Global Phase 3 MANEUVER study of pimicotinib in tenosynovial giant cell tumor (TGCT) met the primary endpoint, demonstrating objective response rate at week 25 of 54.0% versus 3.2% for placebo (p<0.0001) MANEUVER met all five key secondary endpoints, with statistically significant and clinically meaningful improvements in pain, stiffness, range of...
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By Raffaele Pereno – PhD, MBA VIENNA, Austria – A discussion with AATech CEO Rüdiger Jankowsky at BioEurope highlighted a decisive shift in how alpha‑1 antitrypsin (AAT) may be deployed in respiratory medicine. While most companies in the field continue to focus on treating the rare genetic disorder alpha‑1 antitrypsin...
Philadelphia, January 2, 2024 – Findings from a pioneering study in The American Journal of Pathology, published by Elsevier, reveal that administration of the neuropeptide α-melanocyte–stimulating hormone (α-MSH) promotes corneal healing and restores normal eye function to an otherwise degenerating and diseased cornea by providing protection against cell death and...
In patients with fibrotic interstitial lung diseases (ILDs) other than idiopathic pulmonary fibrosis (IPF), adding pirfenidone to treatment may attenuate disease progression, according to the results of a multicenter, double-blind, phase 2b trial (EU Clinical Trials Register Identifier: EudraCT 2014-000861-32) published in the journal Lancet Respiratory Medicine. Pirfenidone slows disease progression in...
PITTSBURGH – Researchers from the University of Pittsburgh Cancer Institute (UPCI) have identified eight genes that help predict a melanoma patient’s response to treatment. The new findings are being presented at the 45th annual meeting of the American Society of Clinical Oncology (ASCO), May 29 to June 2, in Orlando,...
COPENHAGEN, Denmark — Ascendis Pharma A/S (Nasdaq: ASND) today announced that pivotal Week 52 results from its randomized double-blind, placebo-controlled ApproaCH Trial of investigational once-weekly TransCon® CNP (navepegritide) in children with achondroplasia have been published in JAMA Pediatrics, a journal of the American Medical Association. In the publication, titled “Once-Weekly Navepegritide in Children with Achondroplasia:...
WATERTOWN, Mass. – PlateletBio, a preclinical-stage biotechnology company pioneering an entirely new platform of allogeneic cell therapies based on platelet biology, today announced that it has raised $75.5 million in Series B financing. The round includes new investors SymBiosis, K2 HealthVentures and Oxford Finance as well as participation from existing...
Every few weeks, Derek Carter gets an e-mail that brings a tear to his eye. Parents around the world have contacted the Carter family seeking more information on Batten’s Disease, a rare, fatal degenerative neurological disorder with no cure. “It makes you cry because I already know what’s going to...
In many ways, Andrew Kriebel of DuBoistown is like any other 8-year-old boy. He loves jumping on his trampoline, swinging and spending time with his family. But unlike most other children, Kriebel suffers from a rare neurological disorder known as Angelman Syndrome, which makes him prone to seizures, keeps him...
NIJMEGEN, Netherlands – Pleco Therapeutics BV, a specialty biopharmaceutical company developing novel combination chelating agents to detoxify the cancer micro-environment today announces that it has entered into a strategic partnership with Hyloris Pharmaceuticals SA (Euronext Brussels: HYL), a specialty biopharma company committed to addressing unmet medical needs through reinventing existing medications, to...