Ultragenyx and Mereo BioPharma to Present Setrusumab Data

NOVATO, Calif. and MOUNTAIN VIEW, Calif. and LONDON, Sept. 09, 2022 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), (“Ultragenyx”), a biopharmaceutical firm centered on the event and commercialization of novel merchandise for severe uncommon and ultra-rare genetic ailments and Mereo BioPharma Group plc (NASDAQ: MREO), (“Mereo”), a clinical-stage biopharmaceutical firm centered on uncommon ailments and oncology, at this time introduced that extra knowledge from Mereo’s Phase 2b ASTEROID research associated to setrusumab (UX143) and info on the Phase 2/3 Orbit research shall be offered on the American Society for Bone and Mineral Research 2022 Annual Meeting (ASBMR) being held September 9-12, 2022, in Austin, Texas. Setrusumab is a monoclonal antibody in improvement for the remedy of Osteogenesis Imperfecta (OI), a uncommon illness affecting roughly 60,000 people within the US and Europe.

Ultragenyx will current the most recent outcomes from the beforehand offered ASTEROID research and an outline of the design of the continuing international, seamless Phase 2/3 Orbit research on the assembly. Data may also be offered from the IMPACT Survey, the biggest assortment of information in OI and a joint analysis mission between the Osteogenesis Imperfecta Foundation (OIF), and the umbrella group Osteogenesis Imperfecta Federation Europe (OIFE) and their members, supported by Mereo BioPharma.

Details of the ASBMR knowledge displays are as follows:

Abstract Title: Orbit: A Randomized, Double-Blind, Placebo-controlled, Phase 2/3 Study to Assess the Efficacy and Safety of Setrusumab in Pediatric and Young Adult Participants with Osteogenesis Imperfecta (Ominsky M et al.)
Presentation: Poster
Session Date and Time: Saturday, September 10, 2022, 13:00-15:00 Central Time
Presentation Number: LB SAT-897

Abstract Title: The IMPACT survey offers distinctive insights into the experiences of adults with osteogenesis imperfecta (OI) via self-reported knowledge (Rauch F et al.)
Presentation: Poster
Session Date and Time: Sunday, September 11, 2022, from 13:00-15:00 Central Time
Presentation Number: SUN-667

Abstract Title Changes in BMD and Bone Turnover Markers Following Discontinuation of Setrusumab After 1 Year of Treatment in Adults with Osteogenesis Imperfecta (Javaid Okay et al.) 
Presentation: Oral Presentation
Session Date and Time: Monday, September 12, 2022, from 11:45-12:00 Central Time
Presentation Number: 1112

About Setrusumab (UX143)
Setrusumab is a totally human monoclonal antibody that inhibits sclerostin, a protein that acts on a key bone-signaling pathway that inhibits the exercise of bone-forming cells. The objective of blocking inhibitory results of sclerostin is to improve new bone formation, bone mineral density, and bone power. Sclerostin inhibition additionally reduces extreme bone resorption, additional enhancing its influence on bone density. In varied mouse fashions of OI, the usage of anti-sclerostin antibodies was proven to stimulate bone formation, enhance bone mass and density, and scale back bone fragility as mirrored in elevated lengthy bone power and lowered the variety of fractures.

Mereo BioPharma’s Phase 2b research (ASTEROID) remedy section of the dose-finding research of setrusumab for the remedy of OI in 112 adults was concluded in 2019. The ASTEROID research demonstrated remedy with setrusumab resulted in a transparent, dose-dependent and statistically vital impact on bone formation and bone density at a number of anatomical websites amongst grownup individuals with OI. Off-treatment outcomes had been adopted.

Ultragenyx and Mereo are collaborating on the event of setrusumab globally primarily based on the collaboration and license settlement between the events. The corporations are planning a complete late-stage program to proceed improvement of setrusumab in pediatric and younger grownup sufferers throughout OI sub-types I, III and IV.

About Ultragenyx
Ultragenyx is a biopharmaceutical firm dedicated to bringing novel merchandise to sufferers for the remedy of significant uncommon and ultra-rare genetic ailments. The firm has constructed a various portfolio of permitted therapies and product candidates aimed toward addressing ailments with excessive unmet medical want and clear biology for remedy, for which there are usually no permitted therapies treating the underlying illness.

The firm is led by a administration group skilled within the improvement and commercialization of uncommon illness therapeutics. Ultragenyx’s technique is based upon time- and cost-efficient drug improvement, with the objective of delivering secure and efficient therapies to sufferers with the utmost urgency. For extra info on Ultragenyx, please go to the corporate’s web site at: www.ultragenyx.com.

