect,” “estimate,” “seek,” “predict,” “future,” “project,” “potential,” “continue,” “target,” “upcoming” and similar words or expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from any forward-looking statements contained in this press release, including, without limitation, risks associated with: uncertainties inherent in the initiation, timing and design of future clinical trials, the availability and timing of data from ongoing and future trials and the results of such trials, whether preclinical results will be indicative of the results of clinical trials, the ability to successfully demonstrate the safety and efficacy of drug candidates, the timing and outcome of planned interactions with regulatory authorities, the availability of funding sufficient for our operating expenses and capital expenditure requirements and other factors. These risks and uncertainties are described in greater detail in the section entitled “Risk Factors” in the most recent Quarterly Report on Form 10-Q and in subsequent filings with the SEC. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We explicitly disclaim any obligation to update any forward-looking statements. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.

Investor and Media Contact: 

Justine Koenigsberg
Vice President, Investor Relations
[email protected]
[email protected]
857-285-5300 

WAYNE, Pa., May 09, 2025 (GLOBE NEWSWIRE) — Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel product candidates for immuno-inflammatory diseases, today announced that Aclaris’ Chief Executive Officer Neal Walker and other members of the Aclaris senior leadership team will participate in a fireside chat during the virtual HC Wainwright “HCW@Home” series.

The virtual fireside chat will take place on Friday May 16, 2025, at 1:00 PM EDT. A live and archived webcast of the event will be accessible on the Events page of https://www.aclaristx.com/. The webcast will be available on the Aclaris website for at least 30 days.

About Aclaris Therapeutics, Inc.

Aclaris Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing a pipeline of novel product candidates to address the needs of patients with immuno-inflammatory diseases who lack satisfactory treatment options. The company has a multi-stage portfolio of product candidates powered by a robust R&D engine. For additional information, please visit www.aclaristx.com and follow Aclaris on X (formerly Twitter) at @AclarisTx and on LinkedIn.

Aclaris Therapeutics Contact:

Will Roberts

Senior Vice President
Corporate Communications and Investor Relations
(484) 329-2125
[email protected]

NEW YORK, May 09, 2025 (GLOBE NEWSWIRE) — Taboola (Nasdaq: TBLA), a global leader in delivering performance at scale for advertisers, today announced that members of its management team will participate in the following investor conferences:

Event: Needham Technology, Internet & Telecom Conference
Date: May 13, 2025
Fireside Chat: 2:15 p.m. ET

Event: Seaport Growth Conference
Date: May 15, 2025
Fireside Chat: 1:00 p.m. ET

Event: B. Riley Institutional Investors Conference
Date: May 21, 2025

Event: TD Cowen Technology, Media & Telecom Conference
Date: May 28, 2025
Fireside Chat: 1:50 p.m. ET

A live webcast and replay of the fireside chats will be available on Taboola’s investor relations website at investors.taboola.com.

About Taboola

Taboola empowers businesses to grow through performance advertising technology that goes beyond search and social and delivers measurable outcomes at scale.

Taboola works with thousands of businesses who advertise directly on Realize, Taboola’s powerful ad platform, reaching approximately 600M daily active users across some of the best publishers in the world. Publishers like NBC News, Yahoo, and OEMs such as Samsung, Xiaomi and others use Taboola’s technology to grow audience and revenue, enabling Realize to offer unique data, specialized algorithms, and unmatched scale.

Investor Contacts:

Jessica Kourakos
Aadam Anwar
[email protected]

Press Contact:

Dave Struzzi
[email protected]

FRIENDSWOOD, Texas, May 09, 2025 (GLOBE NEWSWIRE) — Castle Biosciences, Inc. (Nasdaq: CSTL), a company improving health through innovative tests that guide patient care, today announced new data from the first independent validation of the recently published Collaborative Ocular Oncology Group Study No. 2 (COOG2.1) by Harbour et al.¹ The data, from a real-world cohort of 1,297 patients with uveal melanoma (UM), was presented at the Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting in Salt Lake City. The findings provide further support for adding Preferentially Expressed Antigen in Melanoma (PRAME) gene expression information to the DecisionDx-UM test result to further refine metastatic risk prediction for patients with UM, which is a rare but aggressive eye cancer.

“This real-world validation of the landmark COOG2.1 study both confirms the long-term performance of the DecisionDx-UM test and affirms the clinical value of PRAME as an adjunct biomarker that may further refine a patient’s metastatic risk when combined with the DecisionDx-UM class result,” said Rebecca Critchley-Thorne, Ph.D., vice-president, research and development, of Castle Biosciences. “This enhanced approach to risk stratification is designed to equip clinicians with more precise information to help guide surveillance strategies and enable more personalized treatment planning with the goal of helping to improve outcomes for patients with UM.”

