Jazz Pharmaceuticals plc is buying Chimerix Inc. for $8.55 a share, bringing the deal in at about $935 million. Jazz expands its cancer pipeline with the new acquisition’s lead candidate, dordaviprone, a small molecule for treating a rare glioma often found in children and young adults. The U.S. FDA has set an Aug. 18 PDUFA date for the NDA. So far, there are no FDA-approved therapies specifically for treating H3 K27M-mutant diffuse glioma. Chimerix’s stock (NASDAQ:CMRX) was getting a solid bump from investors at midday, as shares were trading at $8.44 each, 70% higher than they were at the opening bell.

Parkinson’s phase IIb fails, Irlab shares plunge

Irlab Therapeutics AB rolled out top-line results from the phase IIb Parkinson’s disease (PD) study with pirepemat called React-PD. The results show that treatment with the drug, designed to function as a 5-HT 7 receptor antagonist and alpha adrenoceptor antagonist, at 600 mg daily reduced the fall rate by 42%, but the effect fell short of statistical significance vs. placebo. Irlab is analyzing the data further to determine next steps. Shares of the Gothenburg, Sweden-based firm (STO:IRLAB-A) were trading at SEK5.62 (US54 cents), down SEK7.53, or 57%.

Persica’s PP-353 wins in back pain, advances to phase III

Persica Pharmaceuticals Ltd. has delivered positive 12-month follow-up data from its phase Ib study of PP-353, an injectable antibiotic for treating chronic lower back pain, and is now looking for a partner and further financing to take the product into phase III development. There was a statistically significant response in treated patients, with a 3.4-point (50%) reduction in pain from baseline, compared to a 1.4-point (30%) reduction across both the active and placebo group, at 12 months. The PP-353 group reported reductions in pain from baseline at three months and onwards, and was continuing to improve at 12 months, while patients in the placebo group, who received a sham injection, did not achieve a clinically meaningful change.

Protagonist’s rusfertide hits phase III goals in rare leukemia

Rusfertide could become a blockbuster therapy for polycythemia vera, H.C. Wainwright analyst Douglas Tsao wrote March 4 after the injectable hepcidin mimetic peptide hit its primary endpoint and all four key secondary endpoints in the ongoing phase III Verify study. More than a year ago when Takeda Pharmaceutical Co. Ltd. and Protagonist Therapeutics Inc. inked their $1.7 billion license deal for rusfertide, Protagonist’s hepcidin asset was “overlooked by investors,” Tsao said, primarily by the shade thrown from negative mouse model clinical trials in 2021, and the consequent rescinding of breakthrough status by the U.S. FDA in April 2022. Despite the fallout, Newark, Calif.-based Protagonist beat back with the strength of phase II Revive data presented at the American Society of Hematology meeting in December 2023, Tsao noted, which also had encouraging safety data on rusfertide. Results from the global ongoing three-part, randomized, placebo controlled Verify trial now tip the scale to Protagonist’s favor.

UK firm Trimtech emerges with $31M seed round for TRIM21 bispecifics

Trimtech Therapeutics closed a £25 million (US$31 million) oversubscribed seed funding round to advance its targeted protein degradation (TPD) treatments for neurodegenerative and inflammatory diseases. With the funds, the Cambridge, U.K.-based company will further develop its pipeline of central nervous system penetrant therapeutics, which are based on aggregate-selective degrader molecules called Trimtacs, for severe disorders within the two therapeutic areas. As part of its efforts, Trimtech leverages the innate properties of the E3 ubiquitin ligase tripartite motif containing 21 (TRIM21) through its Trimtacs to selectively and potently degrade protein aggregates related to several diseases that are not addressed by other TPD approaches. The approach also preserves functional monomers and can address large patient populations that have few treatment options.

Bhattacharya shares his vision for US NIH

Jay Bhattacharya will have his work cut out for him if he wins confirmation as the next director of the U.S. NIH. Besides getting NIH committees back on track to evaluate grant applications and calming the fears of researchers and other staff who have seen about 1,200 colleagues cut from their ranks in recent weeks, Bhattacharya will face the task of rebuilding public trust in the NIH itself. “You’ve got a hard job in front of you,” Sen. Tommy Tuberville, R-Ala., told Bhattacharya during today’s Senate Health, Education, Labor and Pensions Committee confirmation hearing. In his opening remarks, Bhattacharya laid out his five goals for the agency should he be confirmed, including the creation of a culture that allows for dissent and scientific discourse. Rather than forcing conformity, science should be a force for freedom, he said.

Sofinnova raises €1.2B to invest in med tech, biotech, digital health

Sofinnova Partners raised a whopping €1.2 billion (US$1.26 billion) over the past year to invest in life sciences companies ranging from incubation to later-stage growth, and spanning biotech, med tech, industrial biotech and digital medicine. The funds raised, which include €1 billion secured in the last quarter of 2024, is a boon for the sector in Europe which has been struggling for investment. “We've never had so much money coming into our funds at Sofinnova [...] and we’re not finished,” Antoine Papiernik, chairman and managing partner of Sofinnova Partners, told BioWorld.

Aspargo Companion can spray most meds, alert to missed doses

In an advance that could significantly lighten the load for caretakers in the “sandwich generation” and reduce loneliness in elderly patients, Aspargo Labs Inc. developed a metered delivery device that optimizes absorption of pharmaceuticals and reminds users to take their medications. Its Companion delivery system offers peace of mind with biometric security and mobile connectivity that alerts others if they miss doses, making it ideal for elderly patients who may be forgetful or children who need to take medications during school, CEO Michael Demurjian told BioWorld. “The genesis of this is my mother started to develop dementia, and it was embarrassing every time we had to sneak crushed pills into her apple sauce,” Demurjian explained. “It’s humiliating for everybody. I remember her looking at me with tears in her eyes and saying, ‘Why are you doing this? I’m not a baby.’”

Also in the news

Acelyrin, Alumis, Amneal, Attmos, Axsome, Beigene, Biogen, Bioxcel, Bristol Myers Squibb, Capricor, Celltrion, Centauri, Concentra, Corcept, Dianthus, Evotec, Garuda, Genentech, Hutchmed, Ideaya, Johnson & Johnson, KSQ, Lexicon, MBX, Myosin, Nanonewron, Nanoscope, Neurenati, Nippon Shinyaku, Orbis, Osivax, Pepgen, Regenxbio, Seres, Tenaya