Shares in Akero Therapeutics Inc. more than doubled in value after the biotech announced results from a phase IIb nonalcoholic steatohepatitis (NASH) trial that it believes shows lead candidate efruxifermin could challenge other candidates in development for the fatty liver disease targeted by so many biotechs and big pharmas. Akero is yet to finalize a design for its phase III study for efruxifermin but the investor response shows that they agree with Akero’s thesis that it could outperform rivals such as Intercept Pharmaceuticals Inc.’s obeticholic acid, which was rejected by the U.S. FDA in June 2020 and is set for a refile in the coming months. There are no approved drugs for NASH, but other potential competitors include semaglutide from Novo Nordisk A/S and Eli Lilly and Co.’s tirzepatide, both of which were first developed for diabetes.

Rapid ‘Rayze’: Rayzebio closes $160M series D round, targets lead asset at Lutathera failures

Rayzebio Inc. raised $160 million in a series D round and unveiled its lead targeted radiopharmaceutical drug candidate, RYZ-101, which has entered clinical development in patients with gastroenteropancreatic neuroendocrine tumors (GEP-NETs) expressing the somatostatin receptor type 2. The rapidly moving firm aims to secure the industry’s first approval for a drug that deploys the alpha-emitting radioisotope Actinium-225 (²²⁵Ac), which delivers a highly potent and concentrated dose of radiation. It can be readily attached to a targeting moiety, such as a peptide, to focus the radiation’s cell-killing effects on the tumor, while minimizing systemic effects.

Rubius restructures to improve its platform

Rubius Therapeutics Inc. is taking a giant step back so that it can attempt taking a few steps forward. The company said it’s restructuring to focus on taking its platform from what it considers a good thing and turning it into an even better one. Rubius engineers red blood cells to become cancer fighters. It will axe two candidates that are in phase I studies for treating advanced solid tumors and do the same with about 75% of its workforce. It’s also considering selling its Smithfield, R.I., manufacturing facility. The savings is thought to extend Rubius’ cash runway until the end of 2023. At midday, the Cambridge, Mass.-based company’s stock (NASDAQ:RUBY) had dropped 15%, putting it below a dollar a share at around 91 cents each.

US drug pricing caught between the proverbial ‘rock and a hard place’

Pricing new drugs for the U.S. market, especially those treating rare diseases, is getting a lot more complex now that the Medicare inflation rebate is in play. The rebate provision in the newly enacted Inflation Reduction Act incentivizes companies to set higher launch prices for drugs that will be used by Medicare beneficiaries since future price increases will be limited to the rate of inflation. But at the same time, pricing advocates and the patient community, which often helps with the development of orphan drugs, are pushing for lower prices, particularly when a new drug is intended to be added to an already costly standard-of-care regimen.

Cuban drug for Parkinson's and Alzheimer´s diseases moves to phase III

An investigational drug developed in Cuba for the potential treatment of Alzheimer´s and Parkinson's diseases has moved forward to phase III trials there, where scientists are optimistic about results of previous studies. The candidate, named Neuroepo and branded Neuralcim, was developed by the Center of Molecular Immunology (or known by its Spanish acronym CIM), a state-owned lab in the Caribbean nation.

Occurx raises AU$16M to progress lead candidate OCX-063 in chronic kidney disease as it looks for partners

Melbourne-based Occurx Pty Ltd. announced a AU$16 million (US$11 million) series B round that will progress lead candidate OCX-063 to phase II trials in chronic kidney disease (CKD). The capital raise was jointly led by Brandon Biocatalyst and Uniseed and includes a AU$1.5 million grant from Australian biomedical incubator Cureator. The funds raised will take the company through the end of the phase II trial, which will start in the first quarter of 2023, Occurx CEO Darren Kelly told BioWorld.

Oncology drug dosing is evolving rapidly at FDA's Oncology Center of Excellence 

The toxicity associated with oncology therapies is the stuff of legend with clinicians and patients, and thus the U.S. FDA and manufacturers have been working to fine tune these dosing regimens. However, the FDA's Oncology Center of Excellence includes a programmatic effort to optimize dosing regimens, which led to an editorial by FDA officials that calls on industry to consider whether the maximum tolerated dose paradigm is really the optimal approach to oncology drug development.

Infectious disease efforts are 96% of bio/nonprofit deal values in 2022 

While COVID-19 remains a top research priority globally for government and nonprofit entities in partnership with biopharma companies, deal activity also is heavily focused on other infectious diseases, such as smallpox and influenza. In fact, only about half of the infectious disease deals between biopharma and nonprofits are focused on the current pandemic. The others, 57 in total, are working toward solutions for anthrax, HIV, malaria, respiratory infections, chickenpox, dengue fever, yellow fever, monkeypox and others.

The BioWorld Insider Podcast: $1B+ biopharma deals keep values afloat, even amid muted volume 

BioWorld writers bring you the details of new R&D alliances, financings, IPOs and M&A every day. These stories are the heartbeat of the relationships between industry giants and their smaller, more innovative peers. But what's the top-line read for dealmaking these days? With nearly 1,000 biopharma deals completed through early August amid a volatile economic climate, there's a lot to talk about. Join the BioWorld Insider podcast as we talk with Senior Analyst Karen Carey, whose data-driven research delivers a detailed picture of the ups and downs.

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