Market Newsdesk

MONACO, Jan. 12, 2025 (GLOBE NEWSWIRE) — Scorpio Tankers Inc. (NYSE: STNG) (“Scorpio Tankers” or the “Company”) through its Norwegian investment banks, will arrange a series of fixed income investor calls commencing on January 13, 2025. A five-year USD denominated senior unsecured bond issue may follow, subject to inter alia market conditions.

The net proceeds from the contemplated bond issue are intended to be used for the refinancing of the Company’s existing 7.0% senior unsecured notes due June 30, 2025 which have an outstanding principal balance of USD 70.6 million, and for general corporate purposes.

The senior unsecured bonds, if issued, will be offered in the United States or its territories only to persons reasonably believed to be qualified institutional buyers in reliance on Rule 144A under the Securities Act of 1933, as amended (the “U.S. Securities Act”). The bonds, if issued, will not be registered under the Securities Act or any state securities laws and may not be offered or sold in the United States absent registration or an applicable exemption from registration requirements. This press release shall not constitute an offer to sell or the solicitation of an offer to buy any securities of Scorpio Tankers Inc., nor shall it constitute an offer, solicitation or sale in any jurisdiction in which such offer, solicitation or sale is unlawful, and is being issued in the United States pursuant to and in accordance with Rule 135c under the Securities Act.

For further information, please contact:
James Doyle – Head of Corporate Development & Investor Relations
Tel: +1 203-900-0559
Email: [email protected]

About Scorpio Tankers Inc.

Scorpio Tankers Inc. is a provider of marine transportation of petroleum products worldwide. Scorpio Tankers Inc. currently owns or lease finances 99 product tankers (39 LR2 tankers, 46 MR tankers and 14 Handymax tankers) with an average age of 8.8 years. Additional information about the Company is available at the Company’s website www.scorpiotankers.com, which is not a part of this press release.

Forward-Looking Statements

Matters discussed in this press release may constitute forward‐looking statements. The Private Securities Litigation Reform Act of 1995 provides safe harbor protections for forward‐looking statements in order to encourage companies to provide prospective information about their business. Forward‐looking statements include statements concerning plans, objectives, goals, strategies, future events or performance, and underlying assumptions and other statements, which are other than statements of historical facts. The Company desires to take advantage of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and is including this cautionary statement in connection with this safe harbor legislation. The words “believe,” “expect,” “anticipate,” “estimate,” “intend,” “plan,” “target,” “project,” “likely,” “may,” “will,” “would,” “could” and similar expressions identify forward‐looking statements.

The forward‐looking statements in this press release are based upon various assumptions, many of which are based, in turn, upon further assumptions, including without limitation, management’s examination of historical operating trends, data contained in the Company’s records and other data available from third parties. Although management believes that these assumptions were reasonable when made, because these assumptions are inherently subject to significant uncertainties and contingencies which are difficult or impossible to predict and are beyond the Company’s control, there can be no assurance that the Company will achieve or accomplish these expectations, beliefs or projections. The Company undertakes no obligation, and specifically declines any obligation, except as required by law, to publicly update or revise any forward‐looking statements, whether as a result of new information, future events or otherwise.

In addition to these important factors, other important factors that, in the Company’s view, could cause actual results to differ materially from those discussed in the forward‐looking statements include unforeseen liabilities, future capital expenditures, revenues, expenses, earnings, synergies, economic performance, indebtedness, financial condition, losses, future prospects, business and management strategies in response to epidemic and other public health concerns including any effect on demand for petroleum products and the transportation thereof, expansion and growth of the Company’s operations, risks relating to the integration of assets or operations of entities that it has or may in the future acquire and the possibility that the anticipated synergies and other benefits of such acquisitions may not be realized within expected timeframes or at all, the failure of counterparties to fully perform their contracts with the Company, the strength of world economies and currencies, general market conditions, including fluctuations in charter rates and vessel values, changes in demand for tanker vessel capacity, changes in the Company’s operating expenses, including bunker prices, drydocking and insurance costs, the market for the Company’s vessels, availability of financing and refinancing, charter counterparty performance, ability to obtain financing and comply with covenants in such financing arrangements, changes in governmental rules and regulations or actions taken by regulatory authorities, potential liability from pending or future litigation, general domestic and international political conditions, including the impact of the conflict in Ukraine and the developments in the Middle East, including the armed conflict in Israel and Gaza, potential disruption of shipping routes due to accidents or political events, vessels breakdowns and instances of off‐hires, and other factors. Please see the Company’s filings with the U.S. Securities and Exchange Commission for a more complete discussion of certain of these and other risks and uncertainties.

• Radio-DARPin MP0712 against DLL3, in co-development with Orano Med, to enter first-in-human study in 2025
• Mesothelin named as second target in Radio-DARPin pipeline, program to be co-developed with Orano Med
• Orano Med partnership on Radio-DARPins now expanded to 10 programs
• MP0533 clinical data show improved response rate and depth in cohort 8 with steeper step-up and more frequent dosing; additional dose densification planned in cohort 9, updates expected in 2025
• CD3 Switch-DARPin research proof-of-concept of conditional T cell activation and CD2 co-stimulation shown in solid tumors, further data in Q2 2025
  

ZURICH-SCHLIEREN, Switzerland and CONCORD, Mass., Jan. 12, 2025 (GLOBE NEWSWIRE) — Ad hoc announcement pursuant to Art. 53 LRMolecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics (“Molecular Partners” or the “Company”), today provided an update on its programs, development plans and guidance on key milestones expected in 2025, to be presented at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco, California.

“We are excited to enter 2025 with upcoming key value inflection points, on the Radio-DARPin side as well as Switch-DARPin and clinical T-cell engagers, to build on our achievements through 2024. Our recently expanded strategic partnership with Orano Med ensures us access to ²¹²Pb, to arm our Radio-DARPins for up to 10 products. MP0712, our most advanced Radio-DARPin targeting DLL3, is moving into clinical development in 2025. Further, we have selected Mesothelin as the second target in the Orano Med partnership, with unique DARPin binders that only bind to juxtamembrane Meso while not being inhibited by the shed target,” said Patrick Amstutz, Ph.D., CEO of Molecular Partners.

