Xconomy: Bio Roundup: Amyloid Angst, NASH News, Brammer Bagged & More

Xconomy National— A week after the failure of Biogen’s Alzheimer’s drug aducanumab, the ripple effects are still being felt—and not just by Biogen, which lost billions of dollars in market value in a flash.

We’ll kick off this week’s roundup with the aducanumab reaction, which includes plenty of thoughts about the future of the so-called “amyloid hypothesis” and what’s next for another Biogen Alzheimer’s drug that has had mixed results.

We’ll also check in on the gene therapy deal spree, the big week in national healthcare policy, and the preparations several companies are making for one of the year’s biggest medical meetings for liver disease. Let’s get to it!


—In the wake of the crushing failure of Biogen’s (NASDAQ: BIIB) Alzheimer’s drug aducanumab, various stories emerged questioning whether the “amyloid hypothesis” commonly used to explain the origins of the disease is dead, what’s next for the field, and what other drugs are in development.

—Days after aducanumab’s failure eviscerated almost a third of its market value, the Cambridge, MA, firm responded with a plan to buy back $5 billion of its shares.

—Is there a “rough patch” in the Alzheimer’s drug partnership between Biogen and Eisai? Right after aducanumab failed, the Japanese pharma announced plans to plow ahead with a Phase 3 trial of BAN2401—another amyloid-busting drug that has shown mixed results in early testing. Biogen’s response to the decision included a carefully crafted statement, STAT reports.


—The FDA approved siponimod (Mayzent), a pill to treat certain forms of multiple sclerosis. The Novartis (NYSE: NVS) drug gets the jump on a rival product from Celgene (NASDAQ: CELG), which botched its first attempt at an FDA review. However…

—Celgene said this week it has refiled its FDA application for the drug, ozanimod. The drug is a key component of Celgene’s pipeline and of the rationale that potential acquirer Bristol-Myers Squibb (NYSE: BMY) is touting for its $74 billion takeout proposal.

—Activist investor Starboard Value kept up its opposition to the deal, however. The firm sent a letter to Bristol shareholders asking them to vote against the buyout, calling it a “highly risky and likely value-destructive acquisition.”

—The Federal Trade Commission is scrutinizing the merger as well, keying in on the psoriasis treatments the two companies have.


—The Trump administration shocked Democrats and Republicans alike by reviving hostilities against the Affordable Care Act, aka Obamacare, thereby renewing a rallying cry that worked well for Democrats in the midterm elections. Over the objections of HHS Secretary Alex Azar and Attorney General William Barr, the Justice Department will no longer defend parts of the law against GOP challenges, according to Politico.

—A federal judge struck down Medicaid rules in Arkansas and Kentucky that deny coverage to “able-bodied” recipients unless they work, study, or volunteer. Kentucky’s governor has said he would appeal.

—House Democrats unveiled this week a bill to bolster Obamacare that does not include “Medicare for All,” meaning the party now has two competing visions for healthcare reform.

—Purdue Pharma agreed to pay $270 million to settle Oklahoma’s lawsuit claiming that the company’s marketing of its painkiller Oxycontin helped fuel the nationwide opioid drug crisis. Meanwhile, New York filed suit pinning responsibility on eight members of the Sackler family, which owns Purdue. The Sacklers issued a statement calling the suit “a misguided attempt to place blame where it does not belong.”


—Genfit (NASDAQ: GNFT), a French company with one of the most advanced experimental treatments for the fatty liver disease nonalcoholic steatohepatitis, raised $135 million in a U.S. IPO. Genfit stock is already traded in Europe. Its lead drug elafibranor should produce Phase 3 results later this year.

—NASH competitor NGM Biopharmaceuticals set the terms for its IPO, which could price next week and raise $100 million. STAT has more.

—Madrigal Pharmaceuticals (NASDAQ: MDGL) started a Phase 3 trial of resmetiron in NASH patients with fibrosis.

—Abstracts, or, snippets of data dropped this week ahead of the European Association of the Study of the Liver’s annual meeting. Vantage previewed the conference, which should include data updates from closely watched studies in NASH, hepatitis B, acute hepatic porphyria and more.

—One of those abstracts featured data from Viking Therapeutics (NASDAQ: VKTX)—whose drug, VK2809, works the same way as Madrigal’s resmetiron. The abstract outlined the results of Viking’s successful Phase 2 study in nonalcoholic fatty liver disease, a potential precursor to NASH. Viking could file papers later this year to begin a mid-stage NASH study.

