After rising significantly pre-market on Oct. 5 – when positive phase IIa results with Lomecel-B in Alzheimer’s disease (AD) were made public – shares of Longeveron Inc. (NASDAQ:LGVN) sank during the day and again Oct. 6 to close at $1.75, down 34 cents, or 16%.
The dark cloud of what the U.S. FDA called potential “systemic bias” rained on Amgen Inc.’s bid for full approval of Lumakras (sotorasib), a KRAS-G12C inhibitor that was granted accelerated approval in May 2021 for locally advanced or metastatic non-small-cell lung cancer after at least one systemic therapy.
Bad news for Amgen Inc. could mean upside for Mirati Therapeutics Inc., though the meeting of the U.S. FDA’s Oncologic Drugs Advisory Committee (ODAC) has yet to tell the tale regarding Lumakras (sotorasib), the former’s KRAS-G12C inhibitor.
Soleno Therapeutics Inc. (NASDAQ: SLNO) pulled off in a major way its randomized-withdrawal phase III study with DCCR (diazoxide choline) in Prader-Willi syndrome, boosting the shares by $22.37, or 505%, to close Sept. 26 at $26.80. “Our work is not done, but this was a big step,” said CEO Anish Bhatnagar.
A hot biopharma research approach and an equally hot therapeutic area came together in the potential $2.7 billion deal that pairs artificial intelligence (AI) company Valo Health Inc. with Novo Nordisk A/S, which will strive to advance new treatments for cardiometabolic diseases.
Upbeat phase III findings outweighed less encouraging late-stage trial news, as big pharma provided a mixed bag of cancer findings – with one data batch to form the basis of global approval bids, as Astrazeneca plc with Daiichi Sankyo Co. Ltd. unveiled interim results from a study called Tropion-Breast01. Targeting trophoblast cell surface antigen 2, datopotamab deruxtecan (dato) hit the mark in progression-free survival for patients with hormone receptor-positive, HER2-low or negative breast cancer in the study called Tropion-Breast01.
Travere Therapeutics Inc.’s narrow phase III miss in the study called Protect with the approved endothelin and angiotensin II receptor antagonist Filspari (sparsentan) in IgA nephropathy (IgAN) had Wall Street speculating about the fate of the compound, which is available for the indication by way of accelerated approval in the U.S., having been given the nod in February.
Having cast around for other routes, Histogen Inc. is throwing in the towel, its board having adopted a shutdown plan that includes the distribution of remaining cash to stockholders after a wind-down of operations. One of the company’s West Coast peers, Kinnate Biopharma Inc., had news, too. The firm disclosed the layoff of 70% of its workforce, including all employees at the Shanghai-based subsidiary Kinnjiu Biopharma Inc., leaving the outfit with 28 full timers.
Magnet Biomedicine Inc. emerged from stealth mode and pulled down a $50 million series A round co-led by founding investor Newpath Partners alongside Arch Venture Partners. The firm is advancing molecular glue discovery by way of rational selection and design, looking past known protein-protein interactions and what Magnet calls “tangential” degradation approaches to analyze the broader protein landscape and ultimately pair targets with rationally chosen presenters in the tissue where the disease manifests.
Iveric Bio Inc.’s regulatory win Aug. 4 drew attention to the already hot eye-disease space, where intriguing new developments include the possibility of an oral therapy for Stargardt disease. Belite Bio Inc. in late July finished enrollment of a phase III study with once daily tinlarebant, a retinol binding protein 4 antagonist for Stargardt’s. Data from 90 adolescent subjects in the study called Dragon are due in mid-2024.