Molecular Partners has further strengthened and expanded its agreement with Orano Med for co-development of up to ten²¹²Pb-based Radio-DARPins. Molecular Partners holds commercialization rights to MP0712, which is the most advanced program, as well as the second nominated Radio-DARPin candidate, which targets the membrane-proximal portion of cell surface glycoprotein Mesothelin (MSLN). Orano Med will ensure the production of the ²¹²Pb-based Radio-DARPins for clinical trials and commercialization. Further details on this second candidate are scheduled to be unveiled at the Annual Meeting of the American Association of Cancer Research (AACR) in Q2 2025.

Patrick Amstutz continued, “We are equally excited that our work on the MP0533 candidate in R/R AML is starting to yield encouraging results. As we work to implement our previously discussed protocol amendments, we are already starting to see patients benefit from treatment in our ongoing cohort 8, where we introduced an additional dosing timepoint early on. These preliminary data provide us with reassurance that our strategy to further densify early dosing has merit and could enable more patients to benefit longer from MP0533.”

Cash and Cash Equivalents:

As of Dec 31 2024, Molecular Partners reports cash and cash equivalents of CHF 149 M (unaudited) and will provide full YE financial results on March 6, 2025.

Key current program status updates include:

MP0712 & Radio-DARPin pipeline

The Investigational New Drug (IND) application for MP0712, a ²¹²Pb Radio-DARPin candidate against the tumor-associated protein delta-like ligand 3 (DLL3), is in preparation. Dialogue with the U.S. Food and Drug Administration (FDA) is ongoing and Molecular Partners and Orano Med anticipate submitting the IND application for MP0712 in H1 2025, with the first-in-human study to start following regulatory clearance.

The IND submission is being built, in part, on strong MP0712 preclinical results, including new in vivo data presented at the European Association of Nuclear Medicine Congress in October 2024 and the European Targeted Radiopharmaceuticals Summit in December 2024. MP0712 demonstrated high affinity and specificity for DLL3, which is a highly relevant target for radiopharmaceutical therapy. DLL3 has been shown to have homogeneous expression in tumors of patients with small cell lung cancer, and expression in healthy tissues is low.

The second Radio-DARPin program co-developed with Orano Med targets MSLN, which is overexpressed across several cancers with high unmet need, such as ovarian cancer, and largely absent from healthy tissues. The development of therapeutics against MSLN has been hampered by high shedding of MSLN. Leveraging the unique DARPin properties, Molecular Partners has developed Radio-DARPins able to selectively bind to the membrane-proximal portion of MSLN present on cells and are therefore not impacted by shed MSLN.

In addition to the above updates, Molecular Partners continues to progress its Radio-DARPin Therapy (RDT) portfolio of projects in partnership with Novartis and is evaluating additional targets for RDT programs.

MP0533 (multispecific T cell engager)

MP0533 is currently being evaluated in a Phase 1/2a clinical trial for relapsed/refractory acute myeloid leukemia (AML) and myelodysplastic syndrome/AML (ClinicalTrials.gov: NCT05673057). Dose escalation in cohort 1–7 showed an acceptable safety profile and initial activity yet unsustained responses (four responders reported and encouraging blast reductions across additional patients), as presented in December 2024 at the American Society of Hematology meeting.

In the currently ongoing cohort 8, in which an additional early dosing timepoint was introduced to allow steeper and more frequent dosing to reach the MP0533 target dose faster, increased rates and depth of responses are being observed, with three out of the first eight evaluable patients demonstrating responses to-date (data cutoff 16 December 2024). Molecular Partners has submitted an amendment to the study protocol to improve the exposure profile of MP0533 and to further deepen and expand responses being observed in cohort 8. Data on the amended dosing scheme are expected in 2025.

MP0533 is a novel tetraspecific T cell engaging DARPin which simultaneously targets the three tumor-associated antigens (TAAs) CD33, CD123, and CD70, as well as CD3 on T cells. The mechanism of action of MP0533 is designed to preferentially kill AML cells that express at least two of the three TAAs while sparing healthy cells, which express only one or none of these targets. The immune activation against the malignant cells is achieved through CD3-mediated T cell engagement.

Switch-DARPin Platform (next-generation immune cell engagers)

Preclinical proof-of-concept in a solid tumor model for the novel T cell engager Switch-DARPin was presented at the Annual Meeting of the Society for Immunotherapy of Cancer (SITC) in November. The presented data provide further validation of Switch-DARPins showing that conditional Tcell activation with potent co-stimulation in solid tumors, but not in healthy tissues, is feasible.

Specifically, the CD3 Switch-DARPin molecule was shown to effectively induce potent tumor regression in vivo. Reduced cytokine release was observed in healthy tissues compared to tumor tissue. Cytokine release syndrome (CRS) is a significant toxicity event that has been observed with many T cell engagers in the clinic. As such, masking CD3 may prevent T cell activation in the absence of tumor antigens and allow for “silent” T cell engagers outside of tumors, thereby reducing the risk of CRS and providing a better safety profile to T cell engagers. In addition, co-engagement of CD2 led to sustained T cell activation and cytotoxic capacity, thereby enabling the development of potent T cell engagers with improved therapeutic window. Molecular Partners plans to present further in vivo data on the CD3 Switch-DARPin at the AACR Annual Meeting in Q2 2025.

MP0317 (localized agonist)

Molecular Partners presented comprehensive biomarker analyses from the completed Phase 1 clinical trial of the CD40 agonist MP0317 in solid tumors at SITC in November 2024. MP0317 is designed to activate immune cells specifically within the tumor microenvironment by anchoring to fibroblast activation protein (FAP) which is expressed in high amounts in the stroma of various solid tumors. This tumor-localized approach has the potential to deliver greater efficacy with fewer side effects compared to systemic CD40-targeting therapies.

Molecular Partners is in discussion with leading academic centers regarding potential investigator-initiated combination trials of MP0317 in 2025, in combination with immune checkpoint inhibitors and additional standard of care.

J.P. Morgan Presentation Details:

Presenter: Molecular Partners CEO Patrick Amstutz
Time: January 15, 2025, at 9:00 AM PST (6:00 PM CET)
Location: Westin St. Francis, Elizabethan A Ballroom, San Francisco, CA

A webcast will be accessible on the Molecular Partners website, under the Events tab.

