Bio Roundup: ICER Eyes Cost, Azar at HHS, Gates’s ALZ Cash & More

Xconomy National — 

In our roundup this week, Alzheimer’s disease is in the news, thanks to billionaire Bill Gates and his personal pledge of $100 million toward R&D. That could equal the amount one startup hopes to raise in an IPO to develop drugs for Alzheimer’s and other brain diseases. If a treatment became available, could our health system handle it? One report says no.

The Alzheimer’s field has seen big R&D stumbles; this week, other notable trial failures from Juno Therapeutics and Acorda Therapeutics were in the spotlight. Meanwhile, one clinical success story, the gene therapy for blindness called Luxturna, received scrutiny for a hypothetical $1 million price tag. (Its owner hasn’t yet said what it might cost.)

Speaking of cost, Congressional Republicans are scrambling to pay for their proposed massive tax cuts with more attacks on Obamacare. Let’s start the roundup in the nation’s capital and radiate out from there.


—The House passed its tax reform bill, but the Senate version remains a work in progress. This week GOP senators amended it to kill the Affordable Care Act’s individual mandate as a way to save hundreds of billions of dollars and hobble the ACA. It’s unclear if the Republicans who opposed GOP efforts to repeal Obamacare will also oppose the new anti-ACA language.

—Continuing the trend of hiring pharmaceutical officials despite President Trump’s anti-industry rhetoric, The White House nominated former Eli Lilly (NYSE: LLY) executive Alex Azar as the next Health and Human Services secretary. Azar, who worked in the George W. Bush administration’s HHS, is a staunch opponent of the Affordable Care Act. He will appear before the Senate committee overseeing health matters on November 29.

—The FDA laid out more detailed policies toward regenerative medicine and stem-cell treatments this week, promising to streamline some regulations while cracking down on dubious offerings.

—The Senate committee that oversees health matters held a hearing on CRISPR gene editing technology, with senators praising advances and raising questions about safety and patient rights.


—A Rand Corporation report cast doubt on the readiness of the U.S. healthcare system if an Alzheimer’s treatment were to be approved. Too few specialists and a lack of infrastructure would also hamper efforts. The situation hasn’t changed much since Xconomy reported a similar outlook in early 2016.

—Bill Gates said he would put $100 million of his own money into Alzheimer’s disease research. Half will go into the Dementia Discovery Fund. The other half will be invested in startups, according to Reuters, but Gates did not name specific companies. Gates said he wants to encourage more basic research, methods for earlier detection, a more diverse drug pipeline, and more data sharing.

—South San Francisco, CA-based Denali Therapeutics, which has associations with Gates, filed to go public. The firm is pursuing drugs for neurodegenerative diseases, including Alzheimer’s and Parkinson’s.


—Cominbations of cancer immunotherapies have often hit snags in clinical studies, but San Francisco-based Nektar Therapeutics (NASDAQ: NKTR) reported positive early findings from a Phase 1/2 study of its drug NKTR-214 paired with Bristol-Myers Squibb’s (NYSE: BMY nivolumab (Opdivo), which elicited responses in melanoma, kidney cancer, and non-small lung cancer.

—Bayer paid $400 million in cash upfront to grab partial rights to two experimental cancer drugs from Loxo Oncology (NASDAQ: LOXO) both designed to target a specific tumor mutation, regardless of where the cancer resides. The deal is a vote of confidence in broad cancer DNA tests, which have struggled to gain traction.


—The Institute for Clinical Economic Review (ICER) said that voretigene neparvovec (Luxturna), a gene therapy for a rare blindness, might not be cost-effective if owner Philadelphia-based Spark Therapeutics (NASDAQ: ONCE) charges $1 million. Spark got a big thumbs-up from a panel of FDA advisors in October but has not disclosed its pricing strategy; an FDA approval decision is due by January 12. ICER said it would issue a revised report that month.

—Shares of Acorda Therapeutics (NASDAQ: ACOR) fell 40 percent after the company disclosed that five patients died in studies of experimental Parkinson’s disease therapy tozadenant—a critical drug in the Ardsley, NY, company rebuilding plan.

—Sangamo Biosciences (NASDAQ: SGMO) of Richmond, CA, reported the first gene editing treatment delivered directly into a patient. (Other gene therapies have worked inside patients, but they haven’t directly snipped patient DNA.) The in vivo drug, for the rare Hunter syndrome, was administered to a patient in Oakland, CA.

—Last Friday, CAR-T cell therapy developer Juno Therapeutics (NASDAQ: JUNO) discussed its investigation into the five patient deaths that halted its ROCKET leukemia study last year.

—The FDA approved Genentech’s emicizumab-kxwh (Hemlibra), an injectable drug meant to prevent bleeding episodes in a small subset of hemophilia A patients.

—Ultragenyx Pharmaceutical (NASDAQ: RARE) of Novato, CA, also won a nod for vestronidase alfa-vjbk (Mepsevii), a treatment for the ultra-rare mucopolysaccharidosis type VII. The approval comes with a priority review voucher.


—Alnylam Pharmaceuticals (NASDAQ: ALNY) pulled off a whopping $805 million stock offering as it prepared to ask the FDA for approval of patisiran for transthyretin amyloidosis. It would be the first drug based on RNA interference ever approved.

—ImmusanT, of Cambrige, MA, raised $40 million to start placebo-controlled trials of a vaccine meant to help people with celiac disease tolerate gluten.

—Waltham, MA, and Vienna-based Arsanis (NASDAQ: ASNS), an infectious disease startup tied to serial biotech entrepreneur Tillman Gerngross, closed a $40 million IPO.

—Apellis Pharmaceuticals of Louisville, KY, priced its IPO at $150 million late last week. Apellis CEO Cedric Francois was a vocal opponent of the Trump administration’s ban early this year on immigrants and travelers from seven Muslim-majority countries.

—Inozyme Pharmaceuticals of Cambridge, MA, working on a treatment for the ultra-rare … Next Page »

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