About Mereo BioPharma
Mereo BioPharma is a biopharmaceutical firm centered on the event of modern therapeutics for uncommon ailments and in oncology and plans to commercialize chosen uncommon illness packages. The Company has developed a portfolio of six medical stage product candidates. The Company has two uncommon illness product candidates, setrusumab for the remedy of osteogenesis imperfecta (OI) and alvelestat for the remedy of extreme Alpha-1 antitrypsin deficiency (AATD) and Bronchiolitis Obliterans Syndrome (BOS). The Company’s companion, Ultragenyx Pharmaceutical, Inc., has initiated a pivotal Phase 2/3 pediatric research in younger adults (5-25 years outdated) for setrusumab in OI and expects to provoke a research in pediatric sufferers (2-5 years outdated) within the second half of 2022. The partnership with Ultragenyx consists of potential milestone funds of up to $254 million and royalties to Mereo on Ultragenyx territories. Mereo has retained EU and UK business rights and pays Ultragenyx royalties on these territories. Alvelestat has obtained U.S. Orphan Drug Designation for the remedy of AATD and optimistic top-line knowledge had been lately reported from a Phase 2 proof-of-concept research in North America, Europe and the UK. Mereo’s lead oncology product candidate, etigilimab (anti-TIGIT), is at present in an open label Phase 1b/2 basket research evaluating anti-TIGIT together with an anti-PD-1 in a spread of tumor varieties together with three uncommon tumors and three gynecological carcinomas, cervical, ovarian, and endometrial carcinomas. The Company’s second oncology product, navicixizumab, for the remedy of late line ovarian most cancers, has accomplished a Phase 1 research and has been partnered with OncXerna Therapeutics, Inc., previously Oncologie, Inc. The international licensing settlement with OncXerna consists of funds of up to $300 million in milestones and royalties.

Mereo Forward-Looking Statements
This press launch accommodates “forward-looking statements.” All statements aside from statements of historic reality contained on this press launch are forward-looking statements throughout the which means of Section 27A of the United States Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the United States Securities Exchange Act of 1934, as amended (the “Exchange Act”). Forward-looking statements often relate to future occasions and anticipated revenues, earnings, money flows or different points of our operations or working outcomes. Forward-looking statements are sometimes recognized by the phrases “believe,” “expect,” “anticipate,” “plan,” “intend,” “foresee,” “should,” “would,” “could,” “may,” “estimate,” “outlook” and related expressions, together with the unfavourable thereof. The absence of those phrases, nonetheless, doesn’t imply that the statements should not forward-looking. These forward-looking statements are primarily based on Mereo’s present expectations, beliefs and assumptions regarding future developments and business situations and their potential impact on Mereo. While administration believes that these forward-looking statements are cheap as and when made, there will be no assurance that future developments affecting Mereo shall be people who it anticipates.

All of Mereo’s forward-looking statements contain identified and unknown dangers and uncertainties, a few of that are vital or past its management and assumptions that might trigger precise outcomes to differ materially from Mereo’s historic expertise and its current expectations or projections. You ought to rigorously contemplate the foregoing elements and the opposite dangers and uncertainties that have an effect on Mereo’s business, together with these described within the “Risk Factors” part of its newest Annual Report on Form 20-F, experiences on Form 6-Okay and different paperwork furnished or filed from time to time by Mereo with the Securities and Exchange Commission. Mereo needs to warning you not to place undue reliance on any forward-looking statements, which converse solely as of the date hereof. Mereo undertakes no obligation to publicly replace or revise any of our forward-looking statements after the date they’re made, whether or not on account of new info, future occasions or in any other case, besides to the extent required by legislation.

Ultragenyx Forward-Looking Statements and Use of Digital Media
Except for the historic info contained herein, the issues set forth on this press launch, together with statements associated to Ultragenyx’s expectations and projections relating to its business plans and goals, the therapeutic potential and medical advantages of its product candidates, expectations relating to the security and tolerability of its product candidates, and future medical developments for its product candidates are forward-looking statements throughout the which means of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements contain substantial dangers and uncertainties that might trigger the corporate’s medical improvement packages, future outcomes, efficiency or achievements to differ considerably from these expressed or implied by the forward-looking statements. Such dangers and uncertainties embrace, amongst others, the flexibility of the corporate to efficiently develop UX143, the results of the COVID-19 pandemic on the corporate’s medical actions, business and working outcomes, dangers associated to reliance on third social gathering companions to conduct sure actions on Ultragenyx’s behalf; the potential for any license or collaboration settlement, together with Ultragenyx’s collaboration settlement with Mereo, to be terminated, uncertainty and potential delays associated to medical drug improvement, the danger that medical outcomes demonstrated in interim knowledge from Ultragenyx’s medical trials might materially change or elevated incidents of adversarial occasions as affected person enrollment continues and/or extra affected person knowledge turns into out there, Ultragenyx’s capacity to obtain its projected improvement objectives in its anticipated timeframes, dangers and uncertainties associated to the regulatory approval course of, smaller than anticipated market alternatives for the corporate’s merchandise and product candidates, manufacturing dangers, competitors from different therapies or merchandise and different issues that might have an effect on sufficiency of current money, money equivalents and short-term investments to fund operations, the corporate’s future working outcomes and monetary efficiency, the timing of medical trial actions and reporting outcomes from similar, and the provision or business potential of Ultragenyx’s merchandise and drug candidates. Ultragenyx undertakes no obligation to replace or revise any forward-looking statements. For an extra description of the dangers and uncertainties that might trigger precise outcomes to differ from these expressed in these forward-looking statements, in addition to dangers relating to the business of Ultragenyx normally, see Ultragenyx’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on July 29, 2022, and its subsequent periodic experiences filed with the Securities and Exchange Commission.

In addition to its SEC filings, press releases and public convention calls, Ultragenyx makes use of its investor relations web site and social media retailers to publish necessary details about the corporate, together with info that could be deemed materials to buyers, and to adjust to its disclosure obligations beneath Regulation FD. Financial and different details about Ultragenyx is routinely posted and is accessible on Ultragenyx’s investor relations web site (https://ir.ultragenyx.com/) and LinkedIn web site (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/mycompany/).

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