About Molecular Partners AG 
Molecular Partners AG (SIX: MOLN, NASDAQ: MOLN) is a clinical-stage biotech company pioneering the design and development of DARPin therapeutics for medical challenges other drug modalities cannot readily address. The Company has programs in various stages of pre-clinical and clinical development, with oncology as its main focus. Molecular Partners leverages the advantages of DARPins to provide unique solutions to patients through its proprietary programs as well as through partnerships with leading pharmaceutical companies. Molecular Partners was founded in 2004 and has offices in both Zurich, Switzerland and Concord, MA, USA. For more information, visit www.molecularpartners.com and find us on LinkedIn and Twitter / X @MolecularPrtnrs

For further details, please contact:

Seth Lewis, SVP Investor Relations & Strategy
Concord, Massachusetts, U.S.
[email protected]
Tel: +1 781 420 2361

Laura Jeanbart, PhD, Head of Portfolio Management & Communications
Zurich-Schlieren, Switzerland
[email protected]
Tel: +41 44 575 19 35

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, as amended, including without limitation: implied and express statements regarding the clinical development of Molecular Partners’ current or future product candidates; expectations regarding timing for reporting data from ongoing clinical trials or the initiation of future clinical trials; the potential therapeutic and clinical benefits of Molecular Partners’ product candidates and its RDT and Switch-DARPin platforms; the selection and development of future programs; Molecular Partners’ collaboration with Orano Med including the benefits and results that may be achieved through the collaboration; and Molecular Partners’ expected business and financial outlook, including anticipated expenses and cash utilization for 2024 and its expectation of its current cash runway and the expected use of proceeds from the underwritten offering. These statements may be identified by words such as “aim”, “expect”, “guidance”, “intend”, “outlook”, “plan”, “potential”, “will” and similar expressions, and are based on Molecular Partners’ current beliefs and expectations. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Some of the key factors that could cause actual results to differ from Molecular Partners’ expectations include its plans to develop and potentially commercialize its product candidates; Molecular Partners’ reliance on third party partners and collaborators over which it may not always have full control; Molecular Partners’ ongoing and planned clinical trials and preclinical studies for its product candidates, including the timing of such trials and studies; the risk that the results of preclinical studies and clinical trials may not be predictive of future results in connection with future clinical trials; the timing of and Molecular Partners’ ability to obtain and maintain regulatory approvals for its product candidates; the extent of clinical trials potentially required for Molecular Partners’ product candidates; the clinical utility and ability to achieve market acceptance of Molecular Partners’ product candidates; the potential that Molecular Partners’ product candidates may exhibit serious adverse, undesirable or unacceptable side effects; the impact of any health pandemic, macroeconomic factors and other global events on Molecular Partners’ preclinical studies, clinical trials or operations, or the operations of third parties on which it relies; Molecular Partners’ plans and development of any new indications for its product candidates; Molecular Partners’ commercialization, marketing and manufacturing capabilities and strategy; Molecular Partners’ intellectual property position; Molecular Partners’ ability to identify and in-license additional product candidates; unanticipated factors in addition to the foregoing that may impact Molecular Partners’ financial and business projections and guidance; and other risks and uncertainties set forth in Molecular Partners’ Annual Report on Form 20-F for the year ended December 31, 2023 and other filings Molecular Partners makes with the SEC from time to time. These documents are available on the Investors page of Molecular Partners’ website at www.molecularpartners.com. In addition, this press release contains information relating to interim data as of the relevant data cutoff date, results of which may differ from topline results that may be obtained in the future. Any forward-looking statements speak only as of the date of this press release and are based on information available to Molecular Partners as of the date of this release, and Molecular Partners 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.

• New agreement enables both companies to fuel a broad and innovative pipeline of ²¹²Pb-Radio-DARPin candidates, bringing the total number of programs up to ten
• Expanded partnership highlights the parties’ emerging leadership in targeted alpha therapies (TAT), leveraging Orano Med’s expertise in the development of ²¹²Pb-based TAT and vast proprietary supply of ²¹²Pb and Molecular Partners’ unique Radio-DARPins as an ideal vector for radiopharmaceuticals
• Most advanced ²¹²Pb-Radio-DARPin, DLL3-targeted MP0712, starts clinical trials in 2025

ZURICH-SCHLIEREN, Switzerland and CONCORD, Mass., and PARIS, Jan. 12, 2025 (GLOBE NEWSWIRE) — Ad hoc announcement pursuant to Art. 53 LR – Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, and Orano Med, a clinical-stage radiopharmaceutical company and a pioneer in the development of targeted alpha-particle therapies (TAT) with ²¹²Pb (lead-212), today announced the expansion of their strategic collaboration.

The terms of the new agreement include the development of an additional six targeted alpha therapeutics candidates, now representing a total of ten potential programs between the two companies. Molecular Partners will lead development of the additional six programs, subject to a royalty arrangement, and include an option for Orano Med to move two of the six programs into a 50/50 co-development where Orano Med will hold commercialization rights.

In January 2024, Molecular Partners and Orano Med entered into an initial agreement to co-develop four programs. equally sharing costs for preclinical and clinical development and profit from commercialized products. Molecular Partners holds commercialization rights for the first program, MP0712, a DLL3-targeted radio-DARPin, which is expected to move into first-in-human studies in 2025, pending regulatory clearance. Molecular Partners will also hold commercialization rights to the second program, targeting mesothelin, and Orano Med to programs three and four.

The partnership combines Molecular Partners’ unique and innovative Radio-DARPin Platform with Orano Med’s ²¹²Pb supply, research and clinical development capabilities. Both groups have been working closely together over the past years, reducing drug candidate cycle times and enabling the generation of more drug candidates to novel targets, thereby pushing the boundaries of what is presently achievable by other technologies.

“We are excited to expand this relationship with Orano Med, representing the bold ambition and high synergy of both groups to build the largest radiotherapy pipeline in our space today. Both groups, having worked together over the past year, realize the unique potential of the other, and we have confidence in our abilities to provide novel and innovative targets for the delivery of radioactive isotopes, pushing the boundaries of what is presently targetable by other technologies,” said Patrick Amstutz, Ph.D., CEO of Molecular Partners.

