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%.
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%.
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.”
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.
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 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.
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...
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...
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.
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...
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.
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.
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...
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...
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.
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...
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.
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...
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...
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.
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 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...
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...
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 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.
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...
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...
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 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...
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.