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BioWorld - Monday, December 11, 2023
Breaking News: Topic alerts now available for all BioWorld subscribersBreaking News: Topic alerts now available for all BioWorld subscribersBreaking News: Topic alerts now available for all BioWorld subscribers
  • Mast cell releasing histamine during allergic response

    ASH 2023: Phase II case Cogent enough with bezu in mastocytosis? Street skeptical, more data soon

    Wall Street’s measure of how Cogent Biosciences Inc.’s KIT D816V inhibitor bezuclastinib (often shortened to bezu) might fare in mastocytosis against U.S. FDA cleared Ayvakit (avapritinib), the tyrosine kinase inhibitor from Blueprint Medicine Corp., caused the former’s stock (NASDAQ:COGT) to tumble, closing Dec. 11 at $4.06, down $4.58, or 53%.
  • ASH 2023: Gene therapy success is ‘historic’ but small molecules still mean more drugs in more places

  • ASH 2023: CAR T therapies take on autoimmune diseases

  • ASH 2023: Phase II case Cogent enough with bezu in mastocytosis? Street skeptical, more data soon

    Wall Street’s measure of how Cogent Biosciences Inc.’s KIT D816V inhibitor bezuclastinib (often shortened to bezu) might fare in mastocytosis against U.S. FDA cleared Ayvakit (avapritinib), the tyrosine kinase inhibitor from Blueprint Medicine Corp., caused the former’s stock (NASDAQ:COGT) to tumble, closing Dec. 11 at $4.06, down $4.58, or 53%.
  • ASH 2023: Gene therapy success is ‘historic’ but small molecules still mean more drugs in more places

    Spirits were high at the 2023 annual meeting of the American Society of Hematology (ASH), buoyed by the U.S. FDA approval of the first two gene therapies for sickle cell disease (SCD) the day before the conference kicked off in San Diego. The addition of gene therapy to the therapeutic arsenal for SCD is “phenomenal,” Adetola Kassim, director of the Adult Sickle Cell Disease Program and professor of medicine at the Vanderbilt-Ingram Cancer Center, told BioWorld. Nevertheless, at a Saturday, Dec. 9, session titled, “Improving Outcomes for Individuals with Sickle Cell Disease: Are We Moving the Needle?,” which Kassim chaired, the answer remained “maybe.”
  • ASH 2023: CAR T therapies take on autoimmune diseases

    CAR T-cell therapies have worked well at curing blood malignancies, but a group out of the University Hospital of Erlangen have repurposed the technology as a treatment for autoimmune diseases. The expansion into new diseases has required cooperation between multiple departments, with CAR T experts taking the lead on treatment and potential side effects, and rheumatologists measuring the outcomes of the treatment.
  • Today's news in brief

    BioWorld briefs for Dec. 11, 2023.
  • Biomea’s dose escalation in diabetes nearly doubles efficacy

    Biomea Fusion Inc.’s diabetes treatment produced enhanced glycemic control at week 26 courtesy of its 200-mg cohort. It’s the latest advance for the company’s candidate that also has strong prospects in treating leukemia. Top-line data from the ongoing phase II Covalent-111 study of BMF-219, a covalent menin inhibitor for regenerating insulin-producing beta cells, demonstrated that about 40% of participants, four of 11 patients, in the 200-mg cohorts showed a durable reduction, 1% or more, in the amount of blood sugar attached to the type 2 diabetes (T2D) patients’ hemoglobin. The data came from participants who had received the last dose in a four-week treatment.
  • Prescient to advance PTX-100 to phase II for refractory T-cell lymphomas

    Prescient Therapeutics Pty Ltd.’s PTX-100 met primary safety endpoints and showed preliminary efficacy in a phase Ib trial in patients with relapsed and refractory T-cell lymphomas that exceeded the standard of care, and the company hopes to advance to a phase II registrational study in 2024, Prescient CEO Steven Yatomi-Clarke told BioWorld.