“The expansion of our collaboration with Molecular Partners underscores the strength and efficiency of our combined approach. Together, we have established a platform capable of significantly reducing development timelines for lead-212-based Radio-DARPin drug candidates. This partnership exemplifies how strategic synergies can drive innovation and accelerate the delivery of next-generation targeted alpha therapies to patients, and further diversifies vectorization technology in Orano Med’s pipeline,” said Arnaud Lesegretain, CEO of Orano Med.

Financial terms of the agreement are not disclosed. Molecular Partners expects no immediate impact on its financial forecast for the fiscal year 2025 from the expansion of the co-development agreement and maintains its funding guidance into 2027.

About DARPin Therapeutics

DARPin (Designed Ankyrin Repeat Protein) therapeutics are a new class of custom-built protein drugs based on natural binding proteins that open new dimensions of multi-functionality and multi-target specificity in drug design. The flexible architecture, intrinsic potential for high affinity and specificity, small size and high stability of DARPins offer benefits to drug design over other currently available protein-based therapeutics. DARPin candidates can be radically simple, with a single DARPin unit acting as the delivery vector to a specific target; or multispecific, with the possibility of engaging more than five targets, and combining multiple and conditional functionalities in a unique DARPin drug candidate. The DARPin platform is designed to be a rapid and cost-effective drug discovery engine, producing drug candidates with optimized properties and high production yields. DARPin therapeutics have been clinically validated across several therapeutic areas and developed through to the registrational stage.

About

²¹²

Pb-based
Radio-DARPins

Molecular Partners and Orano Med’s Radio-DARPin platform is being developed to provide a unique and innovative delivery system for radioactive payloads, with exquisite targeting capabilities combined with the optimally balanced safety and tumor killing of ²¹²Pb. DARPins are ideal vectors for efficient delivery of therapeutic radionuclides to solid tumors, while overcoming some historic limitations of radioligand therapy approaches, thanks to their small size as well as high specificity and affinity. Molecular Partners and Orano Med are developing ²¹²Pb-Radio-DARPin candidates against up to ten targets, including the tumor-associated protein Delta-like ligand 3 (DLL3) and mesothelin (MSLN).

About Molecular Partners AG 

Molecular Partners AG is a clinical-stage biotech company pioneering the design and development of DARPin therapeutics for medical challenges other drug modalities cannot readily address. The Company has programs in various stages of pre-clinical and clinical development, with oncology as its main focus. Molecular Partners leverages the advantages of DARPins to provide unique solutions to patients through its proprietary programs as well as through partnerships with leading pharmaceutical companies. Molecular Partners was founded in 2004 and has offices in both Zurich, Switzerland and Concord, MA, USA. For more information, visit www.molecularpartners.com and find us on LinkedIn and Twitter/X @MolecularPrtnrs.

About Orano Med SAS

Orano Med, a subsidiary of the Orano Group, is a clinical-stage biotechnology company which develops a new generation of targeted therapies against cancer using the unique properties of lead-212 (²¹²Pb), an alpha-emitting radioisotope and one of the more potent therapeutic payloads against cancer cells known as Targeted Alpha-Emitter Therapy (TAT). AlphaMedix, its most advanced asset in development for GEP-NETs tumors received Breakthrough Designation from the FDA in 2024. The company develops several treatments using ²¹²Pb combined with various targeting agents. Orano Med has ²¹²Pb manufacturing facilities, laboratories, and R&D centers in France and in the US. It is expanding its GMP-manufacturing capacities for ²¹²Pb radiolabeled pharmaceuticals in North America and Europe and building a unique integrated industrial platform to serve the needs of patients globally. For more information, please visit: www.oranomed.com.

As a recognized international operator in the field of nuclear materials, Orano Group delivers solutions to address present and future global energy and health challenges. Its expertise and mastery of cutting-edge technologies enable Orano to offer its customers high value-added products and services throughout the entire fuel cycle. Every day, the Orano group’s 18,000 employees draw on their skills, unwavering dedication to safety and constant quest for innovation, with the commitment to develop know-how in the transformation and control of nuclear materials, for the climate and for a healthy and resource-efficient world, now and tomorrow.

About
Targeted Alpha Therapy

Targeted alpha therapy (TAT) relies on a simple concept: combining the ability of biological molecules to target cancer cells with the short-range cell-killing capabilities of alpha-emitting radioisotopes. Alpha decay consists of the emission of a helium nucleus (alpha particle) together with very high linear energy transfer and a range emission of only few cell layers, resulting in irreparable double strand DNA breaks in cells adjacent only to area of alpha emission. This approach results in an increased cytotoxic potential toward cancer cells while limiting toxicity to nearby healthy cells. As a result, alpha emitters are considered as the most powerful payloads to be found for targeted therapies.

For further details, please contact:

Molecular Partners
Seth Lewis, SVP Investor Relations & Strategy
Concord, Massachusetts, U.S.
[email protected]
Tel: +1 781 420 2361

Laura Jeanbart, PhD, Head of Portfolio Management & Communications
Zurich-Schlieren, Switzerland
[email protected]
Tel: +41 44 575 19 35

Orano Med
Sophie Letournel
Strategy, governance, and communication director
[email protected]
Tel: +33 6 38 44 34 11

