A new deal between Adaptimmune Therapeutics plc and Roche Group's Genentech Inc. aims to develop allogeneic cell therapies for up to five shared cancer targets. Should the agreement win regulatory clearance, still pending, Adaptimmune will receive $150 million up front and additional payments of $150 million over five years, barring early termination of the agreement. In addition, it could be eligible for development, regulatory and commercial milestones payments from Roche exceeding $3 billion, plus royalties.

FDA puts Biomarin gene therapy trial on hold, following expert discussion on safety

The FDA has put a hold on a clinical study of a rare disease gene therapy BMN 307 from Biomarin Pharmaceutical Inc., after several mice developed liver tumors following a high dose in a preclinical trial. The FDA made the ruling after experts from its Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) discussed safety risks of adeno-associated virus (AAV) vector-based gene therapy products like BMN 307. The announcement over the Labor Day holiday sent shares in Biomarin (NASDAQ:BMRN) sharply down when trading resumed Sept. 7.

Servier, Neurochlore strike out with bumetanide in ASD phase IIIs

Les Laboratoires Servier SAS and Neurochlore SAS said the phase III studies with the diuretic blood pressure medicine bumetanide failed to show signs of efficacy in autism spectrum disorder (ASD) vs. placebo in children and adolescents. The trials have been terminated early. Double-blind and randomized, the experiments began four years ago in 14 countries, including 11 in Europe. A total of 422 patients with moderate to severe ASD were enrolled, each study with 211 subjects according to their ages (2 to 6 and 7 to 17 years). They were followed for six months with placebo control, and another six months on the drug. It was the largest phase III European program in the indication, the companies noted.

NMPA grants approval for China’s first independently developed CAR T therapy

JW Therapeutics Co. Ltd.’s CAR T product received marketing approval from the NMPA, making it the first CAR T product to be independently developed in China and approved as a category I biologics product, as well as the sixth approved CAR T product globally. The drug, relmacabtagene autoleucel injection (relma-cel), marketed as Carteyva, is an anti-CD19 autologous CAR T-cell immunotherapy product. It is indicated for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy.

Enterobiotix adds $21.5M series A for microbial therapies

LONDON – Microbiome specialist Enterobiotix Ltd. closed a $21.5 million series A to advance development of its live bacteria fecal transplant drug pipeline. The company already has established a stool sample collection, screening and processing facility that has U.K. Medicines and Healthcare products Agency approval, and also has treated people on a named patient basis. The new money will be used to further develop analytical tools for the precise characterization and standardization of products, for further investment in the supply chain, and to move toward formal clinical trials.

VC rounds reach the summit, IPOs almost there; $87.5B raised to date

One more completed stock market debut and 2021 will have officially matched last year with the highest number of biopharma IPOs on record, and amounts collected through venture capital financings have now risen to the top as well. With all financing types combined, public and private, the industry has raised a total of $87.48 billion through early September, more than any other full year prior to 2020, which had $91.5 billion during the same time frame. The 1,120 completed financings this year compare with 1,058 by this point last year. The value of financings is tracking about 4% shy of last year, which reached $134.5 billion by the end of year. The volume of financings is up by 6%.

India begins approving lost list of banned FDC drugs

India’s drug regulator has approved 31 fixed-dose combination (FDC) drugs out of more than 300 banned products, after an assessment by its drugs technical advisory board. It has also asked for more phase IV postmarketing data for 19 others. The latest notifications by the Central Drugs Standard Control Organisation (CDCSO) at the end of August are part of longstanding efforts to streamline its largely unregulated FDC market. The approved drugs include several painkillers or analgesics.

Eyegene cleared to begin phase I/IIa trial for mRNA COVID-19 vaccine in South Korea

Eyegene Inc. has received the green light from South Korea’s Ministry of Food and Drug Safety (MFDS) for EG-COVID, its mRNA COVID-19 vaccine, to enter the clinic. With the MFDS go-ahead, Eyegene will now begin a phase I/IIa trial, to be conducted in two stages, to evaluate the vaccine’s safety, tolerability and immunogenicity. Stage one will involve 45 participants at Asan Medical Center in Seoul and stage two, involving 125 patients, will start based on the data from stage one. “We are currently looking through applications and aim to dose the first patients for stage one within September 2021,” an Eyegene spokesman told BioWorld. “We target having interim results by the end of November or early December and commercializing the vaccine within 2022.”

Thaler comes up short in district court fight over inventorship rights for AI

The question of whether an artificial intelligence algorithm can be an inventor has been making the rounds in the past couple of years, and the question came up again recently in the U.S. District Court for the Eastern District of Virginia. Stephen Thaler, who developed the DABUS algorithm that has been credited with two inventions, failed to persuade the court that an algorithm qualifies as an “individual,” and thus patents must still be assigned to humans, at least where the U.S. Patent and Trademark Office is concerned.

Holiday notice

BioWorld's offices were closed in observance of Labor Day in the U.S. No issue was published Monday, Sept. 6.

Also in the news

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