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Post by mytakeonit on Sept 2, 2020 2:30:01 GMT -5
Yeah ... it tough being Wonder Woman.
But, that's mytakeonit
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Post by neil36 on Jan 23, 2021 21:18:36 GMT -5
Barron’s article yesterday titled “5 Biotech Stocks That Could Be Worth a Look”. It included the following paragraph: — Acceleron has two potential blockbusters, writes Brill in her Monday report. In partnership with Bristol Myers Squibb (BMY), the company recently launched sales of an anemia drug Reblozyl. An investigational drug called sotatercept did well in Phase 2 trials for pulmonary arterial hypertension, a serious congenital disease that can require lung transplants. — Anyone familiar with “sotatercept”? It’s only in Stage 2 trials, but Barron’s seems to be implying high hopes for its entry into the PAH market – Sotatercept received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) in PAH in 2019 – – In the spring of 2020, sotatercept received Breakthrough Therapy designation from the FDA and Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), both in PAH – December 14, 2020 07:00 AM Eastern Standard Time CAMBRIDGE, Mass.--(BUSINESS WIRE)--Acceleron Pharma Inc. (Nasdaq: XLRN), a biopharmaceutical company dedicated to the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, today announced that the European Commission (EC) has granted orphan designation to sotatercept for the treatment of patients with pulmonary arterial hypertension (PAH). “We’re thrilled at the European Commission’s decision to grant orphan designation to sotatercept in PAH,” said Habib Dable, President and Chief Executive Officer of Acceleron. “We fully intend to take advantage of the benefits that this and other special statuses—including Orphan Drug and Breakthrough Therapy designations in the United States and PRIME designation in Europe—provide to drug developers as we work with health authorities to deliver this potential new backbone therapy in PAH to patients in need as quickly as possible.” The EC grants orphan designation to medicines intended to treat, prevent or diagnose a disease of low prevalence (fewer than 5 individuals per 10,000 population) that is life-threatening or chronically debilitating. To encourage the development of such medicines, the designation carries with it certain incentives, including scientific advice and assistance with clinical trial protocols, and the potential for a 10-year period of market exclusivity. For additional information about orphan designation, visit the EMA website. Acceleron is currently advancing a Phase 3 development plan for sotatercept, beginning with the registrational trial known as STELLAR expected to initiate by the end of this year. About Sotatercept Sotatercept is an investigational reverse-remodeling agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR-II signaling, which is a key molecular driver of PAH. The PULSAR Phase 2 trial evaluating sotatercept in combination with approved PAH-specific medicines in patients with PAH achieved its primary endpoint of improvement in pulmonary vascular resistance and its key secondary endpoint of improvement in 6-minute walk distance. Sotatercept was generally well tolerated in the trial. Adverse events observed in the study were generally consistent with previously published data on sotatercept in other diseases. Following the PULSAR results, sotatercept was granted Breakthrough Therapy designation from the FDA and Priority Medicines designation from the EMA in PAH. Sotatercept is also being evaluated in the SPECTRA Phase 2 exploratory trial. The Company recently presented details of its Phase 3 development plan, including the design for the registrational STELLAR trial, which is expected to be initiated before the end of 2020. Acceleron is planning two additional Phase 3 studies in patients with PAH: the HYPERION trial, exploring early intervention with sotatercept, and the ZENITH trial assessing later-stage intervention. Sotatercept is an investigational therapy that is not approved for any use in any country. Sotatercept is part of a licensing agreement with Bristol Myers Squibb. —————- I think sotatercept is why UTHR is willing to spend $105 million to build the biggest time gap possible |
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Post by neil36 on Jan 24, 2021 13:05:15 GMT -5
Sototeracept is only in Phase 2 trials. But Barron’s hailed it as a potential blockbuster $3 billion a year) PAH drug last month.
Now I understand why Martine spent the money for the accelerated revue process which will address additional indications. They want to build the biggest moat possible before sototeracept even gets to Phase 3 trials
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