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, as amended, including, without limitation: implied and express statements regarding the clinical development of Molecular Partners’ current or future product candidates; expectations regarding timing for reporting data from ongoing clinical trials or the initiation of future clinical trials; the potential therapeutic and clinical benefits of Molecular Partners’ product candidates and its RDT and Switch-DARPin platforms; the selection and development of future programs; Molecular Partners’ collaboration with Orano Med including the benefits and results that may be achieved through the collaboration; and Molecular Partners’ expected business and financial outlook, including anticipated expenses and cash utilization for 2024 and its expectation of its current cash runway. These statements may be identified by words such as “aim”, “expect”, “guidance”, “intend”, “outlook”, “plan”, “potential”, “will” and similar expressions, and are based on Molecular Partners’ current beliefs and expectations. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Some of the key factors that could cause actual results to differ from Molecular Partners’ expectations include its plans to develop and potentially commercialize its product candidates; Molecular Partners’ reliance on third party partners and collaborators over which it may not always have full control; Molecular Partners’ ongoing and planned clinical trials and preclinical studies for its product candidates, including the timing of such trials and studies; the risk that the results of preclinical studies and clinical trials may not be predictive of future results in connection with future clinical trials; the timing of and Molecular Partners’ ability to obtain and maintain regulatory approvals for its product candidates; the extent of clinical trials potentially required for Molecular Partners’ product candidates; the clinical utility and ability to achieve market acceptance of Molecular Partners’ product candidates; the potential that Molecular Partners’ product candidates may exhibit serious adverse, undesirable or unacceptable side effects; the impact of any health pandemic, macroeconomic factors and other global events on Molecular Partners’ preclinical studies, clinical trials or operations, or the operations of third parties on which it relies; Molecular Partners’ plans and development of any new indications for its product candidates; Molecular Partners’ commercialization, marketing and manufacturing capabilities and strategy; Molecular Partners’ intellectual property position; Molecular Partners’ ability to identify and in-license additional product candidates; unanticipated factors in addition to the foregoing that may impact Molecular Partners’ financial and business projections and guidance; and other risks and uncertainties that are described in the Risk Factors section of Molecular Partners’ Annual Report on Form 20-F for the fiscal year ended December 31, 2023, filed with Securities and Exchange Commission (SEC) on March 14, 2024 and other filings Molecular Partners makes with the SEC. These documents are available on the Investors page of Molecular Partners’ website at www.molecularpartners.com. In addition, this press release contains information relating to interim data as of the relevant data cutoff date, results of which may differ from topline results that may be obtained in the future. Any forward-looking statements speak only as of the date of this press release and are based on information available to Molecular Partners as of the date of this release, and Molecular Partners 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.

New biomarkers on track for commercial launch in January 2025

Preliminary financial results indicate record full-year revenue and AVISE CTD trailing twelve-month ASP, with improvements to adjusted EBITDA and cash burn

CARLSBAD, Calif., Jan. 12, 2025 (GLOBE NEWSWIRE) — Exagen Inc. (Nasdaq: XGN), a leading provider of autoimmune testing, today announced it has received conditional approval from the New York State Department of Health for its new systemic lupus erythematosus (SLE) and rheumatoid arthritis (RA) biomarker assays, with a planned commercial launch in January 2025.  The company also announced preliminary unaudited select financial results for the fourth quarter and full year ended December 31, 2024, in line with prior financial guidance.

New AVISE CTD Biomarkers

The company’s new SLE and RA biomarkers will be incorporated into the AVISE CTD platform. Collectively, the company expects these new biomarkers will further improve the clinical utility of AVISE CTD, and provide clinicians with the information they need to definitively diagnose patients and shorten their autoimmune diagnostic journeys.

The new biomarkers are as follows:

• T-Cell Lupus profile comprises new biomarkers; TC4d, TIgG, and TIgM. These markers provide superior sensitivity for SLE compared to conventional SLE biomarkers, further enhancing our industry-leading AVISE Lupus profile for a more comprehensive diagnosis, particularly in clinically ambiguous cases.

• RA profile includes additional anti-RA33 biomarkers; IgA, IgG, and IgM. These markers provide clinicians with more data to confidently identify patients with RA and substantiate a seronegative RA diagnosis.

Preliminary Unaudited Select Financial Results

The preliminary unaudited select financial results reported today represent:

• Record full-year 2024 revenue and AVISE CTD ASP
• AVISE CTD trailing twelve-month ASP improvement of $72 to $76 compared to Q4 2023
• Full-year 2024 adjusted EBITDA improvement of to 37% to 43% compared to full-year 2023
• Net neutral cash usage in the fourth quarter of 2024

“2024 was a remarkable year for Exagen by many accounts. We reached a significant milestone by testing our 1,000,000^th patient with AVISE CTD, an impressive achievement for any proprietary test, but especially one in a field that hasn’t seen robust biomarker innovation for many decades,” said John Aballi, CEO. “We’ve also made great strides in optimizing our company’s operations and continued our progress toward profitability – a goal which is now firmly within reach, all while delivering record reimbursement per test and record overall revenue performance.”

Mr. Aballi continued, “To start 2025 with New York State’s approval of our new assays is another significant milestone, and we now sit in the midst of several growth catalysts with strong momentum to start the year. I’m very grateful to the dedicated team we have, and couldn’t be more excited about what lies ahead.”

Cautionary Note Regarding Select Preliminary Unaudited Financial Results

The company is providing the above preliminary unaudited select financial information and results of operations as of and for the three months and year ended December 31, 2024, based on currently available information. The company’s financial closing procedures with respect to the estimated financial data provided above are not yet complete. These procedures often result in changes to accounts. Our independent registered public accounting firm has not audited, reviewed, compiled or performed any procedures with respect to the preliminary unaudited select financial information and, accordingly, our independent registered public accounting firm does not express an opinion or any other form of assurance with respect thereto. As a result, the company’s final results may vary from the preliminary results presented above. Management undertakes no obligation to update or supplement the information provided above until it releases its audited financial statements prepared in accordance with U.S. Generally Accepted Accounting Principles (GAAP) for the year ended December 31, 2024.

Use of Unaudited Non-GAAP Financial Measures

In addition to the financial results prepared in accordance with GAAP, this press release contains the metric adjusted EBITDA, which is not calculated in accordance with GAAP and is a non-GAAP financial measure. Adjusted EBITDA excludes from net loss interest income (expense), income tax expense (benefit), depreciation and amortization expense, stock-based compensation expense and other expenses or income that management believes are not representative of the company’s operations. Such items could have a significant impact on the calculation of GAAP net loss.

The table below presents the reconciliation of preliminary net loss to adjusted EBITDA, which is a non-GAAP financial measure. See “Use of Non-GAAP Financial Measures (UNAUDITED)” above for further information regarding the company’s use of non-GAAP financial measures.

