Pharming’s PI3K-delta inhibitor, Joenja, wins first FDA nod in APDS
Pharming Group NV’s stock skyrocketed March 24 on news that the U.S. FDA approved Joenja (leniolisib) to treat activated phosphoinositide 3-kinase delta syndrome (APDS) for those ages 12 and older. The nod, which came a few days ahead of the March 29 PDUFA date, sent shares (NASDAQ:PHAR) up 33%, or $3.69, to end the day at $14.96.

Pharming’s PI3K-delta inhibitor, Joenja, wins first FDA nod in APDS
Pharming Group NV’s stock skyrocketed March 24 on news that the U.S. FDA approved Joenja (leniolisib) to treat activated phosphoinositide 3-kinase delta syndrome (APDS) for those ages 12 and older. The nod, which came a few days ahead of the March 29 PDUFA date, sent shares (NASDAQ:PHAR) up 33%, or $3.69, to end the day at $14.96.
Shiver me LIMBER: Incyte’s once-a-day Jakafi meets rough seas with CRL from FDA
Incyte Corp.’s bid for a once-daily vs. twice-daily version of the Janus kinase inhibitor Jakafi (ruxolitinib) was foiled, at least temporarily, by a complete response letter (CRL) from the U.S. FDA.
In a phase III, Dupixent breathes life into COPD
Dupixent (dupilumab) continues to expand its scope, this time potentially leading it and its developers into a new, multibillion dollar blockbuster market. Dupixent hit the primary and all key secondary endpoints in a phase III study of treating chronic obstructive pulmonary disease (COPD).
Can’t resist? Beigene, Nurix ploys in BTK may dodge blood cancer problems
Phase II data that rolled out from Merck KGaA with its Bruton's tyrosine kinase (BTK) inhibitor evobrutinib in relapsed multiple sclerosis (MS) put more eyeballs on the mechanism. It’s already well validated in oncology, but resistance has arisen there, and at least two firms – Beigene Ltd. and Nurix Therapeutics Inc. – are striving for solutions with degrader candidates.
Today's news in brief
BioWorld briefs for March 24, 2023.
Stalicla secures CRADA on strong cocaine abuse data
After Novartis AG decided not to take it forward in-house, the U.S. National Institute on Drug Abuse is to fund phase III development of mavoglurant in the treatment of cocaine abuse disorder. The agreement to back a trial involving up to 330 participants is with Stalicla SA, which in-licensed the glutamate receptor antagonist from Novartis earlier this year.
Bio-Europe Spring 2023: China’s appetite for more complex deals is growing
The increased availability of capital, greater access to talent, strong local governmental support and more focused attention on IP issues have increased the complexity of deals taking place between biotech and big pharma companies in China, according to Michelle Chan, chief business officer of Insilico Medicine Inc, who spoke in a panel discussion on the topic of Asia-Pacific partnering at this year’s Bio-Europe Spring, in Basel, Switzerland on March 21.
Partially blocking inflammatory receptor could lead to treatment for osteoarthritis
Partially blocking a receptor that helps regulate the activity of the inflammatory cytokine molecule interleukin-6 (IL-6) seems to promote tissue regeneration and block degeneration in a model of osteoarthritis. As reported in the March 22, 2023, issue of Science Translational Medicine, the receptor, called glycoprotein 130 (gp130), regulates both positive and negative inflammatory responses that can help regenerate tissue, but also cause degeneration.

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As James Peyer, the CEO of Cambrian Biopharma Inc., watched his grandfather fail every cancer treatment and pass away, he also saw the structure of his future company being born. Waiting until people became sick was the wrong way to treat disease, he found. Instead, Cambrian was created to develop anti-aging therapies, a unique business model that fits the new field of geroscience.

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Analysis and data insight

Biopharma stocks pull back after rising 17% in 2022
As broader markets struggled throughout 2022, the biopharma industry’s largest and most lucrative companies ended the year in a strong position, according to BioWorld’s Biopharmaceutical Index (BBI), which showed the 22 component stocks climbed by 17.3% throughout the year. Neither the Nasdaq...
BIO CEO 2023: Despite high prevalence of pain and addiction, clinical efforts drop, funding flat, BIO report finds
Conferences
The Biotechnology Innovation Organization (BIO) found in a new study that 77% of clinical programs focused on pain therapeutics five years ago are no longer active and that financings of companies working in the space are lackluster at best. Meanwhile, oncology companies, targeting an overall...
Biopharma financings hit $3.9B in January, down by 22% from last year
Financings
Starting out the year slow, biopharma financings are about 22% below where they were at the end of January last year, and they are well behind the first month of the previous two years as well, suggesting company executives may need to continue to prioritize costly programs as resources are...
Shy of the prior year, deals reach $206B in 2022; M&As fall behind by 36%
Deals and M&A
While it made a sturdy effort as biopharma companies opted for licensing deals over M&As in 2022, the year did not surpass 2021 in deal values, falling about 3.5% short. Lackluster M&A values dropped to their lowest levels in five years and were down by 35.6% compared with 2021.

Financings

Mbiomics raises €13M to pivot to microbiome-based therapeutic development
Series A
Mbiomics GmbH raised €13 million (US$14.2 million) in a first close of a series A round that will enable it to pivot from being a microbiome analytics firm to becoming a therapeutics developer.

Science

Fumarate accumulation moderates inflammation through mitochondrial genetic material
Inflammatory
A deficiency in fumarate metabolism could be behind a new mechanism of inflammation mediated by mitochondrial DNA and RNA. Two independent and simultaneous studies described how the accumulation of fumarate in the mitochondria released the genetic material of this organelle through vesicles,...

