New top-line data from Scholar Rock Holding Group’s phase II Topaz trial of apitegromab (SRK-015) in patients with type 2 and type 3 spinal muscular atrophy generated enough proof-of-concept results for the company to plan on initiating a phase III for later this year. SRK-015, a myostatin activator inhibitor, demonstrated no safety signals for the period, and all 57 patients completing the study said they would return for the extension period. However, the Cambridge, Mass.-based company’s stock (NASDAQ:SRRK) was struggling at midday at shares were trading 14% lower.

Mineralys secures $40M series A to advance hypertension drug in-licensed from MTPC

Mineralys Therapeutics Inc., a Philadelphia-based company developing an aldosterone synthase inhibitor in-licensed from Mitsubishi Tanabe Pharma Corp. (MTPC), has raised $40 million in series A funding to complete a phase II proof-of-concept study in hypertension for the candidate, known as MLS-101. The funding will also support preparatory work for a pivotal development program, Mineralys CEO Jon Congleton told BioWorld. HBM Healthcare Investments and Samsara Biocapital co-led the round with participation from founding investors Catalys Pacific and Adam Street Partners.

Proposed NIST rule to protect Bayh-Dole draws strong reaction

Nearly 16,500 groups and individuals added their two cents on a proposed National Institute of Standards and Technology (NIST) rule clarifying that the U.S. government, under the Bayh-Dole Act, can’t march in on patents derived from federally funded research just because it doesn’t consider the price of the resulting product “reasonable.” Many of the comments submitted by the April 5 deadline oppose the clarification proposed in the waning days of the Trump administration. But the clarification simply restates the intent of the 1980 law that has been followed by Health and Human Services officials in both Democratic and Republican administrations, according to Joseph Allen, executive director of the Bayh-Dole Coalition and a former Sen. Birch Bayh (D-Ind.) staffer who helped secure passage of the landmark act more than 40 years ago.

New clinical milestones reached as multiple COVID-19 vaccines and therapies advance

Plans for a late-stage test of Valneva SE's COVID-19 vaccine candidate, clinical progress on a different vaccine from Novavax Inc. and advancement for two midstage therapies targeting the virus made clear Tuesday that industry efforts to battle the pandemic remain in high gear. Meanwhile, a new journal article on the Abcellera Biologics Inc. and Eli Lilly and Co. asset bamlanivimab pointed to "the feasibility of strategies to rapidly identify neutralizing human mAbs as part of an initial response to an evolving pandemic," researchers wrote in Science Translational Medicine.

Europe gears up for MCMs for future threats

The European Commission opened a public consultation to help it develop the Health Emergency Preparedness and Response Authority (HERA), a new initiative to better prepare Europe for future cross-border health threats through the development of medical countermeasures (MCMs). Stemming from the February launch of a European biodefense preparedness plan against COVID-19 variants, HERA will provide a permanent structure for risk modelling, global surveillance, technology transfers, manufacturing capacity, supply chain risk mapping, flexible manufacturing capacity and vaccine and medicine R&D.

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