Reconciliation of Non-GAAP Financial Measures (UNAUDITED)

Management believes that this non-GAAP financial measure, taken in conjunction with GAAP financial measures, provides useful information for both management and investors by excluding certain non-cash and other expenses that are not indicative of the company’s core operating results. Management uses non-GAAP measures to compare the company’s performance relative to forecasts and strategic plans and to benchmark the company’s performance externally against competitors. However, this non-GAAP financial measure may be different from non-GAAP financial measures used by other companies, even when the same or similarly titled terms are used to identify such measures, limiting their usefulness for comparative purposes. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of the company’s operating results as reported under U.S. GAAP

This non-GAAP financial measure is not meant to be considered in isolation or used as a substitute for net loss reported in accordance with GAAP, should be considered in conjunction with financial information presented in accordance with GAAP, has no standardized meaning prescribed by GAAP, is unaudited, and is not prepared under any comprehensive set of accounting rules or principles. In addition, from time to time in the future, there may be other items that management may exclude for purposes of this non-GAAP financial measure, and may in the future cease to exclude items that historically have been excluded for purposes of this non-GAAP financial measure. Likewise, management may determine to modify the nature of adjustments to arrive at this non-GAAP financial measure.

About Exagen

Exagen Inc. (Nasdaq: XGN) is a leading provider of autoimmune diagnostics, committed to transforming care for patients with chronic and debilitating autoimmune conditions. Based in San Diego County, California, Exagen’s mission is to provide clarity in autoimmune disease decision making and improve clinical outcomes through its innovative testing portfolio. The company’s flagship product, AVISE^® CTD, enables clinicians to more effectively diagnose complex autoimmune conditions such as lupus, rheumatoid arthritis, and Sjögren’s syndrome earlier and with greater accuracy. Exagen’s laboratory specializes in the testing of rheumatic diseases, delivering precise and timely results, supported by a full suite of AVISE-branded tests for disease diagnosis, prognosis, and monitoring. With a focus on research, innovation, education, and patient-centered care, Exagen is dedicated to addressing the ongoing challenges of autoimmune disease management.

For more information, please visit Exagen.com or follow @ExagenInc on X (formerly known as Twitter).

Forward Looking Statements

Exagen cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on Exagen’s current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: Exagen’s goals, strategies and ambitions; the potential utility and effectiveness of Exagen’s services and testing solutions, including the newly approved SLE and RA biomarkers; potential shareholder value and growth and profitability; preliminary financial information as of and for December 31, 2024; and guidance. The inclusion of forward-looking statements should not be regarded as a representation by Exagen that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Exagen’s business, including, without limitation: delays in reimbursement and coverage decisions from Medicare and third-party payors and in interactions with regulatory authorities, and delays in ongoing and planned clinical trials involving its tests; Exagen’s commercial success depends upon attaining and maintaining significant market acceptance of its testing products among rheumatologists, patients, third-party payors and others in the medical community; Exagen’s ability to successfully execute on its business strategies; third-party payors not providing coverage and adequate reimbursement for Exagen’s testing products, including Exagen’s ability to collect on funds due; Exagen’s ability to obtain and maintain intellectual property protection for its testing products; regulatory developments affecting Exagen’s business; and other risks described in Exagen’s prior press releases and Exagen’s filings with the Securities and Exchange Commission (SEC), including under the heading “Risk Factors” in Exagen’s Annual Report on Form 10-K for the year ended December 31, 2023, filed with the SEC on March 18, 2024 and any subsequent filings with the SEC. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Exagen undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

Contact:

Ryan Douglas
Exagen Inc.
[email protected]
760.560.1525

Orbit Phase 3 study of setrusumab in osteogenesis imperfecta continuing to planned second interim analysis, expected in mid-2025

Alvelestat, for Alpha-1 Antitrypsin Deficiency-associated Lung Disease, receives positive EMA opinion on European Orphan Designation Application; European Commission expected to issue final decision in first quarter 2025

LONDON, Jan. 12, 2025 (GLOBE NEWSWIRE) — Mereo BioPharma Group plc (NASDAQ: MREO) (“Mereo” or the “Company”), a clinical-stage biopharmaceutical company focused on rare diseases, today provided an update on its lead clinical programs, setrusumab, a monoclonal antibody in Phase 3 clinical development for the treatment of Osteogenesis Imperfecta (OI) through a partnership with Ultragenyx Pharmaceutical, Inc. (Ultragenyx) and alvelestat, an oral neutrophil elastase inhibitor being studied for the treatment of alpha-1 antitrypsin deficiency-associated lung disease (AATD-LD). The Company also reiterated its previous cash runway guidance that its current cash and cash equivalents are expected to fund operations into 2027, through multiple key inflection points.

“Based on the highly promising data from completed studies of setrusumab in OI, including the Phase 2 portion of the Orbit Study, we remain confident in the potential of setrusumab to become the standard-of-care in OI. We look forward to the second interim analysis expected mid-year as we continue our launch readiness activities in the key European markets,” said Dr. Denise Scots-Knight, Chief Executive Officer of Mereo BioPharma. “Additionally, EU Orphan Designation, which follows the granting of both Orphan Drug and Fast Track Designations from the FDA in the U.S., is another important milestone in our ongoing partnering process and our efforts to bring alvelestat to patients worldwide, including earlier stage patients who are not currently eligible for augmentation therapy in many countries. With our cash runway into 2027, we continue to be in a strong position to execute on our key milestones through 2025.”

Setrusumab (UX143)

As announced by the Company’s partner, Ultragenyx, the Phase 3 Orbit Study of setrusumab in OI is continuing to dose patients and progressing towards the planned second interim analysis expected in mid-2025, with a potential final analysis in the fourth quarter of 2025. Additionally, treatment is continuing in Cosmic, an open-label Phase 3 study evaluating setrusumab against intravenous bisphosphonate therapy in patients aged 2 to <7 years. Data from the Cosmic study will be evaluated in parallel with the Orbit interim and final analyses.

Alvelestat (MPH-966)

The European Medicines Agency (EMA)’s Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on the Company’s application for Orphan Designation for alvelestat. The COMP recommendation has been provided to the European Commission, which is expected to issue a final decision on the Orphan Designation in the first quarter of 2025. Alvelestat previously received Orphan Drug Designation and Fast Track Designation from the U.S. FDA in 2021 and 2022, respectively.

European Orphan Designation is awarded to therapeutic candidates targeting the treatment, prevention or diagnosis of life-threatening or chronically debilitating diseases with a prevalence of fewer than 5 in 10,000 people in the European Union which provide a significant benefit over available therapies, or for which no approved therapies exist. Therapeutics receiving EU Orphan Designation are eligible for ten years of marketing exclusivity upon approval, as well as fee reductions for various centralized activities including the Marketing Authorization Application, inspections and protocol assistance. Individual EU Member States also provide specific incentives to support the development, review and availability of Orphan Medicinal Products at the time of HTA evaluations and Pricing and Reimbursement negotiations.

