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Bio Roundup: An Alzheimer’s Head-Scratcher, OUTBio, GSK & Gilead Shakeups  

Xconomy National — 

The devil is in the details, and key clinical trial results made that abundantly clear this week. Eisai and partner Biogen released the highly anticipated details of an Alzheimer’s disease study they had already deemed positive, after an initial failure.

In one sense, the study was the success they have claimed. A high dose of the drug appeared to have a positive impact on patients, compared to placebo. But as the investment community teased out the details that trickled out at a big Alzheimer’s disease meeting, confusion and handwringing reigned. The drug’s different doses didn’t perform in a predictable way. A complicated trial design and imbalances between different groups made it tough to draw conclusions. Add it all up and the details, as one biotech analyst wrote, “call the reliability of this result and any associated inferences into question.”

In other news, Xconomy has a profile of a Boston-based LGBTQ group gaining traction, three biopharma giants shook up their organizations, and investors kept pouring money into startups. We’ve got all the details below.

TOP STORIES

—Three weeks after declaring success in a Phase 2 Alzheimer’s disease study that had initially failed, Eisai and partner Biogen (NASDAQ: BIIB) achieved different results after continuing the trial for six more months. But the results left many unanswered questions about the experimental drug, BAN2401, and Biogen’s shares fell 11 percent.

—From an informal gathering of 11 people in 2015, OUTBio, a grassroots, biopharma LGBTQ group, has grown to around 500 members and hosts monthly events at Boston biopharma companies big and small—all without a website. Xconomy spoke with founder Ramsey Johnson and others about the group, advocating for LGBTQ issues in biopharma, and recruiting C-level executives as champions.

BIOPHARMA SHAKEUPS

—-GlaxoSmithKline (NYSE: GSK), whose R&D team is now run by Genentech veteran Hal Barron, unveiled a new strategy: focusing on genetic medicine and immunologic drugs. As part of that strategy, GSK invested $300 million in 23andMe to kickstart a four-year alliance to develop drugs based on the company’s genetic insights. Here’s more on the R&D plan from Forbes and Endpoints News.

—Roche plans to split its pharmaceuticals business into two organizations, with one based in the U.S. and the other abroad, according to the Wall Street Journal.

—Gilead Sciences (NASDAQ: GILD) president and CEO John Milligan plans to step down at the end of the year, ending a 28-year run with the Foster City, CA, biotech. After taking a break, Milligan says he will move on to “new and different opportunities.” Former CEO and current chairman John Martin plans to retire from the board as well once Gilead finds a new CEO.

—Amgen (NASDAQ: AMGN) R&D chief Sean Harper will retire from the Thousand Oaks, CA, company to “pursue opportunities in the early-stage biotech community,” ending a 16-year stint. David Reese, currently Amgen’s vice president of translational sciences and oncology, will take his place.

—The tough commercial landscape for antibiotics has led South San Francisco, CA-based Achaogen (NASDAQ: AKAO) to lay off 28 percent of its staff. The restructuring comes ahead of Achaogen’s launch of plazomicin (Zemdri), which the FDA approved last month for complicated urinary tract infections.

—Genomics pioneer Craig Venter is facing accusations that he stole trade secrets from Human Longevity, a San Diego company he founded. The San Diego Union Tribune has more details about the allegations, which emerged in a lawsuit that the company filed against the J. Craig Venter Institute.

—An appeals court rejected a bid by Acorda Therapeutics (NASDAQ: ACOR) to block generic competition for its flagship multiple sclerosis drug dalfampridine (Ampyra).

DATA DUMPS

—Celgene (NASDAQ: CELG) said a combination of its flagship blood cancer drug lenalidomide (Revlimid) and rituximab (Rituxan) was better than rituximab alone in a Phase 3 study of patients with follicular and marginal zone lymphoma who have failed previous treatment.

—resTORbio (NASDAQ: TORC) said its experimental drug for lung infections in the elderly succeeded in a mid-stage study. Shares doubled before settling in to a roughly 25 percent gain for the week as investors dissected the data. Here’s more from STAT.

—Merck (NYSE: MRK) said its immunotherapy pembrolizumab (Keytruda) beat chemotherapy in a Phase 3 test in patients with newly diagnosed head and neck cancers and whose tumors express a certain amount of the protein PD-L1. The drug is already approved for head and neck cancer patients who have failed chemotherapy.

