Rigel’s ‘around the clock’ effort pays off, Rezlidhia cleared early for AML
Rigel Pharmaceuticals Inc.’s ahead-of-deadline approval from the U.S. FDA of Rezlidhia (olutasidenib) twice-daily capsules for adults with relapsed/refractory (r/r) acute myeloid leukemia (AML) came as a surprise to the company, but followed encouraging interactions, “including a very positive midcycle review meeting,” said CEO Raul Rodriguez, adding that the firm will “redouble our efforts to make sure we're able to convey the information supporting this product” to the marketplace. Regulators had assigned the compound a Feb. 15, 2023, PDUFA date.

Rigel’s ‘around the clock’ effort pays off, Rezlidhia cleared early for AML
Rigel Pharmaceuticals Inc.’s ahead-of-deadline approval from the U.S. FDA of Rezlidhia (olutasidenib) twice-daily capsules for adults with relapsed/refractory (r/r) acute myeloid leukemia (AML) came as a surprise to the company, but followed encouraging interactions, “including a very positive midcycle review meeting,” said CEO Raul Rodriguez, adding that the firm will “redouble our efforts to make sure we're able to convey the information supporting this product” to the marketplace. Regulators had assigned the compound a Feb. 15, 2023, PDUFA date.
Out of SORTs: Theratechnologies puts trial enrollment on pause as cancer candidate falls short
Theratechnologies Inc. paused enrollment of a phase I trial testing TH-1902, its lead peptide-drug conjugate for sortilin-expressing cancers, after finding efficacy "not convincing enough" nor outweighing adverse events in some participants, an undisclosed number of which experienced neuropathy and eye toxicity, the company said.
Today's news in brief
BioWorld briefs for Dec. 2, 2022.
Anavex’s phase IIb/III study in AD hits its primary endpoint
Anavex Life Sciences Corp. posted positive top-line data from its phase IIb/III study of ANAVEX 2-73-AD-004 (blarcamesine) in treating mild cognitive impairment due to Alzheimer’s disease (AD) and mild AD. The oral small-molecule activator of the sigma-1 receptor (SIGMAR1) met the study’s primary endpoints and key secondary endpoint with statistically significant results.
Cancer stocks languish amid challenging markets
All gains made throughout the summer have vanished as the BioWorld Cancer Index (BCI) has hit its lowest point in 2022. Down 41.9% this year, BCI diverged in October from the path taken by both the Nasdaq Biotechnology Index (NBI) and the Dow Jones Industrial Average (DJIA), both of which are moving upward. Still, NBI is down 7.8% and DJIA is down 5.4% for the year.
US senators call for halt on gain-of-function research until oversight improves
Ironically, one of the casualties of the COVID-19 pandemic is an overdue review and revision of U.S. dual use research of concern (DURC) policies, as well as the Department of Health and Human Services’ Potential Pandemic Pathogen Care and Oversight guidance. Consequently, several senators are asking the White House to halt all ongoing and new viral gain-of-function and DURC studies in the life sciences that involve enhanced pathogens of pandemic potential.

BioWorld Insider Podcast

BioWorld staff writer Lee Landenberger talks to four CEOs, who offer their insights into what happened this year and look at the challenges and opportunities for next year.

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Analysis and data insight

Bebtelovimab out, Kineret in, as US and globe fight to end the pandemic
Coronavirus
The world is emerging from the COVID-19 pandemic, as both cases and deaths have remained consistently low in recent months, despite continuous mutation of the SARS-CoV-2 virus. Nevertheless, there is a shuffling of candidates in the arsenal as new variants bump once-effective therapies and the next...
US approvals and global regulatory activity at lowest point this year
Regulatory
As the days have grown darker throughout November, global regulatory activity and U.S. approvals have continued to drop, marking the month as the slowest of 2022. Compared with this time last year, regulatory news is down by 9% and FDA approvals are down by a quarter. On top of that, new molecular...
As clinical data drops from last year, COVID-19 reports are down 48%
Clinical
Nearing the end of 2022, the COVID-19 pandemic clearly no longer dominates the clinical activity reported by biopharma companies. Overall clinical data tracked by BioWorld is down significantly, as is the proportion focused on the SARS-Cov-2 virus. Therapeutics and vaccines targeting cancer,...
Life sciences SPACs dwindle amid new SEC rules and excise tax
Deals and M&A
As IPOs have slowed significantly in 2022, so have the debuts of special purpose acquisition companies (SPACs) targeting the life sciences industry, primarily due to two looming threats. This time a year ago, BioWorld had tracked 60 IPOs of SPACs searching for biopharma or med-tech targets and...

Financings

Venture financing: Less Zoom but still plenty of capital in correcting market
When the COVID-19 pandemic effectively shut down travel and conferences starting in the first part of 2020, the general lament was that the lack of face-to-face interaction would hamper biopharma companies’ ability to secure deals and investments. Instead, the opposite happened. Now, coming off two...

Science

Amyloid plaques recast as cause of neural network dysfunction
Neurology/Psychiatric
Researchers have identified a link between amyloid plaques and dysfunctional neuronal conduction in animal models of Alzheimer’s disease (AD). Their study, which was published in the Dec. 1, 2022, issue of Nature, suggests new ways to think about AD, as well as badly needed potential alternatives...

Innate immunity can drive radiation-induced abscopal effect
Cancer
A combination of radiation therapy and CD47 blockade induced an abscopal effect in animal studies even in animals that lacked T cells, researchers reported in the Nov. 21, 2022, online issue of Nature Cancer. The findings are “the first demonstration of T-cell-independent abscopal response,”...
Long lung COVID gives broader insights into fibrotic lung disease
Immune
An in-depth investigation of the underlying causes of pulmonary symptoms that in some cases persist for months following recovery from the acute stage of COVID-19 has found a distinctive proinflammatory signature in the plasma and airways of affected patients.