—Bloomberg reports on a Phase 2 study of a drug from Pfizer that helped reduce patients’ liver fat. The drug might be used in a combination regimen in future NASH studies, according to the report.


—The gold rush to acquire gene therapy assets continued this week as Thermo Fisher Scientific (NYSE: TMO) paid $1.7 billion to buy Brammer Bio, a contract manufacturer of the viral “vectors” used to deliver gene therapy treatments. The news follows a string of recent gene therapy deals from Pfizer, Roche, Biogen, and others.

—AstraZeneca (NYSE: AZN) paid Daiichi Sankyo $1.35 billion for partial rights to Daiichi’s trastuzumab deruxtecan, an antibody-drug conjugate in testing for various tumors that express the HER2 protein. The drug should head for an FDA review in breast cancer later this year.

—Dutch RNA drug developer ProQR Therapeutics (NASDAQ: PRQR) spun out QR-313, a treatment it’s been developing for the rare skin disease Dystrophic epidermolysis bullosa (DEB), into a new, Berkeley, CA, company called Wings Therapeutics. The startup is backed by the non-profit EB Research Partnership and led by former executives of Lotus Tissue Repair, a DEB drug developer that Shire bought in 2011.

—Cell therapy startup Sana Biotechnology is licensing key work from the Harvard University lab of Chad Cowan, who is now Sana’s CSO. Fierce Biotechhas more details.

—Inivata raised a $52.6 million Series B to support commercialization of its liquid biopsy test for advanced lung cancer.

—Elicio Therapeutics emerged with $30 million in funding to back research on cancer vaccines that target the lymphatic system.

—Prevail Therapeutics raised a $50 million Series B to continue developing a gene therapy for Parkinson’s disease. The latest funding comes a year after the New York biotech closed a $75 million round.

—Bayer and Johnson & Johnson (NYSE: JNJ) agreed to pay $775 million to settle more than 25,000 lawsuits claiming the blockbuster blood thinner rivaroxaban (Xarelto) led to severe injuries and even deaths. Plaintiffs had said the companies failed to adequately warn physicians and patients about the safety risks linked to the drug.


—Aldeyra Therapeutics (NASDAQ: ALDX) disclosed positive results from a Phase 3 study of experimental anti-inflammation drug, reproxalap, in allergic conjunctivitis. The trial is the first of several reproxalap Phase 3 studies in eye diseases—among them dry eye and noninfectious anterior uveitis—to produce results. Aldeyra shares climbed 36 percent.

—Gilead Sciences (NASDAQ: GILD) and partner Galapagos NV (NASDAQ: GLPG) reported success in two Phase 3 studies of the experimental drug filgotinib in rheumatoid arthritis (RA). If filgotinib is ultimately approved, however, competition will be fierce: It could be the fourth so-called JAK inhibitor for RA to reach the market. A key safety study is ongoing which, if positive, could help separate filgotinib from the pack.

—Sarepta Therapeutics (NASDAQ: SRPT) reported the latest encouraging update from an early stage trial testing its experimental gene therapy for Duchenne muscular dystrophy. Sarepta is currently enrolling patients in a Phase 3 study of the treatment.

—Sarepta also disclosed that it aims to file for approval of casimersen by mid-2019. The drug, like the approved medicine eteplirsen (Exondys 51)and the experimental golodirsen—which is currently under FDA review—is meant to be a chronic therapy for a genetic subset of patients with Duchenne. If golodirsen and casimersen reach the market, Sarepta will have approved therapies for a third of patients with the disease.

—Seattle Genetics (NAASDAQ: SGEN) and partner Astellas Pharma reportedpositive results from a late-stage study testing experimental bladder cancer drug enfortumab vedontin. The companies plan to file for FDA approval later this year.

—Proteostasis Therapeutics (NASDAQ: PTI) of Boston this week released a swath of cystic fibrosis data, centered upon a three-drug cocktail that it hopes will compete with a Vertex Pharmaceuticals (NASDAQ: VRTX) program. Investors weren’t impressed and sent Proteostasis shares into a tailspin.


—Hookipa Pharma revealed plans for an IPO to fund clinical trials testing its experimental drugs for infectious disease and cancer.

—Precision BioSciences (NASDAQ: DTIL) completed an IPO that raised $126 million to support its pipeline of experimental gene editing therapies.


—Willem Scheele left Pfizer for the chief medical officer position at Cambridge, MA, biotech Imara.

—Fomer Novartis executive Sheerem Aradhye was appointed chief medical officer of Axcella Health.

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