Details regarding Castle’s presentation at ARVO are included below. The abstract may be viewed using the ARVO 2025 mobile meeting planner.

• Presentation Number 981: PRAME status as a risk modifier of 15-gene expression profile class: Evidence from a real-world cohort of 1297 uveal melanoma patients
• Presentation Type: Poster Session
• Session Number: 154
• Session Title: Uveal Melanoma
• Summary: An ongoing collaboration with the National Cancer Institute’s Surveillance, Epidemiology and End Results (NCI SEER) Program enabled linkage of UM patient records with such patients’ DecisionDx-UM and PRAME test results. In a large, real-world, population-based cohort of patients with UM, this study aimed to validate the findings from the prospective COOG2.1 study, which found that adding the reported expression of the PRAME gene to the DecisionDx-UM class result can further refine risk by subdividing Class 1 and Class 2 tumors based on PRAME positive (+) versus negative (-) status. Consistent with the COOG2.1 study, the findings shared at ARVO demonstrate that co-reporting of DecisionDx-UM class and PRAME status provides additional insights into a patient’s likelihood of metastasis to better inform treatment pathway decisions.

About DecisionDx-UM

DecisionDx-UM is Castle Biosciences’ 15-gene expression profile (GEP) test that uses an individual patient’s tumor biology to predict individual risk of metastasis in patients with uveal melanoma (UM). DecisionDx-UM is the standard of care in the management of newly diagnosed UM in the majority of ocular oncology practices in the United States. Since 2009, the American Joint Committee on Cancer (AJCC; v7 and v8) Staging Manual for UM has specifically identified the GEP test as a prognostic factor that is recommended for collection as a part of clinical care. Further, the National Comprehensive Cancer Network (NCCN) guidelines for UM include the DecisionDx-UM test result as a prognostic method for determining risk of metastasis and recommended differential surveillance regimens based on a Class 1A, 1B and 2 result. DecisionDx-UM is currently the only prognostic test for UM that has been validated in prospective, multi-center studies, and it has been shown to be a superior predictor of metastasis compared to other prognostic factors, such as chromosome 3 status, mutational status, AJCC stage and cell type. It is estimated that nearly 8 in 10 patients diagnosed with UM in the United States receive the DecisionDx-UM test as part of their diagnostic workup. Learn more at www.CastleBiosciences.com.

About Castle Biosciences

Castle Biosciences (Nasdaq: CSTL) is a leading diagnostics company improving health through innovative tests that guide patient care. The Company aims to transform disease management by keeping people first: patients, clinicians, employees and investors.

Castle’s current portfolio consists of tests for skin cancers, Barrett’s esophagus and uveal melanoma. Additionally, the Company has active research and development programs for tests in other diseases with high clinical need, including its test in development to help guide systemic therapy selection for patients with moderate-to-severe atopic dermatitis seeking biologic treatment. To learn more, please visit www.CastleBiosciences.com and connect with us on LinkedIn, Facebook, X and Instagram. 

DecisionDx-Melanoma, DecisionDx-CMSeq, i31-SLNB, i31-ROR, DecisionDx-SCC, MyPath Melanoma, TissueCypher, DecisionDx-UM, DecisionDx-PRAME and DecisionDx-UMSeq are trademarks of Castle Biosciences, Inc.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are subject to the “safe harbor” created by those sections. These forward-looking statements include, but are not limited to, statements concerning: the ability of the DecisionDx-UM test to guide more informed, risk-aligned management decisions through the precise risk-stratification of patients with UM; the potential increased risk stratification from including PRAME gene expression information to a patient’s DecisionDx-UM test result; and DecisionDx-UM’s value in identifying UM patients who may benefit from enhanced surveillance and management strategies to improve outcomes. The words “believe,” “can” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements that we make. These forward-looking statements involve risks and uncertainties that could cause our actual results to differ materially from those in the forward-looking statements, including, without limitation: subsequent study or trial results and findings may contradict earlier study or trial results and findings or may not support the results obtained in these studies, including with respect to the discussion of our tests in this press release; actual application of our tests may not provide the aforementioned benefits to patients; and the risks set forth under the heading “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2024 and our Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, each filed with the SEC, and in our other filings with the SEC. The forward-looking statements are applicable only as of the date on which they are made, and we do not assume any obligation to update any forward-looking statements, except as may be required by law.

