Otsuka Pharmaceutical Co. Ltd. has gone back on efforts to get its medicine, Asimtufii (aripiprazole), a long-acting maintenance treatment for schizophrenia, approved in Europe, after the EMA gave a provisional negative opinion. Europe’s Committee for Medicinal Products for Human Use (CHMP) criticized the firm’s decision not to compare the drug with the reference medicine (Abilify Maintena) – available in Europe – in the main study it conducted. Also citing risk/benefit concerns, the CHMP wants to revoke the approval of Novartis Inc.’s monoclonal antibody, Adakveo (crizanlizumab), for the prevention of vaso-occlusive crises (pain crises) in patients 16 and older with sickle cell disease. Gilead Sciences Inc. and Novo Nordisk A/S, meanwhile, reported positive CHMP recommendations.

Heartseed raises ¥2B to continue trials of iPSC-derived cardiomyocytes for heart failure

Heartseed Inc. raised ¥2 billion (US$14.3 million) in a series D round to continue the phase I/II Lapis trial of its allogeneic induced pluripotent stem cell (iPSC)-derived cardiomyocytes for heart failure. The lead asset, HS-001, is an investigational cell therapy consisting of clusters of purified heart muscle cells (cardiomyocyte spheroids) derived from iPSCs that are designed to restore heart muscle and function in patients with advanced heart failure. The money raised will allow the Tokyo-based company to continue the phase I/II trial, in which HS-001 is transplanted into the diseased tissue of the heart during open-heart surgery in conjunction with a planned coronary artery bypass graft procedure.

UK investing £121M to revive clinical trial activity as part of overall life sciences boost

The U.K. government has announced £121 million (US$148 million) in funding to reboot the commercial clinical trials system after a sharp decline saw the number of industry-sponsored studies falling by 44% from 2017 to 2021. That took an estimated £930 million out of the National Health Service (NHS) over the five-year period, in reduced income from pharma companies and in the opportunity cost of having to provide standard of care for patients who otherwise would have had their medication provided as part of a clinical trial. The investment in the clinical trials ecosystem is part of £650 million in funding for a variety of initiatives that are intended to promote the development of the life sciences sector as a whole.

Despite setbacks momentum in ALS builds

Lack of efficacy brought the development of two investigational agents for amyotrophic lateral sclerosis (ASL) to a halt over the past week. On May 23, Wave Life Sciences Inc. disclosed that its stereopure antisense oligonucleotide WVE-004 failed to demonstrate clinical benefit after 24 weeks of treatment on a phase Ib/IIa trial in familial ALS patients or frontotemporal dementia patients. And on May 25, Apellis Pharmaceuticals Inc. and its partner, Swedish Orphan Biovitrum International AB, said that pegcetacoplan failed to meet its primary endpoint of a one-year phase II trial in patients with sporadic disease.

US FDA approvals show modest growth in 2023, while global drug approvals decline

U.S. FDA approvals had a modest increase during the first four months of 2023 compared to last year, but still fall short of the peaks seen in 2019 and 2021. With 50 drug approvals granted by the FDA through April, the biopharma industry is demonstrating a steady pace, tracking the same as both 2016 and 2017, albeit below the approvals the first four months of 2018-2021.

Newco news: Adolore comes to the fore with CA8 chronic pain gene therapy 

With overuse of opioids – the standard of care for many chronic pain cases – becoming something of an epidemic in the U.S., the availability of an alternative, non-opioid analgesic is a big draw. Established in 2021, Adolore Biotherapeutics Inc. is one company that could provide the answer, with its locally and long-acting gene therapies potentially providing a breakthrough that “knocks everybody’s socks off.” The biotech’s lead candidates – at the preclinical stage of development – consist of replication-defective vectors that activate carbonic anhydrase-8 (CA8), a compound in the body that is instrumental in regulating pain and its transmission.

Holiday notice

BioWorld's offices will be closed in observance of Memorial Day in the U.S. No issue will be published Monday, May 29.

Also in the news

Abbvie, Accropeutics, Adamis, Affamed, Agile, Annexon, Antabio, Apellis, Applied, Aptose, Bial, Bioinvent, Biontech, Bioxcel, Bristol Myers Squibb, Carmot, Celltrion, CNS, DMK, Dyne, Effector, Harmony, Hi-Bio, Immutep, Jaguar, Janssen, Legend, Lynk, Lyra, Mirati, Myriad Genetics, Novartis, Onconova, Pfizer, Sandoz, Stallergenes Greer, Stemcyte, Talaris, Tscan, Topas, Transgene, Vanda, Vivoryon