Pfizer Inc.’s Beqvez (fidanacogene elaparvovec) won FDA approval for use in adults with hemophilia B, making it the second adeno-associated viral (AAV) vector-based gene therapy available for patients in the U.S., following the late 2022 approval of CSL Behring’s Hemgenix (etranacogene dezaparvovec).
Pfizer Inc.’s Beqvez (fidanacogene elaparvovec) won FDA approval for use in adults with hemophilia B, making it the second adeno-associated viral (AAV) vector-based gene therapy available for patients in the U.S., following the late 2022 approval of CSL Behring’s Hemgenix (etranacogene dezaparvovec).
Amidst a slew of end-of-week, positive EMA Committee for Medicinal Products for Human Use (CHMP) opinions is Takeda Pharmaceutical Co. Ltd.’s Fruzaqla (fruquintinib). The selective inhibitor of vascular endothelial growth factor receptors-1, -2 and -3 is for adults with previously treated metastatic colorectal cancer.
Space Liintech Co. Ltd., of Daejeon, South Korea, raised ₩4 billion (US$2.9 million) in a series A financing round to advance new drug research, development and production in outer space, as the private sector races to harness the orbit for pharmaceutical experiments.
The government of Colombia, after signaling its intent to do so at the end of 2023, issued a compulsory license on April 24 for access to generic versions of HIV drug dolutegravir, without the permission of patent owner Viiv Healthcare Ltd.
Gilead Sciences Inc. is aiming to capitalize on the early August 2022 buyout of privately held U.K. biotech Mirobio Ltd. with the advancement of PD-1 agonist GS-0151 into phase Ib trials for rheumatoid arthritis, a decision that Leerink analyst David Risinger hailed as positive for others at work with the intriguing mechanism. Paying $405 million for Oxford-based Mirobio, Gilead took ownership of the firm’s checkpoint agonists to treat autoimmune diseases.
In March 2024, BioWorld reported on 261 phase I-III clinical trials updates, showing a 10.6% increase from February's count of 236 and up from January’s 252. However, March’s tally is an 18.2% decline from March 2023’s 319 updates. The average monthly count of phase I-III updates in the first three months of 2024 stood at roughly 277, compared to 305 for all of 2023.
After investing $24 billion in three major acquisitions earlier this year, Bristol Myers Squibb Co. said April 25 it would cut 2,200 employees and discontinue 12 programs in an effort to save $1.5 billion and put the company on track for growth by the end of the decade. The number of jobs lost...
There was a time not that long ago when Merck & Co. Inc.’s Keytruda (pembrolizumab), with its multiple cancer indications, was seen as the heir apparent to Humira’s title of the biggest blockbuster drug. Not anymore. That title now belongs to Novo Nordisk A/S’ semaglutide, approved as Ozempic in...
The process of manufacturing autologous T-cell therapies is technically challenging when compared with other oncology drugs, making the overall cost of developing CAR T therapies significantly higher. A challenging reimbursement environment for drugs listed on China’s National Reimbursement Drug...
In the initial quarter of 2024, biopharma deal value totaled $43.99 billion, surpassing the values of two quarters last year and falling short of two others. Biopharma M&As, meanwhile, reached $50.06 billion, marking a decrease from the fourth quarter last year’s $84.03 billion but exceeding the...
In one of the biggest startups ever, Xaira Therapeutics has launched with more than $1 billion from investors. The financing, according to BioWorld records, is roughly equivalent to Roivant Sciences Inc.’s $1.1 billion raise in August 2017 and Galderma Inc.’s $1 billion private placement in June 2023.
The existence of two approved therapies, Lumakras (sotorasib, Amgen Inc.) and Karzati (adagrasib, Mirati Therapeutics Inc.), has been a triumphant success against KRAS, a protein that was once considered undruggable.
Cross-talk between macrophages and tumor cells could modulate cachexia in pancreatic cancer patients. A group of scientists from the University of Oklahoma has discovered a new pathway that promoted muscle wasting after the recruitment of this immune cell in the tumor microenvironment, activating...
