The U.S. FDA followed up an unfavorable advisory committee meeting with a complete response letter (CRL) to Lykos Therapeutics Inc. for midomafetamine (MDMA) capsules, which would be used in combination with psychological intervention in post-traumatic stress disorder. The agency wants another trial. CEO Amy Emerson called the FDA move “deeply disappointing,” and San Jose, Calif.-based Lykos plans to request a meeting with the agency to ask for reconsideration.

Ascendis notches FDA approval for hypoparathyroidism treatment

After many months of jockeying, the U.S. FDA has approved Ascendis Pharma A/S’s hormone replacement therapy Yorvipath (palopegteriparatide) for treating hypoparathyroidism. Ascendis said this is the first and only treatment for adults with the rare endocrine disease. In May 2023, the FDA issued a complete response letter for the NDA, citing concerns about the manufacturing control strategy for variability of delivered dose in the drug/device combination product but not about the clinical data. The FDA later extended the PDUFA target action date by three months to Aug. 14 to provide time for a full review of the submission. Palopegteriparatide received EMA approval for chronic hypoparathyroidism as Yorvipath in November 2023.

MSD acquires Curon’s CD3 x CD19 bispecific antibody for up to $1.3B

Merck & Co. Inc. is acquiring Curon Biopharmaceutical Ltd.’s bispecific antibody CN-201 that targets CD3 and CD19 for B-cell associated diseases, for up to $1.3 billion. Under the terms of the deal, Merck (known as MSD outside of North America) through a subsidiary will acquire full global rights to CN-201 for an up-front payment of $700 million in cash. Curon is also eligible to receive up to $600 million in development and regulatory-based milestone payments. The deal is expected to close in the third quarter of 2024.

ARS wins FDA nod for needle-free allergy drug Neffy

Executives of ARS Pharmaceuticals Inc. are anticipating a quick switch for severe allergy patients following the U.S. FDA approval of Neffy (epinephrine nasal spray 2 mg), marking the first needle-free treatment option. Neffy is cleared for use in treating type I allergic reactions, including anaphylaxis, in adults and children weighing 30 kilograms or more. Type 1 reactions can include allergic responses due to food, medications or insect bites, and typically result in an estimated 500,000 emergency room visits each year. Immediate treatment with epinephrine has proved effective, but the sole availability as an autoinjector has limited use.

BioWorld Cancer Index drops overall; Blueprint, TG catapulted in Q1

The BioWorld Cancer Index (BCI) finished July down 3.94% for the year, maintaining its downward trend after reaching a high of 11.07% at the end of February and closing in March with a 3.92% gain. This follows a stellar performance in 2023, when BCI closed the year with a 76.26% gain. Through July 2024, the BCI saw an even split, with 10 stocks experiencing declines and another 10 posting gains. Leading the pack, Beyondspring Inc. surged by 116% in the first quarter, while TG Therapeutics Inc. was up 16% on its first-quarter results.

Cullinan ends $625M deal, returns Harbour’s cancer drug

Cullinan Therapeutics Inc. terminated development of Harbour Biomed (HBM) Holdings Ltd.’s bispecific B7H4 x 4-1BB immune activator, CLN-418 (HBM-7008), after reviewing phase I data. Cullinan had licensed the asset from China and U.S.-based Harbour for solid tumors in a potential $625 million deal in February 2023, which included $25 million in an up-front payment and $600 million in potential development, regulatory and sales-based milestone payments. Harbour was also eligible to receive tiered royalties of up to 20% on potential future U.S. commercial sales.

US HHS wins another round in price negotiation challenges

U.S. district courts have now handed the Department of Health and Human Services (HHS) an undefeated record in getting constitutional challenges to Medicare price negotiations dismissed. The latest victory occurred in Ohio, where a court that had rejected the department’s early motion to dismiss a challenge brought by the Chamber of Commerce tossed the complaint last week after considering the merits of the case. Since the dismissal was based on lack of standing and improper venue, the court didn’t address the constitutional issues raised. The decision leaves only one challenge remaining at the district court level; all the others are on appeal.

Also in the news

An2, Arrowhead, ARS Pharma, Ascendis, Atavistik, Avidity, Bristol Myers Squibb, Can-Fite, Cantargia, Cerevance, Crispr, Curon, Evotec, Humacyte, Kiromic, Merck, Mind Medicine, Neuren, Sandoz, Scpharmaceuticals, Sernova, Shanghai Junshi, Telomir, Velia, Vertex, Wave, Zevra