Mostly positive phase III data prompts Vertex to seek an NDA
Despite Vertex Pharmaceuticals Inc.’s mostly positive phase III study results of a non-opioid for treating moderate to severe acute pain, the data for a key secondary endpoint show VX-548 is not as effective as Vicodin (hydrocodone, Abbvie Inc.).

Mostly positive phase III data prompts Vertex to seek an NDA
Despite Vertex Pharmaceuticals Inc.’s mostly positive phase III study results of a non-opioid for treating moderate to severe acute pain, the data for a key secondary endpoint show VX-548 is not as effective as Vicodin (hydrocodone, Abbvie Inc.).
As ODAC questions loom, 2seventy ‘doubles down’ on Abecma
2seventy bio Inc. shares (NASDAQ:TSVT) rose 15% or 52 cents, to close Jan. 30 at $4.01 on word that the Cambridge, Mass.-based firm is selling its R&D pipeline to Regeneron Pharmaceuticals Inc., which will move the work forward by way of a new company called Regeneron Cell Medicines.
Cour’s nanoparticles shut off autoimmunity with $105M series A
A company focused on regulating immune response through nanoparticle technology, Cour Pharmaceuticals Development Co. Inc. has raised $105 million in a series A round to move its lead autoimmune disease products into phase IIa trials.
Australia takes first steps toward meaningful reforms to health technology process
The biopharma industry lauded the first steps the Australian government has taken to widen access for drugs and devices via reforms to the current health technology assessment process that has remained unchanged for 30 years.
Through comparative genomics, becoming tough as nails – or squirrels
Humans love to think of our species as unique. But on a genetic level, such uniqueness is surprisingly hard to find. And while that may be a blow to the ego, it also means that an evolutionary lens is one way to search for insights into human diseases. Animals are “adapted to use the same genes that you and I have, but in very different ways,” Ashley Zehnder told BioWorld. Zehnder is co-founder and CEO of Fauna Bio Inc., which uses comparative genomics to identify gene networks that underlie disease resistance in different animal species.
Today's news in brief
BioWorld briefs for Jan. 30, 2024.
Newco news: Vision Care’s iPS retinal cells primed for global market
Vision Care Group CEO Masayo Takahashi led the world's first clinical study of a retinal cell transplant derived from induced pluripotent stem cells (iPS cells) in 2014 when she led the Laboratory for Retinal Regeneration at Japan’s Riken Center for Biosystems Dynamics Research. In 2019, she founded Vision Care and subsequently founded two subsidiary companies dedicated to developing cell and gene therapies.
Combined candidiasis fungicidal appears superior to miconazole alone in phase II
The topical combination therapy miconazole plus domiphen bromide (MCZ-DB) resolved signs and symptoms of acute vulvovaginal candidiasis (VVC) and eradicated the yeast Candida albicans from vaginal swabs to a greater extent than miconazole (MCZ) alone in a phase II study, according to Hyloris Pharmaceuticals SA and Purna Female Healthcare.
Pearsanta acquires MDNA for $25M
Pearsanta Inc., a subsidiary of Aditxt Inc. formed in Feb. 2023, acquired MDNA Life Sciences Inc. for $25 million in stock, marking Aditxt’s second deal announced in a month. The company moved into the women’s health market with the $100 million planned acquisition of Evofem Biosciences Inc., maker of the contraceptive gel Phexxi, which it reported on Dec. 12.

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Analysis and data insight

Clinical trial updates up 5% in 2023 despite year-end slowdown
Clinical
For full year 2023, clinical trial updates saw a 5.23% increase compared to the corresponding period last year. In December, BioWorld tracked 281 phase I-III clinical trial updates, a decline from the 354 noted in November and 416 reported in October.
Biopharma IPO class of 2023 ends the year with performance up 7%
Financings
Despite a barely open IPO window in 2023, 27 biopharma firms went public in the year. It’s the lowest number since 2012 recorded 11 IPOs in the sector. However, encouraging news is that, overall, the U.S. IPO class of 2023 is up an average of 7.48%.
2023 gainers and losers: Positive trial outcomes and M&As propel biopharma stocks to strong finish
Biopharma stocks saw a resurgence in 2023, with the BioWorld Stock Index finishing the year with a gain of 33.59%, in contrast to the 32.75% decline in 2022. An analysis of 516 stocks from the index showed that 193 companies (37%) concluded the year on a positive note, while shares of 322 companies...
2023 ends year with 191 US FDA approvals, including 55 NMEs
Regulatory
The U.S. FDA approved 21 drugs in December, bringing the total for the year to 191, a 26% increase to the 151 U.S. approvals in 2022. New molecular entities (NMEs) fell from six in November to three in December, bringing the annual number of NMEs approved to 55.

Financings

Aravax secures $22M to close series B for peanut allergy therapy
Immune
After raising $20 million in a series B round in December 2022, Aravax Pty Ltd. has raised another $22 million to close out its series B round with $42 million that expands the syndicate with new investors Novartis Venture Fund, Breakthrough Victoria, Uniseed, Unisuper, and Agati Capital. The funds...

Science

Ultrarare transmitted Alzheimer’s cases give clues to common forms
Neurology/psychiatric
Investigators have identified five cases of so-called iatrogenic Alzheimer’s disease (AD), that is, AD that was acquired as a result of undergoing medical procedures. A team led by University College London scientists reported their findings online in Nature Medicine on Jan. 29, 2024.