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Analysis and data insight

  • Dollar arrows pointing upward

    Biopharma funding hits $5.65B in November, with 18% increase YTD

    Financings
    In November, the biopharma industry raised $5.65 billion in total financings. While down from $11.6 billion tracked by BioWorld in October, overall financings are tracking ahead of last year. Biopharma funding has maintained an average of $6.1 billion per month in 2023, surpassing the $5.07 billion...
  • Brain, illustrating pain/injury

    Volatility defines neurological disease stocks in 2023

    Neurology/psychiatric
    Clinical data disappointments and third-quarte results cast a shadow on the BioWorld Neurological Diseases Index, closing November with an 18.37% year-to-date decline. This marks a slight improvement from its low point in October, down 23.79%, in contrast to the previous year’s upward trend, which...
  • Nonprofit stamp

    Biopharma grants see 75% growth YOY, while nonprofit deals decline

    Grant
    Biopharma grants have sustained the upward trajectory noted in August, ending October up nearly 75% compared to the corresponding period last year. In contrast, nonprofit deal value has continued its year-over-year decline, marked by a more than 90% decrease in value.
  • 2024 clock on frozen lake

    The music is slowing: Experts predict ‘biotech winter’ nearing end, cash runway still key

    Financings
    For biopharma, the 2023 post-pandemic reality check has been harsh, replete with sagging stock prices, bankruptcy filings and restructurings, as well as IPOs at their lowest levels in a decade. The good news, according to a handful of industry experts, is that it could be coming to an end, possibly...
More in Analysis and data insight

Today's news in brief

  • Appointments and advancements for Dec. 11, 2023

  • Financings for Dec. 11, 2023

  • In the clinic for Dec. 11, 2023

  • Other news to note for Dec. 11, 2023

  • Regulatory actions for Dec. 11, 2023

Deals and M&A

  • Cerevel casts spell, charms Abbvie to $8.7B takeover

  • Celltrion, Cyron ink $882M deal for polyspecific antibodies

  • Samsung Bioepis, Intocell partner for ADC drug development

  • Abbisko out-licenses pimicotinib to Merck KGaA in $605M deal

  • Tang Capital’s Concentra offers $465M cash buyout of Lianbio

  • ‘Dualing’ obesity drugs: Roche’s $2.7B buyout of Carmot driven by Mounjaro twin mechanism

  • Prism scores up to $660M from Lilly in drug discovery deal

  • To engineer Elahere: Abbvie sees bright future in $10B Immunogen takeover

  • Phenomic signs $509M deal with Boehringer for cancer targets

  • Avidity, BMS sign $2.3B cardiovascular deal for new RNA class

Financings

  • NIH-NCI chromosomes telomeres

    Tessellate Bio draws $8.4M in seed to try alternative synthetic lethality approach

    Newco news
    With an initial €8 million (US$8.4 million) in seed funding in the bank, Tessellate Bio has emerged from stealth to tackle cancers that rely on the less well explored synthetic lethality mechanism of alternative lengthening of telomeres.
  • Graphite’s nula-cel perseveres as Kamau emerges from stealth

    Conferences
  • Pharvaris turns next page in oral HAE therapy with phase II Chapter-1 readout

    Clinical
  • Mesoblast raising AU$97M for registration trials in GVHD, chronic back pain

    Immune
  • CRUK warns UK government of shortfall in funding for cancer research

    Cancer
More in Financings
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Science

  • Coronavirus, mRNA and syringe

    Off-target immune response from modified mRNA impacts future development

    Vaccines
    The Nobel Prize-winning modification that prevents the innate immune system from recognizing injected mRNA as foreign and blocking transcription of the protein it encodes has been found on some occasions to cause ribosomal frameshifting.
  • SfN 2023: Lessons from Huntington’s successes, and failures