—At the International AIDS Conference, GSK and Merck reported data on emerging rival HIV therapies, which both aim to threaten Gilead’s HIV franchise.

—Brigatinib (Alunbrig), the cancer drug Takeda acquired when it bought Ariad Pharmaceuticals in 2017, bested crizotinib (Xalkori) in newly diagnosed lung cancer patients with a specific genetic mutation.

—Acceleron Pharma (NASDAQ: XLRN) reported positive preliminary results from a mid-stage study of an experimental treatment for Charcot-Marie-Tooth disease, an inherited neurological disorder that causes muscle weakness. The treatment, ACE-083, will proceed into a longer, larger Phase 2 test.

FDA MOVES

—An FDA advisory panel rejected GSK’s experimental chronic obstructive pulmonary disease drug, mepolizumab. The FDA, which looks to its advisory panels for input, will decide by Sept. 7 whether to approve the drug.

—The agency halted testing of an experimental Duchenne muscular dystrophy gene therapy from Sarepta Therapeutics (NASDAQ: SRPT), which so far has shown promising early results. But Sarepta noted the move was due to a manufacturing issue—a contaminated sample—and not a safety problem. It doesn’t expect the program to be delayed.

—The FDA approved elagolix (Orilissa), a hormone pill from from AbbVie (NYSE: ABBV) and partner Neurocrine Biosciences (NASDAQ: NBIX) for moderate-to-severe endometriosis pain.

—Agios Pharmaceuticals (NASDAQ: AGIO) won accelerated approval for ivosidenib (Tibsovo), a treatment for patients whose acute myeloid leukemia hasn’t responded to earlier treatment, and whose tumors have a specific genetic mutation. It’s the second drug approval for Agios since 2017.

NEW STARTS & CASH GRABS

—Eli Lilly (NYSE: LLY) plans to spin off its animal health business, Elanco, supported by an IPO. But the Indianapolis pharmaceutical giant plans to keep a minority stake in the company after it joins the public markets.

—Gossamer Bio, of San Diego, raised a $230 million Series B round. The company, led by two former Receptor executives, formed in January to in-license drugs for various diseases.

—Sangamo Biosciences (NASDAQ: SGMO) acquired TxCell, a French developer of next-gen cell therapies for autoimmune diseases, for $84 million.

—Merck paid Sutro Biopharma, of South San Francisco, $60 million to co-develop a group of protein drugs for cancer and autoimmune diseases. Sutro separately raised $85.4 million in Series E funding.

—Biogen paid AliveGen $27.5 million up front to acquire two experimental muscle-boosting drugs that Biogen plans to initially test in spinal muscular atrophy and amyotrophic lateral sclerosis.

—New York-based digital medicines startup Click Therapeutics, which develops apps that doctors would prescribe to treat diseases, raised a $17 million Series A backed by Sanofi Ventures.

—Convelo Therapeutics, of Cleveland, OH, launched with plans to develop drugs that regenerate myelin, which helps nerve cells send signals and breaks down in multiple sclerosis. Derrick Rossi, a co-founder of Moderna Therapeutics, Intellia Therapeutics, and Magenta Therapeutics, is Convelo’s president and CEO.

—Tech investors and pharma giant investment arms were among the backers of Alector’s $133 million Series E, which the South San Francisco biotech will use to develop three antibody drugs for neurodegenerative disorders.

—Zavante Therapeutics, a privately held antibiotics developer based in San Diego, was acquired by Nabriva Therapeutics (NASDAQ: NBRV) in a stock deal valued at $27 million. Zavante’s lead drug, a treatment for complicated urinary tract infections, is headed for an FDA review.

—Liquidia Technologies (NASDAQ: LQDA) raised $50 million in its IPO, which the Research Triangle Park, NC, will use to support late-stage testing of its experimental pulmonary arterial hypertension drug.

LIFETIME ACHIEVEMENT

—Xconomy announced this week that the winner of our 2018 Lifetime Achievement Award is Nancy Hopkins. Now a retired biology professor from MIT, Hopkins is recognized for her career in genetics research and also for her groundbreaking work advocating for women in science.

Frank Vinluan contributed to this report.