From viral peptides, small-molecule drugs far beyond infection
Drug design, drug delivery and technologies
Using interactions between viral peptides and human proteins as a starting point, researchers from Enyo Pharma Inc., the University of Lyon and other institutions were able to bootstrap themselves to a mitochondria-targeting small molecule that showed activity in a mouse model of nonalcoholic...
Proteomic signature can identify long COVID
Proteomics
Researchers at ETH Zurich have identified a proteomic signature that could recognize long COVID six months after acute infection. Biologically, the signature indicated that the complement system remained active in patients with long COVID six months after infection. Translationally, it could lead...

Newco news

Axelia Oncology takes its TLR2/6 agonist into the clinic to harness the innate immune system
Cancer
Axelia Oncology Pty Ltd. was spun out of Ena Respiratory Pty. Ltd., which developed a series of synthetic Toll-like receptor (TLR) 2/6 agonists for nasal delivery to treat respiratory infections, including influenza and SARS-CoV-2. Although the pegylated TLR 2/6 agonist, INNA-051, was initially...
Actimed continues raising funds to progress improved cancer cachexia asset
Cancer
With a sizeable series B financing well underway, Actimed Therapeutics Ltd. is preparing to advance its compound, S-pindolol benzoate (ACM-001), into a phase IIb/III trial to treat cachexia secondary to colorectal cancer, having also recently completed a £4.75 million (US$5 million) series A...
Perpetual aims to be peptide powerhouse
Artificial intelligence
The field of peptides is exploding, Perpetual Medicines Corp. co-founder, chairman and CEO Kerry L. Blanchard recently told BioWorld, “with a projected growth rate far surpassing large and small molecules, and gene therapies. The area is underinvested, too, so this is a good opportunity to focus on...
Another go at NGF as Levicept targets chronic pain due to osteoarthritis
Inflammatory
Just over a year since the last anti-nerve growth factor (NGF) antibody program was axed, the target is poised for a revival, with Levicept Ltd. announcing it has completed recruitment to a phase II trial of LEVI-04 in the control of chronic pain caused by osteoarthritis.
Shinobi Therapeutics launches with $51M series A to advance iPS-T cell therapy platform
Financings
Japan-California startup Shinobi Therapeutics Inc. has emerged from stealth mode with a $51 million series A round to advance its first off-the-shelf induced pluripotent stem cell (iPS)-T cell therapy against glypigan-3 (GPC3)-positive cancers toward the clinic.

U.S.

J&J, Merck CEOs capitulate on HELP invite
No subpoena will be needed to force the CEOs of Johnson & Johnson (J&J) and Merck & Co. Inc. to appear before the U.S. Senate Health, Education, Labor and Pensions (HELP) Committee, as apparently the threat was enough to get the CEOs to agree to testify at a committee hearing on U.S. drug prices.

Europe

Venture investment in UK biotech reaches £1.25B in 2023 despite VC cash crunch
Financings
In the face of the short supply of venture capital, the U.K. biotech sector weathered the storm in 2023, raising a total of £1.25 billion (US$1.6 billion) across 69 deals. That was 6% down on 2022, but has to be seen against the backdrop of U.K. VC investment across all sectors falling by a massive...

EMA grants 77 approvals in 2023, outpaces FDA in biosimilars
Regulatory
The EMA has once again come in behind the U.S. FDA, granting market access to 77 new products in 2023, fewer than half the 157 approvals the FDA processed in the 11 months from January through December 2023.
EMA reviewing CAR T safety over malignancy risks
Regulatory
Six weeks after the U.S. FDA issued an alert, the EMA is following suit and starting a review of the safety of six approved CAR T-cell cancer therapies, following 23 reports of patients developing secondary cancers. The EMA said such malignancies were considered “an important potential risk” at the...

Clinical

Sarepta’s next-gen exon-skipper positive in phase II; still needs to find its place in DMD
Neurology/psychiatric
Sarepta Therapeutics Inc.’s next-generation peptide-conjugated PMO therapy, SRP-5051 (vesleteplirsen), looks set to stake its claim in the Duchenne muscular dystrophy (DMD) space, as phase II data unveiled dystrophin expression that outperforms...
Corbus rockets on early clinical data for next-gen Nectin-4 ADC candidate
Conferences
Shares of Corbus Pharmaceuticals Inc. (NASDAQ:CRBP) doubled on opening Jan. 26 and ended the day up a whopping 249% as investors got a look at data from a first-in-human study testing next-generation Nectin-4-targeted antibody-drug conjugate (ADC)...
Immaculate inception? Vera’s Origin phase IIb OLE in IgAN augurs well for ongoing phase III
Genitourinary/sexual function
Wall Street got what it wanted from Vera Therapeutics Inc. with atacicept in IgA nephropathy (IgAN) and rewarded shares of the Brisbane, Calif.-based firm (NASDAQ:VERA), pushing them to close Jan. 25 at $25.31, up $8.29, or 49%.
Hear here: Lilly’s Akouos gene therapy restores hearing loss in an 11-year-old
Ocular
The hearing has returned for the first person who has received gene therapy for treating genetic hearing loss in the U.S. Initial results from Akouos Inc.’s phase I/II study showed that within 30 days of receiving AK-OTOF-101, pharmacologic...
Buntanetap data gap uncapped in Parkinson’s; Annovis to report phase III ‘very soon’
Neurology/psychiatric
About two months after reporting that enrollment of its phase II/III study with buntanetap in Alzheimer’s disease had completed enrollment, Annovis Bio Inc. disappointed Wall Street by saying that findings from the phase III in Parkinson’s disease...
Sciwind moves to once-weekly dosing after oral GLP-1 delivers strong phase I obesity data
Diabetes
Sciwind Biosciences Co. Ltd.’s oral ecnoglutide (XW-004) produced strong weight loss results after short-term treatments, up to a 6.8% body weight decrease in obese and overweight participants after once-daily dosing for six weeks, according to...

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