    Conferences
    The gene for Huntington’s disease “was cloned in 1993, and everyone thought there was going to be a treatment right around the corner,” Sarah Tabrizi told the audience at the 2023 Annual Meeting of the Society for Neuroscience. Then, “it took 25 years for the first trial targeting the Huntington...
  • 1st chimeric monkey born with large embryonic stem cell contribution

    Drug design, drug delivery and technologies
    Investigators at the Chinese Academy of Sciences have generated a chimeric monkey by injecting an embryonic stem cell into the morula, which is an extremely early embryo consisting of 16 to 32 cells. The animal survived for only 10 days, and it is not the first live birth of a chimeric primate. But...
  • Study identifies nearly 100 abdominal aortic aneurysm risk genes

    GWAS
  • In HIV, gene therapy could be alternative path to functional cure

    Conferences
  • ESMO 2023: Latest advances in gynecological cancer

    Clinical
  • ESMO 2023: New approaches to old targets, including HER2 and p53

    Conferences
More in Science

Newco news

  • Digital lungs illustration

    Alveogene secures seed, advances AVG-001 for rare respiratory disease

    Respiratory
    Arriving on the gene therapy scene with an undisclosed seed funding sum, Alveogene is tackling respiratory diseases with high unmet need via a next-generation lentiviral delivery platform to advance into the clinic a candidate for rare inherited disorder alpha-1 antitrypsin deficiency.
  • Cells and DNA helix

    ‘A trillion in each of us’: Flagship-backed Quotient takes on somatic genomics

    Financings
    Launching a company based on knowledge that “the fundamental principle that most people hold to be true is off by a trillion” is a rare opportunity, said Jake Rubens, co-founder and president of Quotient Therapeutics Inc., a company that emerged from stealth this week, backed by two years of...
  • T cells attacking cancer cells

    T-Therapeutics raises $59M in series A for T-cell receptors

    Cancer
    Newco T-Therapeutics Ltd. has raised £48 million (US$59 million) in a series A to advance development of T-cell receptors generated by its transgenic mouse platform for the treatment of solid tumors, autoimmune diseases and infections.
  • Illustration of cell receptors, antibodies

    Etcembly aims at cancer with AI-driven, rapid TCR engineering technology

    Cancer
    Etcembly emerged from stealth mode in August with something it regards as seemingly impossible: A machine learning platform that has the ability to predict and engineer – at never-seen-before scale and speed – T-cell receptors (TCR) that enable bispecific T-cell engager antibodies targeting cancer...
  • 3D dollar sign

    Getting its bear-ings: Orsobio raises a $60M series A

    Financings
    Orsobio Inc., already in the clinic with three candidates, has completed its $60 million series A financing. The company, CEO Mani Subramanian told BioWorld, has taken its time to find the right programs, put them together and only raised capital when it saw the programs had legs. Even the series A...
More in Newco news

Regulatory

  • US FDA approves first CRISPR-based gene therapy Casgevy for sickle cell; Lyfgenia gets nod, too

  • ‘Oh when the government goes marching in’ a rising US threat?

  • Post-pandemic inspection load an ongoing struggle for FDA

  • FDA tells industry to get serious about UDI compliance

  • US senator: New payment model needed for gene therapies

  • US FDA approves Novartis’ factor B inhibitor for rare blood disease

  • China’s NMPA greenlights Jingxin’s dimdazenil for insomnia

  • Lilly’s Jaypirca gets another accelerated nod for lymphoma

  • Redhill shares climb eightfold on Talicia exclusivity

  • US warns of safety issues from seizure medications; UK restricts valproate

U.S.

