Top-line success for Biocryst Pharmaceuticals Inc. in a phase IIa proof-of-concept trial called OPuS-1 (Oral ProphylaxiS-1) with oral kallikrein inhibitor BCX4161 against hereditary angioedema (HAE) brought competitor Dyax Corp. along for the ride, as backers of both companies guessed about what's in store for the busy therapeutic space.
What analysts called "upside potential" for PTC Therapeutics Inc.'s ataluren in Europe came to pass with a recommendation for conditional approval of the candidate, to be marketed as Translarna for nonsense mutation Duchenne muscular dystrophy (DMD) in ambulatory patients 5 years and older.
A skirmish is brewing over the apparently botched isomeric structure described in the patent for Oncoceutics Pharmaceuticals Inc.'s cancer drug ONC201. It's too early to tell whether the dispute could hamstring the early stage candidate or for how long, but the bigger question involves what the fight may mean for the patients who need effective new drugs – in this case and others like it that could arise.
More than half the gross proceeds of Chimerix Inc.'s $103.8 million from a public offering will go toward advancing the oral nucleotide analogue lipid-conjugate drug brincidofovir, the company said in its 424B5 filing with the SEC, pricing 7.3 million shares at $14.22 each in a deal set to close next week.
Ophthotech Corp.'s potential $1 billion-plus, ex-U.S. deal with Novartis AG for Fovista, the antiplatelet-derived growth factor (PDGF) agent capable of enhancing the efficacy of anti-VEGF therapies in wet age-related macular degeneration (AMD), added juice to a growing $5 billion market – one about to take the next step into co-formulation.
The implausibility of comparing data with would-be competitors from separate trials did not stop industry wizards from following their 'Inpulsis' and guessing anyway, with the upshot that overall chances seemed to ASCEND for Intermune Inc.'s Esbriet (pirfenidone) in idiopathic pulmonary fibrosis (IPF), over Boehringer Ingelheim GmbH's phase III outcomes with the tyrosine kinase inhibitor nintedanib.
Still signing up patients for its phase III trial with Fovista, the antiplatelet-derived growth factor agent that would be used with anti-VEGF therapy in wet age-related macular degeneration (AMD), Ophthotech Corp. pulled down a $200 million up-front ex-U.S. deal with Novartis AG that could be worth more than $1 billion, not including royalties, which are also part of the deal.
Kamada Ltd. execs said they hope a good secondary endpoint showing from the phase II/III trial in Europe and Canada with its inhaled therapy for alpha-1 antitrypsin (AAT) deficiency – when combined with results from an ongoing phase II study in the U.S. – will impress European regulators enough to grant approval.
With eagerly awaited abstracts unleashed on the world from the American Society of Clinical Oncology (ASCO) annual meeting, pundits and investors were ransacking the batch for clues to success. ASCO unveiled no fewer than 5,000 studies from which results were to be offered at the cancer meeting, which starts in about two weeks.
The already-bright hope for Bristol-Myers Squibb Co.'s (BMS) PD-1 checkpoint inhibitor nivolumab in non-small-cell lung cancer (NSCLC) was bolstered by a deal to test the compound with Celldex Therapeutics Inc.'s CD27-targeting varlilumab, on a day that saw another oncology deal in the same space between Incyte Corp. and Medimmune, part of London-based Astrazeneca plc.
