A better-than-expected second-quarter profit at Celgene Corp. and news of a planned exit by Gilead Sciences Inc. CEO John Milligan led a parade of earnings news, carried by a drumbeat of estimate-beating results at companies including Alexion Pharmaceuticals Inc., Vertex Pharmaceuticals Inc. and Alkermes plc as demand for key products at the companies grew. Pricing pressures remained a specter, gaining at least some outward acknowledgment at Celgene, while tariff troubles dogging other sectors remained, blissfully for biopharma investors, at bay. Amgen Inc. also reported earnings following the market's close.
A successful phase IIb trial testing the power of Restorbio Inc.'s oral TORC1 inhibitor to prevent respiratory tract infection (RTIs) in high-risk elderly patients has put it on track to complete a pivotal phase III program in 2019 and a new drug application in 2020, assuming all goes well. Top-line results of the study showed that, relative to placebo, RTB-101 reduced the percentage of patients with one or more lab-confirmed RTIs by nearly a third during a 16-week window, an outcome that drove company shares (NASDAQ:TORC) 49.5 percent higher to close at $13.47.
A strong second quarter at Biogen Inc., buoyed by increasing demand for the spinal muscular atrophy drug Spinraza (nusinersen) and a better-than-expected quarterly profit, helped the company beat consensus earnings estimates and raise its 2018 financial guidance. Appreciation for completed enrollment in the company's two phase III studies of the Alzheimer's disease (AD) candidate aducanumab and interest in a newly announced acquisition of a muscle enhancement program from Alivegen Inc. also helped.
Privately held Anima Biotech Inc., a New Jersey-based small-molecule discovery platform company, has reached a multiyear deal with Eli Lilly and Co. to develop translation inhibitors for undisclosed target proteins of Lilly's choosing. Anima, formerly based in Tel Aviv, Israel, gains $30 million up front and $14 million in research funding under the terms of the agreement. It could receive up to $1.05 billion by achieving future development and commercial milestones and low to mid single-digit tiered royalties on sales of any Lilly products resulting from the collaboration.
Pliant Therapeutics Inc., a startup developing small-molecule therapies against fibrotic diseases, has raised $62 million to carry a potential new therapy for idiopathic pulmonary fibrosis (IPF) through proof of concept next year, creating momentum toward a pivotal program to start in late 2019 or early 2020. Studies of its lead candidate, a dual-selective integrin inhibitor, are also in the works in primary sclerosing cholangitis (PSC).
Shares of Zogenix Inc. (NASDAQ:ZGNX) climbed 20.7 percent to close at $55.90 on Thursday after a second pivotal study of ZX-008 (fenfluramine) showed the drug maintaining its ability to diminish seizures among young people with rare and severe epilepsy type Dravet syndrome, even when added to standard-of-care therapy. Participants achieved a 54.7 percent greater reduction in mean monthly convulsive seizures vs. placebo during the phase III trial and remained free of heart valve damage, easing safety concerns raised by fenfluramine in an earlier chapter of its use. (See BioWorld, Oct. 2, 2017.)
Tokyo-based Otsuka Pharmaceutical Co. Ltd. is taking its first step into the world of biologics with a $430 million all-cash acquisition offer for Visterra Inc., a company offering an antibody design and engineering platform that President and Director Tatsuo Higuchi said would provide it with "a powerful new drug creation engine."
New ad hoc subgroup analyses of data from the first trial testing Medicinova's MN-166 (ibudilast) in amyotrophic lateral sclerosis (ALS) have given the company "the necessary clinical data for powering assumptions for the next study" and the confidence to seek a meeting with the FDA regarding that trial's design, the company said.
Spero Therapeutics Inc. is laying the groundwork to move its lead candidate, the carbapenem SPR-994, into a phase III trial for people with complicated urinary tract infections (cUTI) around year-end, encouraged by interim phase I data suggesting the candidate can be just as safe and effective as its progenitor, Meiji Seika Pharma Co. Ltd.'s Asia-marketed Orapenem (tebipenem pivoxil). Spero's phase III trial, subject to FDA review, would be a noninferiority study pitting oral SPR-994 against I.V. Merck & Co. Inc.-marketed Invanz (ertapenem), the most commonly prescribed carbapenem for cUTI.
Dynacure SAS, a French company developing an antisense drug for a rare genetic muscular disorder, has closed a €47 million (US$55 million) financing intended to help advance its lead program, Dyn-101, into the clinic.
