Eli Lilly and Co. took receipt of a complete response letter (CRL) from the U.S. FDA regarding the hoped-for accelerated approval of donanemab for early symptomatic Alzheimer's disease (AD). Regulators said the CRL was because of the limited number of AD patients with at least 12 months of drug exposure data provided in Lilly’s data package for the compound, which targets a modified form of beta amyloid plaque called N3pG. Shares of Indianapolis-based Lilly (NYSE:LLY) were trading midday at $347.33, down $3.75.

Cidara brings rezafungin to an FDA adcom

Next week, Cidara Therapeutics Inc. will make its case for rezafungin, an injectable treatment for candidemia and invasive candidiasis in adults, before the U.S. FDA’s Antimicrobial Drugs Advisory Committee. The FDA accepted the next-generation, once-weekly echinocandin derivative’s NDA in September and granted it a priority review, with March 22 PDUFA date. Top-line results from the phase III study of Restore in December 2021 showed rezafungin met the primary endpoint for the FDA’s NDA submission of all-cause mortality at day 30 and the primary endpoint for the EMA’s submission of global cure at day 14. The adcom convenes Jan. 24.

Mabwell out-licenses preclinical iron metabolism MAb to Disc Medicine in $412M deal

Just in time for the Chinese New Year, Mabwell Bioscience Co. Ltd. announced that its U.S. subsidiary, Mabwell Therapeutics Ltd., closed a licensing deal with Disc Medicine Inc. worth up to $412 million for preclinical candidate 9MW-3011, a humanized monoclonal antibody (MAb) that regulates iron metabolism. Under the terms of the deal, Disc gains exclusive rights to develop and commercialize the MAb in “all “territories outside of greater China and certain Southeast Asian countries,” Disc Medicine CEO John Quisel told BioWorld. In exchange, Mabwell receives a $10 million up-front payment and could receive up to $412.5 million in milestone payments plus double-digit sales-based royalties.

Seagen’s Tukysa gains US FDA accelerated approval for second cancer indication

The U.S. FDA granted accelerated approval to Seagen Inc. on Jan. 19 for Tukysa (tucatinib), extending the therapy’s reach beyond HER2-positive metastatic breast cancer, and clearing its way for RAS wild-type, HER2-positive unresectable or metastatic colorectal cancer. The drug will be used in combination with trastuzumab in adult patients whose disease has progressed after being treated with chemotherapy (fluoropyrimidine, oxaliplatin and irinotecan), and final approval is subject to additional trials confirming clinical benefit. Accelerated approval is based on phase II Mountaineer trial results that showed a 38% overall response rate, including complete responses in 3.6% of patients and partial responses in 35%. Analyst Jessica Fye, of JP Morgan Research, said the probability of success was already reflected in her company’s model. “Still, good news is good news, and we continue to expect investor interest in the stock to pick up as the focus in the story returns to fundamentals.”

FDA lifts clinical hold on Astellas Fortis AAV gene therapy trial in Pompe disease

The U.S. FDA has lifted the clinical hold on Astellas Pharma Inc.’s Fortis phase I/II trial evaluating adeno-associated virus (AAV) gene replacement therapy AT-845 in adults with late-onset Pompe disease. In June 2022, the FDA placed a clinical hold on the Fortis study following a serious adverse event of peripheral sensory neuropathy in one of the trial participants. When the hold was placed, the FDA informed Astellas that it did not have sufficient information to assess the risks to trial participants, and the agency required additional information about the adverse event, which was classified by the site investigator as grade 1 (mild in severity) and deemed serious due to its medical significance.

Neophore boosts DNA mismatch repair work with series B extension

Neophore Ltd. announced a £6 million (US$7.4 million) extension to its series B, bringing the total for the round to £21.5 million and enabling it to take forward a second DNA mismatch repair program. By inhibiting mismatch repair, the company’s small molecules alter the genotype of solid tumors, prompting the generation of neoantigens that are recognized by the immune system and leading on to an antitumor response. The new money “is to really push forward on at least one additional target, because the biology is turning out to be so fruitful from a therapeutic perspective,” said Matthew Baker, CEO.

India drug regulations under the scanner again after fresh WHO alert on contaminated cough syrup

India’s drug regulatory system is under the lens again after the World Health Organization’s (WHO) latest medical product alert on two substandard cough syrup manufactured in the country, varieties of which have led to the deaths of scores of children in late 2022. An alert was issued by WHO on Jan 11, 2023, on cough syrup identified in Uzbekistan and reported to WHO in late December 2022. “The reports are severely damaging for the industry at large,” Pushpa Vijayaraghavan, lead, health care and life sciences practice at Sathguru Management Consultants, an industry analyst firm based in Hyderabad, told BioWorld. “It is unfortunate that the entire industry’s reputation is at stake due to the callous actions of few non-compliant companies.”

Also in the news

Apex Labs, Apexigen, Beigene, Bellerophon, Biocorrx, Cadrenal, Celltrion, Cyteir, Deciphera, Endevica, Evaxion, Exelixis, Genentech, Hemostemix, Hyloris, Intercept, Janssen, Jazz, Linnaeus, Mineralys Mitsubishi Tanabe, Neochromosome, Nrx, Palatin, Panbela, Sonnet, Sotio, Stemedica, Sumitovant, Summit, Viracta, Windtree, Xortx, Zymeworks