  • FDA investigating risk with CAR T-cell therapies

    Regulatory
    Reports of T-cell malignancies, including lymphoma, have the U.S. FDA investigating the risks for patients who received certain autologous CAR T-cell immunotherapies.
  • Biden to expand HHS authorities to address US drug shortages

    Regulatory
  • Springworks wins first FDA nod for gamma-secretase inhibitor in desmoid tumors

    Regulatory
  • OIG asked to take deep dive into US vertical integration trend

    Regulatory
  • FDA's Janet Woodcock plans to retire in 2024

    Regulatory
More in U.S.
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Europe

  • DNA NGS genome sequencing

    A Google Maps for genomics: UK Biobank opens access to 15 years of genome sequencing data

    After five years and 350,000 hours of DNA sequencing, the UK Biobank has opened up access Nov. 30 to the whole genome sequences of half a million people who volunteered to give samples 15 years ago.
  • Bioxodes banks €12M series A round for intracerebral hemorrhage treatment

    Financings
    Bioxodes SA has set the stage for the phase IIa study of its novel anticoagulant in the treatment of intracerebral hemorrhage (ICH), after raising a €12 million (US$13 million) series A. Days before announcing the closure of the round, the first patient in the proof-of-concept study was treated, on...
  • UK pricing scheme an ‘important compromise’ that supports innovation

    Regulatory
    The long-running row over the U.K. voluntary scheme that controls the national drugs budget has been settled, in what the industry is describing as a “tough deal.” Under the scheme there is an annual cap on total sales of branded drugs to the National Health Service, with sales over the agreed...
  • Historic MHRA exa-cel win bagged, Crispr/Vertex await FDA nod

    Regulatory
  • Jeffries says health care M&A activity expected to rise next year

    Conferences
  • Flagship launches new offices in APAC, UK to build biotechs overseas

    Financings
  • Henlius, Intas allies again for serplulimab in Europe, India in $195M deal

    Deals and M&A
More in Europe

Clinical

  • Stock price reflects Intensity of enthusiasm for phase II breast cancer prospect

    Conferences
    Injecting a combination of cisplatin and vinblastine – engineered by way of the company’s Dfuserx platform – turned out to be just the ticket for early stage breast cancer in the phase II study called Invincible conducted by Intensity Therapeutics...
  • Three phase IIIs: BMS, Merck and Sanofi release new cancer data

    Cancer
    Three giants have produced new phase III study data for their already approved, big name therapies. Two were positive and the third was stopped for futility.
  • Arvinas, Pfizer broaden scope of ER-targeting therapy after impressive phase Ib breast cancer readout

    Conferences
    While early stage and involving a relatively small patient population, the interim phase Ib readout from the combination cohort testing estrogen receptor (ER)-targeting candidate vepdegestrant in combination with CDK4/6 inhibitor Ibrance...
  • Genentech’s inavolisib improves survival for PIK3CA-mutated breast cancer

    Cancer
    The oral drug inavolisib, when added to two other therapies, significantly improved progression-free survival in the first-line phase III treatment of advanced hormone receptor-positive, HER2-negative breast cancer in which patients have a PIK3CA...
  • Upbeat phase IIb data from Paradigm shift Neurosense focus to pivotal bid

    Neurology/psychiatric
    Neurosense Therapeutics Ltd. failed to impress Wall Street with top-line data from its phase IIb study called Paradigm with PrimeC, a fixed-dose combination of ciprofloxacin and celecoxib, in amyotrophic lateral sclerosis.
  • Replimune’s lead candidate misses two phase II endpoints

    Cancer
    Replimune Group Inc.’s lead candidate, RP-1 (vusolimogene oderparepvec), took a solid hit as it missed both primary endpoints in the Cerpass phase II study of skin cancer. Despite some better results from the study and good news from other studies...
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:

  • Six biopharma executives consider a tough 2023 and are hopeful for a better 2024
  • AI: driving drug development from effective to remarkable
  • The struggle is real: The first half of 2023 was an uphill climb
  • The biosimilars challenge to Humira revs up
  • Preventing opioid overdoses with a smart patch
  • Better times ahead for the biopharma sector? Could be, the new numbers say
  • Radiopharmaceuticals: The next big disrupter?
  • 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

View all

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