Holding back its wet age-related macular degeneration (AMD) program from the potential $1 billion deal with Biogen Inc. will let Applied Genetic Technologies Corp. (AGTC) reap more value in a collaboration later, as the firm deploys "a better understanding of vector design [that] has come about in the last five to seven years," which allows for "really being able to target the capsid, the promoter, the physical delivery method," CEO Sue Washer told BioWorld Today.
Cerespir Inc. submitted a special protocol assessment (SPA) plan for the phase III trial with CSP-1103 to treat mild cognitive impairment (MCI) Alzheimer's disease (AD) – "the right drug at the right time," CEO Daniel Chain told BioWorld Today.
With a progressive strategy that's "building an ecosystem," Biondvax Pharmaceuticals Ltd. offered upbeat preliminary results from the BVX-006 phase II trial gauging the worth of M-001 in the Ness Ziona, Israel-based firm's quest for a universal influenza vaccine, and analyst Ashok Kumar at Aegis Capital Corp. tagged what could be alluring prospects for would-be partners or buyers of the firm.
As a paper last month in Nature predicted a "bumpy road" ahead for RNA polymerase II research, pointing to an "additional level of complexity" that has turned up in the work, Pharmamar SA – bolstered by impressive phase II results – forged ahead with the phase III experiment testing PM1183 (lurbinectedin) in patients with platinum-resistant ovarian cancer (PROC).
News of Celladon Corp.'s possible liquidation after the CUPID2 phase IIb failure in April of gene therapy candidate Mydicar for heart failure (HF) may have sent shivers through the gene therapy sector, but progress ought to provide a heartening counterbalance as researchers continue to refine their methods.
With "fivefold increase in half-life" and "no safety complications whatsoever," CSL Behring's long-acting fusion protein that links recombinant coagulation factor IX with recombinant albumin (rIX-FP) "is going to benefit patients in an extraordinary way," Debra Kennedy, head of coagulant and oncology therapeutics, told BioWorld Today.
More-careful and precise testing in a focused treatment group together with "about five times as many patients" as in the phase II trial puts muscle on data from Celator Pharmaceuticals Inc.'s phase III experiment in acute myeloid leukemia (AML) with CPX-351, a liposomal form of cytarabine and daunorubicin, CEO Scott Jackson told BioWorld Today.
Biomarin Pharmaceutical Inc. is considering the design to be sought for the pivotal phase III trial with BMN 111 (vosoritide), an analog of C-type natriuretic peptide (CNP), in children with achondroplasia, thanks to strong results with the highest of three doses – 15 µg/kg/daily – in a phase II experiment that enrolled 26 children with a mean age of 7.8 years.
While Cormedix Inc. fights for patent rights in Germany and shops for a partner that could help pay for trials with the antimicrobial catheter-lock solution Neutrolin, guidance from U.S. gatekeepers regarding the second pivotal, phase III trial with the product spiced shares of the Bedminster, N.J.-based firm (AMEX:CRMD), which closed Wednesday at $5.09, up 33 cents, having traded as high as $5.75.
