HHS, FDA dis­pute spills out on­to Twit­ter

In the fi­nal months and weeks of the Trump ad­min­is­tra­tion, the De­part­ment of Health and Hu­man Ser­vices (HHS) has ad­vanced sev­er­al poli­cies to con­sol­i­date its au­thor­i­ty over the US Food and Drug Ad­min­is­tra­tion’s (FDA).

To­day, with just one day left in the Trump ad­min­is­tra­tion, the lat­est dis­pute be­tween HHS and its sub­agency spilled in­to the pub­lic.

Fol­low­ing me­dia re­ports last week that FDA Com­mis­sion­er Stephen Hahn re­fused to sign a mem­o­ran­dum of un­der­stand­ing (MoU) that would cede FDA’s over­sight of ge­net­i­cal­ly mod­i­fied an­i­mals to the US De­part­ment of Agri­cul­ture (US­DA), HHS pushed ahead with the pol­i­cy. A copy of the MoU post­ed to the US­DA web­site shows the doc­u­ment was signed by As­sis­tant Sec­re­tary for Health Brett Giroir and Sec­re­tary of Agri­cul­ture Son­ny Per­due and, telling­ly, lists a li­ai­son for US­DA but leaves the cor­re­spond­ing sec­tion for FDA’s li­ai­son blank.

In a stun­ning move, Hahn tweet­ed that FDA would not abide by the MoU, a rare pub­lic air­ing of a dis­pute be­tween top ad­min­is­tra­tion of­fi­cials that fol­lows height­ened po­lit­i­cal pres­sure on the agency to quicky au­tho­rize ther­a­peu­tics and vac­cines for COVID-19. That pres­sure has led to dis­cus­sion among health ex­perts as to whether FDA should have greater in­de­pen­dence from po­lit­i­cal in­flu­ence, some­thing Hahn him­self has said should be on the ta­ble.

In a state­ment to Fo­cus, an HHS spokesper­son said the MoU was pushed through at the di­rec­tion of the White House. “To­day’s MOU an­nounce­ment in­volv­ing HHS and US­DA re­gard­ing agri­cul­tur­al an­i­mal biotech­nol­o­gy is the re­sult of a for­mal pol­i­cy process co­or­di­nat­ed by the White House. Sec­re­tary Azar was and re­mains sup­port­ive of Com­mis­sion­er Hahn’s and FDA’s po­si­tion on the MOU, but at the di­rec­tion of the White House, the de­ci­sion was made to ex­e­cute the MOU,” the spokesper­son said.

Aaron Kessel­heim, di­rec­tor of Har­vard Med­ical School and Brigham & Women’s Hos­pi­tal’s Pro­gram on Reg­u­la­tion, Ther­a­peu­tics and Law, told Fo­cus that the MoU fits in with the oth­er “last-minute rad­i­cal pol­i­cy changes” be­ing pur­sued by HHS. “These changes are uni­form­ly bad in that they di­min­ish the FDA’s vi­tal role in pro­tect­ing the pub­lic health,” he said, adding that he hopes that “the in­com­ing ad­min­is­tra­tion of Pres­i­dent Biden re­vers­es as many of them as he can as quick­ly as he can.”

For­mer FDA Com­mis­sion­er Scott Got­tlieb al­so crit­i­cized the MoU and HHS’ at­tempt to strip FDA of its over­sight of gene-edit­ed an­i­mals, call­ing it an “un­prece­dent­ed usurp­ing of FDA pub­lic health au­thor­i­ty. FDA should reg­u­late an­i­mal biotech as part of its man­date. This pol­i­cy move threat­ens the health and well-be­ing of Amer­i­cans and should be im­me­di­ate­ly re­versed.”

Oth­er re­cent moves by HHS in­clude rein­ing in del­e­gat­ed au­thor­i­ties by re­quir­ing the sec­re­tary of HHS’ sig­na­ture on all de­part­ment rules, lift­ing reg­u­la­to­ry re­quire­ments for cer­tain med­ical de­vicesim­pos­ing “term lim­its” on di­rec­tor-lev­el staff and sun­set­ting reg­u­la­tions if they are not as­sessed and re­viewed every 10 years. HHS al­so over­ruled FDA re­gard­ing its over­sight of lab­o­ra­to­ry de­vel­oped tests last fall, as­sert­ing that the agency “will not re­quire pre­mar­ket re­view of [such tests] ab­sent no­tice-and-com­ment rule­mak­ing” and re­cent­ly award­ed a con­tract to a pri­vate con­sult­ing firm to re­view emer­gency use au­tho­riza­tion (EUA) re­quests for some LDTs.

In an un­usu­al turn of events last week, HHS and FDA, in­de­pen­dent­ly from one an­oth­er, named dif­fer­ent in­di­vid­u­als to be FDA’s top lawyer af­ter for­mer Chief Coun­sel Sta­cy Cline Amin stepped down.

Got­tlieb and Kessel­heim’s state­ments both raise a point about the dura­bil­i­ty of this and oth­er re­cent poli­cies en­act­ed by HHS. Some ac­tions, such as the MoU, could be rel­a­tive­ly straight­for­ward for the new ad­min­is­tra­tion to re­verse, where­as rules may be chal­lenged in court or over­turned via the Con­gres­sion­al Re­view Act.

So­cial im­age cred­it: Get­ty Im­ages

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Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

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#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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Jerome Durso, Intercept Pharmaceuticals CEO

In­ter­cep­t's OCA fails a PhI­II NASH tri­al, rais­ing fresh doubts about its years­long quest for an OK

Intercept Pharmaceuticals has run into another big setback in its yearslong quest to win an approval for OCA in NASH. The biotech put out word Friday morning that its Phase III REVERSE study failed the primary endpoint for the liver disease, sending its share price into a tailspin.

There was no significant improvement in fibrosis among the patients suffering from cirrhosis who were treated with obeticholic acid, with investigators hunting for a minimum 1-stage histological improvement in the disease after 18 months of therapy.

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Bo Cumbo, new Solid Bio CEO

Sol­id Bio gets a new CEO, $75M cash and drops lead drug as big in­vestors ju­ry-rig a merg­er deal

Three months after Endpoints News broke the story that gene therapy outfit AavantiBio had gutted its CMC group in a reorganization, the biotech’s CEO has helped engineer a merger with struggling penny stock player Solid Bio. And he’s going to remain in charge of the combined operation, as Solid founder Ilan Ganot steps aside.

The merger news this morning features some high-profile investors.

Perceptive Advisors, RA Capital Management and Bain Capital Life Sciences are leading a $75 million raise to add to the pool of cash Solid will have after the tie-up. That will leave Solid $215 million in cash as Bain’s Adam Koppel jumps on the board — enough to pay for ops and get through some key data milestones on their way into 2025.

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