1. Harbour JW, Correa ZM, Schefler AC, et al. 15-Gene Expression Profile and PRAME as Integrated Prognostic Test for Uveal Melanoma: First Report of Collaborative Ocular Oncology Group Study No. 2 (COOG2.1). J Clin Oncol. 2024;42(28):3319-3329. doi:10.1200/JCO.24.00447

Investor Contact:

Camilla Zuckero
[email protected]

Media Contact:

Allison Marshall
[email protected]

CAMBRIDGE, Mass., May 09, 2025 (GLOBE NEWSWIRE) — Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, today announced that its management team is scheduled to present at the 2025 RBC Capital Markets Global Healthcare Conference on Wednesday, May 21, 2025, at 8:00 am ET.

The live webcast will be accessible on the Investors section of the company’s website (www.agios.com) under the “Events & Presentations” tab. A replay of the webcast will be archived on the company’s website for at least two weeks following the presentation.

About Agios

Agios is the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases. In the U.S., Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company’s deep scientific expertise in classical hematology and leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, myelodysplastic syndromes (MDS)-associated anemia and phenylketonuria (PKU). In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera. For more information, please visit the company’s website at www.agios.com.

Contacts:

Investor Contact

Chris Taylor, VP, Investor Relations and Corporate Communications
Agios Pharmaceuticals
[email protected]

Media Contact

Eamonn Nolan, Senior Director, Corporate Communications
Agios Pharmaceuticals
[email protected]

NEW YORK, May 09, 2025 (GLOBE NEWSWIRE) — Tiziana Life Sciences, Ltd. (Nasdaq: TLSA) (“Tiziana” or the “Company”), a biotechnology company developing breakthrough immunomodulation therapies with its lead development candidate, intranasal foralumab, a fully human anti-CD3 monoclonal antibody, today announced results of a PET scan showing a marked reduction in microglia activation associated with neuroinflammation in a patient suffering from moderate Alzheimer’s disease (AD) who was treated for three months with intranasal foralumab under an expanded access program. The microglial TSPO PET scan revealed a significant decrease in microglia activation, a key indicator of neuroinflammation associated with Alzheimer’s disease progression.

Figure 1.

Figure 1. Decrease in PET signal after foralumab treatment (bottom row) as compared to pre-treatment baseline (top-row). Adapted from Singhal T et al. Clinical Nuclear Medicine 2025 (in press).

The above data was presented by Dr. Howard Weiner at the 2025 AD/PD Conference in Vienna and is featured in the AlzForum. The complete report is contained in Singhal et al, “Dampening of microglial activation with nasal foralumab administration in moderate Alzheimer’s Disease dementia”, Clinical Nuclear Medicine 2025, in press.

Link to article in AlzForum: https://www.alzforum.org/news/conference-coverage/therapies-aim-tame-t-cells-brain

Microglial activation is increasingly recognized as a critical component of neurodegenerative diseases, including Alzheimer’s, secondary progressive multiple sclerosis (MS), amyotrophic lateral sclerosis (ALS), and Parkinson’s disease. In Alzheimer’s, this activation is thought to contribute to disease pathogenesis alongside increased beta-amyloid and tau protein levels. Cognitive decline in AD may have multiple etiologies apart from amyloid and tau accumulation, highlighting the need for treatments beyond amyloid-targeting therapies.

Nasal foralumab, a fully human anti-CD3 monoclonal antibody, has shown efficacy in dampening microglial activation through the induction of Tregs, which travel to the brain and reduce neuroinflammation. This therapeutic effect has been confirmed in animal models and observed in subjects with secondary progressive MS. Nasal foralumab also holds promise as a potential adjunctive therapy to recently approved products that are focused on amyloid reduction.

Dr. Howard Weiner, Co-director of the Ann Romney Center for Neurologic Diseases at Brigham and Women’s Hospital, a founding member of Mass General Brigham healthcare system, commented on the findings: “There has been a substantial drop in TSPO signal over the three-month period, suggesting that the foralumab treatment might be reducing microglial activation and this is highly encouraging. This suggests that Foralumab may play a crucial role in mitigating neuroinflammation in Alzheimer’s patients. We are pleased to say we have also not seen any side effects so far in the study and the patient continues to be treated with foralumab.”

Tarun Singhal, M.B.B.S., M.D., Director of the PET Imaging Program in Neurologic Diseases, associate neurologist and nuclear medicine physician at Brigham and Women’s Hospital, added: “We have seen a measurable, widespread reduction in microglial PET signal, which is quite remarkable. This reduction is consistent with what has been seen in patients with multiple sclerosis treated with intranasal foralumab. Further studies incorporating additional cases and PET analytical approaches in moderate Alzheimer’s disease, which is an area of huge unmet need, are urgently needed.”