Somatic genome editing pricey, germline editing still risky, researchers conclude
Conferences
Somatic human genome editing has made huge strides in the past five years, but the likely extremely high prices will be unsustainable. A global commitment to affordable, equitable access is urgently needed because the costs and infrastructure needs of this form of treatment are not manageable for...
Prenatal gene-editing treatment offers curative potential for serious inherited disease
The researcher who pioneered prenatal surgery to correct neural tube defects has turned her attention to using CRISPR-edited gene therapies to correct severe monogenic diseases in utero. The availability of prenatal genetic diagnosis and advances in treating fetuses, and also in gene therapy/gene...

Newco news

‘Not a slowdown, a reversal’: Truebinding tackling Alzheimer’s via galectin-3
In Alzheimer’s, the amyloid beta hypothesis has proved most persistent in terms of drug development efforts to date, but aggregation of other pathogenic factors – phosphorylated tau (p-tau), APOE4, TREM2 and alpha-synuclein, for example – have also emerged as hallmarks of the disease. It’s that...
Teitur Trophics targeting neurodegenerative disease with €28M series A round
Neurology/Psychiatric
Newco Teitur Trophics ApS has raised €28 million (US$30.1 million) in a series A with which it will lay out a new route to targeting sortilin in the treatment of neurodegenerative diseases. The company is targeting the sortilin-related Vps10p domain containing receptor, which plays a role in...
Flagship puts $50M toward targeted tissue-homing tech startup
In an ideal world, when a patient takes a medicine, it acts only at the specific site of disease in the human body whilst sparing healthy tissues. But it almost goes without saying that with many drug regimens, side effects or complications are part of the package. Working behind the scenes to...
Rapport launches with $100M series A plus J&J precision neuroscience platform
Neurology/Psychiatric
Rapport Therapeutics Inc. launched with $100 million in series A funding and ambitious plans to bring a hitherto unprecedented level of precision to therapies for neurological disease. Although the Boston-based company is new to the world, its underlying platform has been a decade in the making,...
‘Biology no one else will have seen;’ startup Relation seeks new genetic targets
Musculoskeletal
Newco Relation Therapeutics Ltd. is showing its colors after raising $25 million in a seed round to work on integrating single cell transcriptomics, functional genomics and machine learning – and cut through previously undecipherable combinatorial space – to find and validate drug targets in the...

U.S.

Oh drat, OTAT: FDA’s turnaround on adcom for DMD prospect clips Sarepta shares
Regulatory
Sarepta Therapeutics Inc. CEO Doug Ingram said the U.S. FDA has promised to schedule “expeditiously” an advisory committee meeting on the BLA related to SRP-9001 (delandistrogene moxeparvovec) for Duchenne muscular dystrophy (DMD).

Europe

Seamless Therapeutics closes $12.5M seed round for new gene editing platform
Financings
Seamless Therapeutics GmbH raised $12.5 million in seed financing to take forward a novel gene editing technology based on reprogramming recombinase enzymes.

HSBC UK buying out UK arm of SVB, to relief of country’s biopharma, tech startups
Regulatory
The U.K. arm of Silicon Valley Bank (SVB) is to be acquired by HSBC UK Bank plc for a symbolic £1, after senior ministers and the Bank of England worked all weekend on a rescue package. The acquisition is to complete immediately, averting what the U.K. government had acknowledged would be a crisis...
ABPI proposes new UK drug pricing scheme; government negotiations up next
Regulatory
First the stick and now the carrots. The pharma industry in the U.K. on March 1 published its proposal for a new pricing scheme, under which it is offering to pay a fixed rebate of 6.88% across all eligible drug sales, an offer it said will deliver more than £1 billion (US$1.2 billion) per annum...

Clinical

89bio’s phase IIb outcome Enlivens NASH space, whets enthusiasm for phase III
Gastrointestinal
89bio Inc.’s top-line phase IIb data from the 216-subject study called Enliven testing pegozafermin in nonalcoholic steatohepatitis (NASH) pushed shares (NASDAQ:ETNB) to a closing price March 22 of $13.68, up $2.75, or 25%. The drug, due next for...
Astellas’ Claudin 18 inhibitor zolbetuximab meets endpoints in phase III Glow trial
Cancer
Astellas Pharma Inc.’s zolbetuximab, a monoclonal antibody targeting Claudin 18.2, met the primary endpoint for progression-free survival as well as secondary endpoints for overall survival in the phase III Glow trial in CLDN18.2-positive,...
Mereo’s restructuring leads to a potential approval path for alvelestat
Respiratory
Mereo Biopharma Group plc said its path to approval for alvelestat is a phase III study that would preclude an additional confirmatory trial. After meetings with the U.S. FDA and the EMA to discuss next steps, Mereo said it is designing a single,...
Gout doubts Dissolve with dual phase III outcomes from Selecta, Sobi
Inflammatory
Selecta Biosciences Inc. and Swedish Orphan Biovitrum AB unveiled top-line phase III data from the Dissolve I and II trials testing SEL-212 in adults with chronic refractory gout (CRG) – results that position the companies for a regulatory filing...
Likang cleared for China’s first clinical trial testing personalized neoantigen-targeted cancer vaccine
China
Likang Life Sciences Holdings Ltd. received approval from China’s NMPA to start a clinical trial of its candidate, LK-101 injection, for advanced solid tumors. The company claims this is the first personalized neoantigen vaccine and mRNA editing...
Karxt PANSS out again for Karuna; NDA slated for midyear
Neurology/Psychiatric
With phase III data in hand from the Emergent-3 study of Karxt (xanomeline plus trospium) in adults with schizophrenia, Karuna Therapeutics Inc. continues to target mid-2023 for an NDA submission to the U.S. FDA. The 256-subject trial met its...

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