Astrazeneca plc is acquiring rare diseases specialist Amolyt Pharma SA in a $1.05 billion deal, taking ownership of eneboparatide (AZP-3601), which is in phase III development for the treatment of hypoparathyroidism. Of the total, $800 million will be paid up front, with the balance of $250 million contingent upon achievement of a regulatory milestone. That was not specified, but it might be assumed relates to success in the phase III trial of eneboparatide, which is due to deliver top-line data before the end of 2024, and subsequent progress in filing and marketing approval. Lyon, France-based Amolyt will be integrated into Astrazeneca’s Alexion rare diseases business, extending the pharma’s franchise into rare endocrinology indications.

Geron making its case for imetelstat to ODAC

Changing care guidelines and a disagreement over whether a duration of response should be limited to responders or calculated across the study population are just two of the issues the U.S. FDA’s Oncology Drugs Advisory Committee (ODAC) is considering today as it evaluates Geron Corp.’s imetelstat as a treatment for transfusion-dependent anemia in adults with low- to intermediate-1 risk myelodysplastic syndromes (MDS) who have failed or no longer respond to erythropoiesis stimulating agents (ESAs), or who are not eligible for ESA treatment. After Geron started its phase III trial of imetelstat with the FDA-approved primary endpoint of eight-week red blood cell transfusion independence, MDS care guidelines changed as the general consensus among MDS experts was that only a 16-week or longer period of transfusion independence is clinically meaningful. Those guidelines are a moving target, Michael Savona, director of hematology research and a professor of medicine and cancer biology at Vanderbilt University School of Medicine, told the committee.

Spruce tumbles on phase II data in CAH

Shares of Spruce Biosciences Inc. (NASDAQ:SPRB) took a major hit to trade midday at $1.10, down $4.09, or 78%, after the South San Francisco-based firm disclosed mixed top-line results from two studies: Cahmelia-203 (phase IIb) with tildacerfont in adult classic congenital adrenal hyperplasia (CAH) and Cahptain-205 (phase II) with the same candidate in pediatric classic CAH. Tildacerfont is a second-generation, once-daily oral antagonist of the CRF1 receptor. The disease is made up of a group of genetic conditions that result in an enzyme deficiency altering the production of adrenal hormones.

Ocuterra struggles as lead eye drug fails in diabetic retinopathy

Top-line phase II results of privately held Ocuterra Therapeutics Inc.’s novel selective small-molecule RGD integrin inhibitor, nesvategrast (OTT-166), showed it failed to meet primary and key secondary endpoints in patients with diabetic retinopathy, prompting the Boston-based company to seek strategic alternatives. OTT-166 is Ocuterra’s only pipeline drug. While it was shown to be safe and well-tolerated at the 5% eye drop dose used in the DR-EAM (diabetic retionoapthy – early active management) trial, it did not demonstrate a statistically significant improvement on the primary endpoint, showing at least a two-step improvement on the diabetic retinopathy severity scale (DRSS) compared to placebo. Diabetic retinopathy, the most common diabetic eye disease, is the leading cause of vision loss, affecting nearly 10 million Americans.

Wuxi quits BIO in wake of US national security risk allegations 

Wuxi Apptec quit its membership in the Biotechnology Innovation Organization (BIO) after U.S. Congressman Rep. Mike Gallagher (R-Wis.) sent a March 5 letter to Attorney General Merrick Garland, asking the Department of Justice to investigate BIO because its lobbying efforts on behalf of Wuxi suggested it was operating as an unregistered agent of a foreign company while advancing the interests of the People’s Republic of China and the Chinese Communist Party.

Atrogi’s series B for novel beta-2 agonist in type 2 diabetes

Atrogi AB is raising a €30 million to €35 million (US$32.9 million to $38.4 million) series B round after announcing positive clinical data for ATR-258, a novel beta-2 adrenergic receptor agonist that is being lined up as a potential first-in-class insulin-independent treatment for type 2 diabetes (T2D).The results of the phase I/b double-blind, placebo-controlled safety trial showed ATR-258 is safe and well-tolerated in healthy volunteers and in T2D patients, when dosed over 28 days. That was too short to have an effect on glycosylated hemoglobin levels, the gold standard indicator of long-term control of glucose levels, but it adds to other clinical data Atrogi has gathered with a generic beta-2 agonist, paving the way for a phase II trial to start later in 2024.

Newco news: Asgard’s ‘direct reprogramming strategy’ garners €30M series A

Asgard Therapeutics AB has raised €30 million (US$32.8 million) in a series A round to advance a novel approach to cancer immunotherapy, in which it is proposed to reprogram cancer cells into functional antigen-presenting dendritic cells in vivo, activating a host immune response against the tumor. The therapy will use an adenoviral viral vector to deliver genes for the transcription factors PU.1, IRF8 and BATF3, which Asgard has shown can reprogram more than 100 different mouse and human tumor cells from a broad spectrum of tumor types, into conventional type 1 dendritic cells. That restarts formerly supressed cancer antigen presentation and sparks a patient-specific antitumor immune response. “[This is] a direct reprogramming strategy, which means we convert a cell type, in this case a cancer cell, into a completely new cell type directly,” Cristiana Pires, co-founder and CEO of Asgard, told BioWorld.

Hyundai Bioscience to acquire 23% stake in CRO firm ADM Korea

Hyundai Bioscience Corp. is set to become the largest shareholder of ADM Korea Inc., a Seoul, South Korea-based contract research organization (CRO) firm, by purchasing 5.02 million shares for ₩20.4 billion (US$15.5 million) – a 23% stake. Hyundai Bioscience agreed to acquire the Kosdaq-listed shares by May 20, 2024, from Mobiis Co. Ltd., a Korean nuclear fusion software and equipment company, which is divesting the CRO to focus on its core business. For Hyundai Bioscience, the move is expected to help it gain ground in the novel drug development space, specifically in the general-purpose antiviral drug market where it has focused since 2018.

Also in the news

4P-Pharma, Aderans, Aignostics, Alithea Genomics, Allergy, Alzprotect, Anocca, Astrazeneca, Astrocyte, Atsena, Bavarian Nordic, Bayer, Biocardia, Clearmind Medicine, Coherus, Crinetics, Cybin, Dimerix, Emendobio, ERS Genomics, Eyenovia, Imunon, Innocare, Ionis, Lexeo, Longboard, Mesoblast, Mission, Neurobo, Nicox, Novabay, Ocugen, Orexo, Pacira, Pepgen, Pfizer, Prilenia, Psyence, Puretech, Relation, Ricoh, Silence, Siolta, S. M. Discovery Group, Stemcardia, Stemson, Takeda, Tubulis, Zealand