About Mereo BioPharma

Mereo BioPharma is a biopharmaceutical company focused on the development of innovative therapeutics for rare diseases. The Company has two rare disease product candidates: setrusumab for the treatment of osteogenesis imperfecta (OI); and alvelestat, primarily for the treatment of severe alpha-1 antitrypsin deficiency-associated lung disease (AATD-LD). The Company’s partner, Ultragenyx Pharmaceutical, Inc., has completed enrollment in the Phase 3 portion of a pivotal Phase 2/3 study in pediatrics and young adults (5 to 25 years old) for setrusumab in OI and in the Phase 3 study in pediatric patients (2 to <7 years old) in the first half of 2024. The partnership with Ultragenyx includes potential additional milestone payments of up to $245 million and royalties to Mereo on commercial sales in Ultragenyx territories. Mereo has retained EU and UK commercial rights and will pay Ultragenyx royalties on commercial sales in those territories. Setrusumab has received orphan designation for osteogenesis imperfecta from the EMA and FDA, PRIME designation from the EMA, and Breakthrough Therapy designation and rare pediatric disease designation from the FDA. Alvelestat has received U.S. Orphan Drug Designation for the treatment of AATD and Fast Track designation from the FDA. Following results from ASTRAEUS and ATALANTa in AATD-lung disease, the Company has aligned with the FDA and the EMA on the primary endpoints for a Phase 3 pivotal study which, if successful, could enable full approval in both the U.S. and Europe. In addition to the rare disease programs, Mereo has two oncology product candidates, etigilimab, an anti-TIGIT; and navicixizumab for the potential treatment of late-line ovarian cancer. Navicixizumab has been partnered with Feng Biosciences, Inc. in a global licensing agreement that includes milestone payments and royalties. Mereo has also entered into an exclusive global license agreement with ReproNovo SA, a reproductive medicine company, for the development and commercialization of leflutrozole, a non-steroidal aromatase inhibitor.

Forward-Looking Statements

This press release contains “forward-looking statements” that involve substantial risks and uncertainties. All statements other than statements of historical fact contained herein are forward-looking statements within the meaning of Section 27A of the United States Securities Act of 1933, as amended, and Section 21E of the United States Securities Exchange Act of 1934, as amended. Forward-looking statements usually relate to future events and anticipated revenues, earnings, cash flows or other aspects of our operations or operating results. Forward-looking statements are often identified by the words “believe,” “expect,” “anticipate,” “plan,” “intend,” “foresee,” “should,” “would,” “could,” “may,” “estimate,” “outlook” and similar expressions, including the negative thereof. The absence of these words, however, does not mean that the statements are not forward-looking. These forward-looking statements are based on the Company’s current expectations, beliefs and assumptions concerning future developments and business conditions and their potential effect on the Company. While management believes that these forward-looking statements are reasonable as and when made, there can be no assurance that future developments affecting the Company will be those that it anticipates.

All of the Company’s forward-looking statements involve known and unknown risks and uncertainties some of which are significant or beyond its control and assumptions that could cause actual results to differ materially from the Company’s historical experience and its present expectations or projections. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical development process; the Company’s reliance on third parties to conduct and provide funding for its clinical trials; the Company’s dependence on enrollment of patients in its clinical trials; and the Company’s dependence on its key executives. You should carefully consider the foregoing factors and the other risks and uncertainties that affect the Company’s business, including those described in the “Risk Factors” section of its Annual Report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in the Company’s subsequent filings with the Securities and Exchange Commission. The Company wishes to caution you not to place undue reliance on any forward-looking statements, which speak only as of the date hereof. The Company undertakes no obligation to publicly update or revise any of our forward-looking statements after the date they are made, whether as a result of new information, future events or otherwise, except to the extent required by law.

Preliminary 2024 total revenue of $555 million to $560 million, exceeding top end of guidance, 
including Crysvita® revenue of $405 million to $410 million, and Dojolvi® revenue of $87 million to $89 million

2025 expected total revenue guidance of $640 million to $670 million

UX143 (setrusumab) Phase 3 Orbit study for osteogenesis imperfecta progressing to 
second interim analysis in mid-2025

GTX-102 Phase 3 Aspire study for Angelman syndrome expected to complete enrollment in second half of 2025

NOVATO, Calif., Jan. 12, 2025 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today reported preliminary unaudited 2024 revenue results, cash and investments at year end 2024, and provided financial guidance for 2025. The company also provided key program updates including that the UX143 (setrusumab) Phase 3 Orbit study is progressing with the second interim analysis in mid-2025.

“In 2024 we grew our business with four products in five indications globally, exceeding the updated revenue guidance we provided in August, and continuing our path toward profitability,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “In 2025, we will continue to expand our commercial base of business while we also prepare for the potential launch of our first gene therapy, in Sanfilippo syndrome, and to file a BLA for our second gene therapy, in Glycogen Storage Disease Type Ia. We are also executing on one of the most valuable late-stage pipelines in rare disease as we anticipate important pivotal Phase 3 results in osteogenesis imperfecta and completion of enrollment in our Phase 3 trial in Angelman syndrome. This progress puts us in the unique position to potentially launch three to four new therapies over the next couple of years, accumulating a total of eight to nine approved products over a 10-year period.”

Ultragenyx will present at the 43ᵗʰ annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025 at 10:30 AM PT. The live and archived webcast of the presentation will be accessible from the company’s website at https://ir.ultragenyx.com/events-presentations.

Financial Update

2024 Preliminary Revenue (unaudited) and 2025 Revenue Guidance

Total revenue for 2024 is estimated to be $555 million to $560 million, which exceeds the updated guidance range provided in August 2024, and represents approximately 29% growth versus 2023. Crysvita revenue for 2024 is estimated to be $405 million to $410 million, which also exceeds the guidance range, and represents approximately 24% growth versus 2023. Dojolvi revenue for 2024 is estimated to be $87 million to $89 million, also exceeding the guidance range, and represents approximately 25% growth versus 2023.

In 2025, total revenue is expected to be between $640 million and $670 million and the company expects to provide guidance on 2025 Crysvita and Dojolvi revenue as part of its fourth quarter and fiscal year 2024 financial disclosures in February 2025.