Newco news

Opna Bio recasts mental retardation protein as cancer immunotherapy target
Cancer
Swiss-American startup Opna Bio SA launched this week with a $38 million series A, a Science paper on one of its targets and a pipeline stretching from preclinical to phase II.
Fundamental discovery for halting neurodegeneration draws €10M seed round
Neurology/Psychiatric
Fundamental Pharma GmbH has raised €10 million (US$10.3 million) in a seed round to develop a new class of glutamate inhibitors, after uncovering a route to maintaining the protective effects of the neurotransmitter in the synapses while preventing neurotoxicity when it is released elsewhere.
Atriva taking host-targeted antiviral approach into basket study
Infection
Atriva Therapeutics GmbH, a small firm founded in 2015 to develop a host-targeted antiviral approach for treating respiratory viral infections, seems to have found itself in thick of it. As the U.S. CDC and other health agencies warn of an uptick in respiratory viral infections – the so-called...
Breye launched to develop oral therapy for diabetic retinopathy
Ocular
Newco Breye Therapeutics ApS is poised to repurpose an intravenously administered drug that failed in ischemia reperfusion injury as an orally available treatment for diabetic retinopathy, after closing a €4 million (US$4.1 million) seed round.
Replay launches first HSV-focused firm, Eudora, with eye on retinal disease
Ocular
“Big genomics” specialist Replay Holdings LLC has unveiled the first of four satellite genomic medicine companies it is forming to apply its high capacity herpes simplex viral (HSV) vector to next generation gene therapies. Eudora Therapeutics will specialize in inherited retinal eye diseases. It...

U.S.

Ferring’s fecal microbiota approval is a first for the FDA
Infection
The U.S. FDA has approved its first fecal microbiota treatment. Rebyota (fecal microbiota, live-jslm), from privately held Ferring Pharmaceuticals Inc., is now approved to prevent recurring Clostridioides difficile infection (CDI) in adults. The Nov. 30 approval came about two months after the...

Europe

NICE says no to five COVID-19 treatments in the UK
Regulatory
Britain’s National Institute for Health and Care Excellence (NICE), a key gatekeeper on the path to the U.K. market, is backing away from five COVID-19 treatments. No longer recommended in a draft guidance were Ronapreve (casirivimab + imdevimab) from Roche Holding AG, Xevudy (sotrovimab) from GSK...

EFPIA: Time for Europe to regain its Rx innovation
Clinical
Europe is losing its innovative edge in biopharma, especially when it comes to advanced therapy medicinal products, including tissue, gene and cell therapies used to prevent, treat and cure rare conditions and some cancers.
ECTRIMS 2022: Network lens could explain paradox in multiple sclerosis
Conferences
Circuit dysfunction is clearly recognized as a driver of neuropsychiatric disease, and some neurodegenerative diseases such as Parkinson’s disease. And at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) 2022 Congress, researchers made an argument that the same is...

Clinical

Researchers establish proof of concept for germline-targeting HIV vaccine
Infection
A diverse group of government and academic researchers, marking World AIDS Day 2022, have published details of an investigational vaccine they said safely induced broadly neutralizing antibody-precursors against HIV in nearly all participants in a...
TED turner? Veridian prospect cable-ready to take on Tepezza
Ocular
Word that big pharma firms are sniffing around Horizon Therapeutics plc as a takeover candidate caused not only that firm’s shares to jump but also provided a boost for Viridian Therapeutics Inc., which has a thyroid eye disease (TED) candidate...
More clarity on lecanemab as Eisai and Biogen present trial data
Conferences
Eisai Co. Ltd. and Biogen Inc. presented the full data set for the Clarity Alzheimer’s disease trial of lecanemab at the 2022 Clinical Trials on Alzheimer’s Disease, which was simultaneously published in the New England Journal of Medicine. The...
Inhibrx glitch in phase I foregrounds chondrosarcoma push
Cancer
Wall Street’s reaction to phase I news in chondrosarcoma from Inhibrx Inc. puzzled some onlookers and brought renewed attention to the rare disease, which is also the most common form of primary bone cancer in adults. Big pharma and smaller...
Two deaths reported ahead of Eisai-Biogen’s presentation of phase III data on Alzheimer’s drug lecanemab
Neurology/Psychiatric
The shadow of two reported patients’ deaths hovers over Eisai Co Ltd. and Biogen Inc.’s Alzheimer’s disease treatment, lecanemab, as the companies prepare to present phase III study data on the drug later today at the 15th Clinical Trials on...
Axsome strengthens with more positive Alzheimer's data
Neurology/Psychiatric
Axsome Therapeutics Inc.’s AXS-05 (dextromethorphan + bupropion) has notched another success by hitting its phase III primary endpoint in treating a notoriously difficult Alzheimer’s disease (AD) indication when compared to placebo. Newly released...

podcast microphone, sound waves on purple background Breakthrough medicines, billion-dollar deals, spectacular clinical successes and crushing failures all play a part in biopharma’s dynamic story. Developers make scientific advancements with the potential to change everything, only to face regulatory conundrums and ever-fluctuating markets. BioWorld tracks key events in the fast-moving sector every business day. Now, the BioWorld Insider podcast lets you hear directly from the movers and shakers whose collective work is changing how we all live. Join us each week for a new conversation.

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