Four companies are joining a collaborative research effort – and helping, to an undisclosed extent, with funds – in acute myeloid leukemia (AML) called the Beat AML Master trial, led by the Leukemia & Lymphoma Society (LLS) with participation by other oncology researchers.
Hung up in the U.S. and overseas, PTC Therapeutics Inc.'s appeal to the FDA regarding the effort with Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy (DMD) "does not have much of a leg to stand on" in the view of RBC Capital Markets analyst Simos Simeonidis.
Ocular Therapeutix Inc.'s potential $315 million pact with Regeneron Pharmaceuticals Inc. brought no up-front payment and only $10 million upon the exercise of an option – a carefully considered move, said Chief Financial Officer Brad Smith.
CEO Simon Tomlinson told BioWorld Today that Akriveia Therapeutics LLC's approach "exploits the differential biochemistry of the tumor micro-environment to activate an otherwise inert biologic molecule in the vicinity of the tumor," a rational-design strategy to "bioengineer the optimal tumor-targeted biologic."
In the wake of Alcobra Ltd.'s FDA-mandated clinical hold on the phase III study with metadoxine extended release in adults with ADHD, Supernus Pharmaceuticals Inc. – which chalked up positive data Tuesday in a phase IIb trial with SPN-812 – finds itself in what CEO Jack Khattar called a "horse race" with Sunovion Pharmaceuticals Inc.
While many in the biopharma world had their eyes on the European Society of Medical Oncology meeting in Copenhagen and scratched for more data – or for a sightline on the meaning of the avalanche of results that spilled out – Nicox S.A. took a back seat with its complete response letter (CRL) for the NDA related AC-170 for ocular itching in allergic conjunctivitis (AC).
Alnylam Pharmaceuticals Inc. CEO John Maraganore said the company "would like to have a complete picture here before we get into any further detail" about the 18 trial deaths that caused halting phase III development of RNA interference (RNAi) drug revusiran for hereditary transthyretin amyloidosis (ATTR) with cardiomyopathy.
As buzz grows approaching the European Society for Medical Oncology (ESMO) meeting in Copenhagen this weekend, therapeutic prospects in first-line renal cell carcinoma (RCC) have drawn particular interest, with big pharma and biotech players waiting for data rollouts.
Psoriasis trial results "tend to vary a little bit" between experiments, Dermira Inc.'s chief development officer Luis Peña told BioWorld Today, but consistency of data in two yet-to-report phase III studies with Cimzia (certolizumab pegol) is "nothing we're concerned about."
