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BioWorld - Sunday, March 26, 2023
Breaking News: Pharming’s PI3K-delta inhibitor, Joenja, wins first FDA nod in APDSBreaking News: A special invitation for new customers – 40% discount on any BioWorld subscriptionBreaking News: A special invitation for new customers – 40% discount on any BioWorld subscription
  • FDA Approved stamp with pills

    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

  • In a phase III, Dupixent breathes life into COPD

  • Can’t resist? Beigene, Nurix ploys in BTK may dodge blood cancer problems

  • Today's news in brief

  • 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.

BioWorld Insider Podcast

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

  • Stock prices with loss/gain arrows

    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...
  • Pills spilling out of prescription bottle

    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...
  • Money-financing

    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...
  • Gold wireframe handshake

    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.
More in Analysis and data insight

Today's news in brief

  • Appointments and advancements for March 24, 2023

  • Financings for March 24, 2023

  • In the clinic for March 24, 2023

  • Other news to note for March 24, 2023

  • Regulatory actions for March 24, 2023

Deals and M&A

  • Biohaven acquires early stage TYK2/JAK1 inhibitor from Highlightll in $970M deal

  • Royalty Pharma buys Puretech royalty in Karuna’s Karxt in potential $500M deal

  • Dewpoint and Novo partner on diabetes drugs targeting biomolecular condensates

  • Bio-Europe Spring 2023: Despite funding woes, biotechs weather the storm with deals

  • Livzon licenses GERD drug from Onconic in $127.5M deal

  • In war against T1D onset, Tzield-bearer Provention’s comrade Sanofi plans $2.9B takeover

  • Pfizer to buy Seagen for $43B

  • Modex codex may read out new story in EBV with Merck vaccine pact worth $922M-plus

  • CFIUS finally clears merger with F-star and Invox

  • Affibody in $637M respiratory disease deal with Chiesi

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.
  • $123M: Series B round ignites Flare Therapeutics program in urothelial cancer

    Cancer
  • AI drug discovery startup Protai raises $12M in seed funding

    Private
  • UK’s MHRA adds £10M in funding to speed up drug approvals

    Europe
  • A $4 million handshake with investor helps propel First Wave Biopharma’s CF offering

    Newco news
More in Financings
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Science

  • Concept art for Mitochondrial DNA.

    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...
  • ESMO TAT: Tumor therapies find new ways into the brain, by finesse or force

    Conferences
  • Biocom 2023: There’s science going on 250 miles above your head

    Conferences
  • HIV cure, a less uncertain journey

    Conferences
  • Path to a broadly effective HIV vaccine is coming into focus

    Conferences
More in Science

Newco news

  • Elderly woman holding illustration of brain with missing puzzle piece

    ‘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...
  • White Euro symbol on blue background

    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...
  • Targets with arrows

    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...
  • Abraham Ceesay, CEO, Rapport

    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,...
  • DNA NGS genome sequencing

    ‘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...
More in Newco news

Regulatory

  • China greenlights CSPC’s mRNA COVID-19 vaccine for emergency use

  • Aurion victorious with Vyznova cell therapy approval in Japan

  • Almost, but not quite: Adcom is mixed on Biogen’s ALS drug

  • US FDA approves Incyte’s PD-1 inhibitor Zynyz for Merkel cell carcinoma

  • Cidara prevails with US FDA approval of Rezzayo in candidemia, IC; partner Melinta to launch this summer

  • Not just Moderna on the HELP hot seat

  • US FTC: Apokyn suit has broad implications

  • Implementation of EU diagnostics reg forcing drug trial delays

  • US health spending less than expected

  • Weight-management training sponsorship lands Novo Nordisk in hot water

U.S.

  • DNA in drug capsules

    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).
  • Adcom gives thumbs up to Paxlovid, counters rebound myth

    Regulatory
  • What’s next for Paxlovid

    Regulatory
  • US RX pricing controls begin to kick in

    Regulatory
  • Leqembi that, CMS

    Regulatory
More in U.S.
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Europe

  • DNA illustration

    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...
  • UK pharma criticizes looming rise in revenue clawbacks

    Regulatory
  • UK begins review of what’s needed to revive clinical trial scene

    Regulatory
  • Debate heating up over EU voucher proposal for tackling AMR

    Regulatory
  • Long-term vision for UK’s drug manufacturing aims to reverse declining investment

More in Europe

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...
More in Clinical

BioWorld Insider Podcast

One-on-one with medical innovators

podcast microphone, sound waves on purple backgroundBreakthrough 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.


Recent episodes:

  • Cambrian carves out a new niche as it works to keep people from getting sick
  • Rethinking obesity: Fitness may be more directly linked to health than weight
  • Looking ahead to 2023: CEOs contemplate the new normal
  • Psychedelic evolution: Mindset Pharma looks to change mental health treatment
  • $1B+ biopharma deals keep values afloat, even amid muted volume
  • Extending the human lifespan
  • New therapies vie to change fatal course of amyotrophic lateral sclerosis
  • Biopharma’s correction? 2022 1Q investments are both up and down
  • Who advises the CDC on big COVID decisions and how’s that working out?
  • Long COVID: Potentially the next public health crisis

View all

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