Dr. Ivor Elrifi, CEO of Tiziana Life Sciences, emphasized the significance of these findings: “This PET scan data represents a critical milestone for Tiziana Life Sciences and underscores our commitment to advancing innovative treatments for Alzheimer’s disease. With no approved therapies currently available for moderate Alzheimer’s, we believe Foralumab holds promise not only as a standalone treatment but also potentially in combination therapies for mild Alzheimer’s disease.”

Gabriele Cerrone, Executive Chairman and Founder of Tiziana Life Sciences, commented: “This PET scan result showing a dampening of microglial activation by nasal foralumab in a patient with moderate Alzheimer’s disease, demonstrates the potential for foralumab to fight many neurologic diseases by treating neuroinflammation. This reduction of microglia activation is consistent with the results we have seen in our ongoing study of patients with Secondary Progressive Multiple Sclerosis, who have shown a stabilization or improvement of symptoms of disease through prolonged foralumab treatment.”

Alzheimer’s disease affects over 50 million people globally, with numbers expected to triple by 2050. The urgent need for effective treatments, especially in the moderate stages, highlights the importance of innovative research and development efforts such as those undertaken by Tiziana Life Sciences.

About Foralumab

Foralumab, a fully human anti-CD3 monoclonal antibody, is a biological drug candidate that has been shown to stimulate T regulatory cells when dosed intranasally. At present, 10 patients with Non-Active Secondary Progressive Multiple Sclerosis (na-SPMS) have been dosed in an open-label intermediate sized Expanded Access (EA) Program (NCT06802328) with either an improvement or stability of disease seen within 6 months in all patients. In addition, intranasal foralumab is currently being studied in a Phase 2a, randomized, double-blind, placebo-controlled, multicenter, dose-ranging trial in patients with non-active secondary progressive multiple sclerosis (NCT06292923).

Foralumab is the only fully human anti-CD3 monoclonal antibody (mAb) currently in clinical development. The non-active SPMS intranasal foralumab Phase 2 trial (NCT06292923) began screening patients in November of 2023. Immunomodulation by intranasal foralumab represents a novel avenue for the treatment of neuroinflammatory and neurodegenerative human diseases.^[1],[2]

About Tiziana Life Sciences

Tiziana Life Sciences is a clinical-stage biopharmaceutical company developing breakthrough therapies using transformational drug delivery technologies to enable alternative routes of immunotherapy. Tiziana’s innovative nasal approach has the potential to provide an improvement in efficacy as well as safety and tolerability compared to intravenous (IV) delivery. Tiziana’s lead candidate, intranasal foralumab, which is the only fully human anti-CD3 mAb currently in clinical development, has demonstrated a favorable safety profile and clinical response in patients in studies to date. Tiziana’s technology for alternative routes of immunotherapy has been patented with several applications pending and is expected to allow for broad pipeline applications.

For more information about Tiziana Life Sciences and its innovative pipeline of therapies, please visit www.tizianalifesciences.com.

Forward-Looking Statements

Certain statements made in this announcement are forward-looking statements. These forward-looking statements are not historical facts but rather are based on the Company’s current expectations, estimates, and projections about its industry, its beliefs, and assumptions. Words such as ‘anticipates,’ ‘expects,’ ‘intends,’ ‘plans,’ ‘believes,’ ‘seeks,’ ‘estimates,’ and similar expressions are intended to identify forward-looking statements. These statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties, and other factors, some of which are beyond the Company’s control, are difficult to predict, and could cause actual results to differ materially from those expressed or forecasted in the forward-looking statements. The Company cautions security holders and prospective security holders not to place undue reliance on these forward-looking statements, which reflect the view of the Company only as of the date of this announcement. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties related to market conditions and other factors described more fully in the section entitled ‘Risk Factors’ in Tiziana’s Annual Report on Form 20-F for the year ended December 31, 2024, and other periodic reports filed with the Securities and Exchange Commission.The forward-looking statements made in this announcement relate only to events as of the date on which the statements are made. The Company will not undertake any obligation to release publicly any revisions or updates to these forward-looking statements to reflect events, circumstances, or unanticipated events occurring after the date of this announcement except as required by law or by any appropriate regulatory authority.

For further inquiries:

Tiziana Life Sciences Ltd

Paul Spencer, Business Development, and Investor Relations
+44 (0) 207 495 2379
email: [email protected]

[1] https://www.pnas.org/doi/10.1073/pnas.2220272120

[2] https://www.pnas.org/doi/10.1073/pnas.2309221120

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/248d2d9e-1743-4aa0-b112-11086779a2fa