Merck & Co. Inc.'s deal to take over Oncoethix SA for $110 million up front and $265 million in potential milestone payments raised not only the increasingly high profile of bromodomain (BET) inhibitors, but also the hopes of others in the space for similarly lucrative deals.
Potential benefit with longer therapy for elderly patients with acute myeloid leukemia (AML) kept hope alive for them as well as Cyclacel Pharmaceuticals Inc., which heard from the data safety and monitoring board (DSMB) that the phase III study known as Seamless with lead candidate sapacitabine has crossed the futility boundary and is unlikely to reach a statistically significant level of survival.
The administrative interim analysis of three-month efficacy proved disappointing in Sophiris Bio Inc.'s 12-month phase III PLUS-1 trial with PRX302 (topsalysin) for benign prostatic hyperplasia (BPH), but CEO Randall Woods advised investors to "just sit tight for right now," since the peek is "just a snapshot" done for planning purposes.
The onslaught of new genomics information and what it means for figuring out how to come up with drugs for rare subsets of patients were subjects for an all-day public workshop sponsored by the FDA, which ended with few answers but included plenty of potentially useful interchanges between experts.
Gearing up for a phase III trial with its anti-CD19 chimeric antigen receptor (CAR) T-cell therapy next year, Kite Pharma Inc. upsized its follow-on public offering by 485,000 shares, pricing about 3.4 million shares at $54 each for gross proceeds of about $188.1 million.
Top-line data from the first of four phase III trials with baricitinib, the Janus kinase (JAK) inhibitor for moderate to severe rheumatoid arthritis (RA) from Incyte Corp. and partner Eli Lilly and Co., set analysts buzzing about how once-daily "bari" might otherwise distinguish itself from the approved JAK inhibitor Xeljanz (tofacitinib) from Pfizer Inc., taken twice daily for RA.
Spark Therapeutics Inc. got the jump on what one analyst called "a defining year in gene therapy" with a deal that brought $20 million from Pfizer Inc. and includes the potential for as much as $260 million in milestone payments, as the pair strives for new therapies against hemophilia B.
With positive top-line phase IIa/b results disclosed last month and another phase II trial under way with its pain candidate, TRV130, Trevena Inc. priced a public offering for gross proceeds of about $45 million, selling about 11 million shares at $4 each in a deal expected to close Wednesday.
Aiming to cover the landscape of hyperoxaluria – overproduction of oxalate in the kidneys, which can lead not only to painful stones but also, in other forms, complications as serious as end-stage renal disease – Allena Pharmaceuticals Inc. raised $25 million in a series B financing.
Taking what CEO Adam Grossman called "a different spin on intravenous immunoglobulin [IVIG]" while "trying to provide a product that's very targeted, niche-focused, and is going to provide an advantage to a subset" of the patient population, Adma Biologics Inc. reported positive top-line phase III results in primary immune deficiency diseases (PIDD) with lead compound RI-002.