2024 Ending Cash Position (unaudited) and Decreasing 2025 Net Cash Used in Operations

Cash, cash equivalents, and available-for-sale investments were approximately $745 million as of December 31, 2024. In 2025, revenues are expected to grow approximately 14-20% compared to 2024 and the company will continue to prioritize expense management, leading to a decline in 2025 net cash used in operations compared to 2024.

The 2024 revenues and cash position included in this release are preliminary and are therefore subject to adjustment. The preliminary revenue results are based on management’s initial analysis of operations for the year ended December 31, 2024. The Company expects to issue full financial results for the fourth quarter and fiscal year 2024 in February 2025.

Recent Updates and 2025 Clinical Milestones

UX143 (setrusumab) monoclonal antibody for osteogenesis imperfecta (OI): Phase 3 Orbit study progressing to second interim analysis (IA2) expected in mid-2025 

Patients are being dosed in the ongoing Phase 3 Orbit and Cosmic clinical trials, which evaluate setrusumab in pediatric and young adult patients with OI. The randomized, placebo-controlled Phase 3 portion of the Orbit study is progressing towards the second interim analysis in mid-2025 and a potential final analysis in the fourth quarter 2025. Patients in the Cosmic study also are continuing to be treated with either setrusumab or intravenous bisphosphonates (IV-BP) therapy and will be evaluated in parallel with the Orbit interim and final analyses.

GTX-102 an antisense oligonucleotide for Angelman syndrome: Phase 3 study enrolling; expect enrollment completion in second half of 2025 

Enrollment in the global Phase 3 Aspire study began in December 2024 and is expected to enroll approximately 120 children ages four to 17 with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. Participants will be randomized 1:1 to receive GTX-102 by intrathecal injection via lumbar puncture or to the sham comparator group during the 48-week primary efficacy analysis period. The primary endpoint will be improvement in cognition assessed by Bayley-4 cognitive raw score, and the key secondary endpoint (with a 10% allocation of alpha) will be the Multi-domain Responder Index (MDRI) across the five domains of cognition, receptive communication, behavior, gross motor function, and sleep. Enrollment in the Phase 3 Aspire study is expected to complete in the second half of 2025.

The Phase 2/3 Aurora study, which will evaluate GTX-102 in other Angelman syndrome genotypes and ages, is expected to initiate in 2025.

UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): Biologics license application (BLA) submitted; expect Prescription Drug User Fee Act (PDUFA) decision on the application and launch in second half of 2025 

In December 2024, Ultragenyx submitted a BLA to the U.S. Food and Drug Administration for UX111 supported by the available data, including from the ongoing pivotal Transpher A study, demonstrating treatment with UX111 resulted in rapid and sustained decreased levels of heparan sulfate (HS) in cerebral spinal fluid (CSF) in patients with Sanfilippo syndrome type A. The sustained reduction in CSF HS exposure over time was correlated with improved long-term cognitive development compared to the decline observed during the same period of time in natural history data. A PDUFA decision and launch are expected in the second half of 2025.

DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): BLA filing expected in mid-2025 

Ultragenyx previously announced positive topline results from the Phase 3 GlucoGene study for the treatment of patients aged eight years and older. The study achieved its primary endpoint, demonstrating that treatment with DTX401 resulted in a statistically significant and clinically meaningful reduction in daily cornstarch intake compared with placebo at Week 48.

After the 48-week primary efficacy analysis period, crossover patients (previously treated with placebo) were eligible to receive DTX401. These patients were able to titrate cornstarch much more rapidly once they were confirmed to have been treated and had timely direct access to their glucose levels. Patients from the original DTX401 treatment arm who have reached 78 weeks also continued to reduce their daily cornstarch intake, while maintaining glycemic control. DTX401 has demonstrated a consistent and acceptable safety profile with no new safety concerns identified as of the data cut-off.

These results have been discussed with regulatory authorities in a pre-BLA meeting and will be included as part of a BLA submission in mid-2025.

UX701 AAV gene therapy for Wilson Disease: Phase 1/2/3 study ongoing; expect Cohort 4 enrollment completion in second half of 2025 

In Stage 1 of the Phase 1/2/3 Cyprus2+ study, 15 patients across three sequential dose cohorts were enrolled and demonstrated clinical activity as well as improvements in copper metabolism. Multiple responders completely tapered off their standard-of-care treatment with responses seen in all three dose cohorts.

The company expects to enroll a fourth cohort in Stage 1 at a moderately increased dose and with an optimized immunomodulation regimen to enhance the efficiency and efficacy of the gene therapy, with the objective of having the majority of patients come off standard-of-care treatment before selecting a dose for the randomized placebo-controlled stage of the study. Enrollment in Cohort 4 is expected to complete in the second half of 2025.

DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 study dosing patients; expect enrollment completion in early 2025 

Ultragenyx is randomizing and dosing patients in the ongoing Phase 3 study. The pivotal, 64-week study will include up to 50 patients, randomized 1:1 to DTX301 or placebo. The primary endpoints are response as measured by change in 24-hour ammonia levels and removal of ammonia-scavenger medications and protein-restricted diet. Enrollment is expected to be completed in early 2025.

About Ultragenyx 
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company’s website at: www.ultragenyx.com.

Forward-Looking Statements and Use of Digital Media 

Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx’s expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause the Company’s clinical development programs, commercial success of its products and product candidates, continued collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others,
the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, risks related to serious or undesirable side effects of our product candidates, the company’s ability to achieve its projected development goals in its expected timeframes, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, our limited experience in generating revenue from product sales, risks related to product liability lawsuits, our dependence on Kyowa Kirin for the commercial supply of Crysvita, fluctuations in buying or distribution patterns from distributors and specialty pharmacies, the transition back to Kyowa Kirin of our exclusive rights to promote Crysvita in the United States and Canada and unexpected costs, delays, difficulties or adverse impact to revenue related to such transition, smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, competition from other therapies or products, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and drug candidate
. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 6, 2024, and its subsequent periodic reports filed with the SEC.

In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/).

Contacts Ultragenyx Pharmaceutical Inc.


Investors

Joshua Higa
[email protected]

Media

Carolyn Wang
[email protected]