Prior to this year’s Annual Meeting of the American Association for Cancer Research (AACR), it had been 14 years since metastasis had been the subject of a plenary session. So, the Tuesday session on “Evolution of the genome, microenvironment, and host through metastasis” had plenty of new insights...
Newco Outrun Therapeutics Ltd. has raised $10 million in a seed round to develop small-molecule E3 ubiquitin ligase inhibitors that prevent programmed protein degradation. The lead program targeting an E3 ligase that tags a tumor suppressor protein for destruction will now advance to preclinical...
Led by executives who helmed The Medicines Co., Metsera Inc. has launched with $290 million in financing to develop drugs for treating weight loss, obesity-related conditions and metabolic diseases. The company is stepping into high profile and lucrative glucagon-like peptide-1 territory forged by...
On the heels of a $4.6 million series A round in December 2023, cell therapy company Rxcell Inc. is planning to raise another $15 million in 2024 to take its iPSC-derived photoreceptors to the clinic for retinitis pigmentosa and other degenerative diseases of the retina.
With a $128 million series A financing, Diagonal Therapeutics Inc. launched to develop its lead program using agonist antibodies for treating, among other indications, the rare disease hereditary hemorrhagic telangiectasia. The antibodies are designed to activate a receptor complex in the TGF-β...
Addex Therapeutics has offloaded its portfolio of preclinical allosteric modulation drugs into Neurosterix, a new company that arrives on the scene with a $63 million series A round.
Acrivon Therapeutics Inc.’s $130 million financing disclosed April 9 hiked confidence in then-pending data with ACR-368 (prexasertib), the selective small-molecule inhibitor that targets checkpoint kinase 1 (CHK1) and CHK2. Undergoing tests in a potentially registrational phase II trial across...
Oncolytic virus specialist Theolytics Ltd. has raised £19 million (US$23.7 million) in its latest financing round, as it prepares to start phase I development of the lead product, THEO-260, later this year.
“The development of psychedelic medicines should adhere to the current European regulatory framework, at both the pan-European and member state level. It is imperative that developers understand and thoroughly apply the regulatory guidance and requirements that are in place.” That was the somewhat...
Essential Pharma Ltd. is paying up to £70 million (US$88.7 million) to acquire Renaissance Pharma Ltd., taking ownership of Hu14.18, a treatment for high-risk neuroblastoma. The antibody has completed a 64-patient phase II trial conducted by St Jude’s Children’s Research Hospital, in which it...
About a year after the U.S. FDA cleared Krystal Biotech Inc.’s topical gene therapy, Vyjuvek (beremagene geperpavec), as the first drug for recessive or dominant dystrophic epidermolysis bullosa, competitor Abeona Therapeutics Inc. disclosed a...
Suzhou, China-based Kintor Pharmaceutical Ltd. reported positive findings from a phase II study of GT-20029, a topical therapy for male androgenetic alopecia, on April 21 – boosting both the company’s stock and its chances of a late-stage clinical...
Barinthus Biotherapeutics plc’s immunotherapeutic against persistent, high-risk human papillomavirus (HPV) infections, VTP-200, was generally well-tolerated in a phase Ib/II study, but did not demonstrate significant signs of efficacy, pooled...
Eli Lilly and Co. is planning to file for U.S. FDA approval later this year after reporting that tirzepatide met all primary and key secondary endpoints in two phase III trials in obstructive sleep apnea (OSA). Assuming approval, tirzepatide could...
After Sage Therapeutics Inc. reported a phase II failure with oral dalzanemdor, also known as SAGE-718, in mild cognitive impairment related to Parkinson’s disease (PD), Wall Street’s eyes turned to ongoing mid-stage efforts with the same...
Neurotech International Ltd.’s cannabinoid NTI-164 met the primary and secondary endpoints in a phase II/III trial in children with autism spectrum disorder. The company also reported positive top-line data for Rett syndrome in a phase I/II trial.
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.