With pos­i­tive long-term da­ta, Eli Lil­ly's RET drug notch­es full FDA OK 2 years af­ter ac­cel­er­at­ed ap­proval

Eli Lil­ly has man­aged to con­vert the ac­cel­er­at­ed ap­proval on its RET drug to a full ap­proval af­ter a year and a half of fol­low-up.

First de­vel­oped at Loxo On­col­o­gy, Retev­mo (or selper­ca­tinib) is tar­get­ed at lo­cal­ly ad­vanced or metasta­t­ic non-small cell lung can­cer with a RET, or re­arranged dur­ing trans­fec­tion, gene fu­sion.

The FDA of­fered its ini­tial OK in 2020 based on ini­tial over­all re­sponse rate and du­ra­tion of re­sponse among 144 pa­tients. To notch the reg­u­lar ap­proval, Lil­ly sub­mit­ted da­ta from 172 more pa­tients and 18 months of ad­di­tion­al fol­low-up as­sess­ment on dura­bil­i­ty of re­sponse.

Look­ing at the fi­nal da­ta, ORR was 84% among 69 treat­ment-naïve pa­tients and 61% among the 247 who were pre­vi­ous­ly treat­ed with plat­inum-based chemother­a­py. In terms of DOR, the chemo-treat­ed pa­tients ac­tu­al­ly re­port­ed a longer DOR of 28.6 months, com­pared with 20.2 in the treat­ment-naïve group.

The drug was a key part of Lil­ly’s $8 bil­lion buy­out of Loxo — a prime ex­am­ple of its strat­e­gy of fo­cus­ing on ge­net­i­cal­ly-de­fined sub­sets of can­cer pa­tients. Blue­print Med­i­cines, part­nered with Roche, has a ri­val drug aimed at RET fu­sions on the mar­ket. So far this year, Lil­ly has record­ed al­most $87 mil­lion in Retev­mo sales, in­clud­ing $45 mil­lion in Q2, while Blue­print and Roche re­port­ed less than $15 mil­lion in sales for Gavre­to.

The con­ver­sion al­so comes amid clos­er scruti­ny from the FDA on ac­cel­er­at­ed ap­provals — es­pe­cial­ly the “dan­gling ones” that have failed their con­fir­ma­to­ry stud­ies. The agency’s ad­vi­so­ry com­mit­tee for on­col­o­gy drugs is set to meet to­day to dis­cuss two such drugs from Spec­trum and On­copep­tides.

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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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.

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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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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#AAO22 conference in Chicago (Photo credit: Associate editor Kyle LaHucik)

#AAO22: In bid for first FDA nod in ge­o­graph­ic at­ro­phy, Apel­lis claims an­oth­er first in eye dis­ease field

CHICAGO — Eight weeks before patients and industry find out if the FDA approves the first treatment for geographic atrophy, an advanced form of age-related macular degeneration, the biotech behind the drug is out with some new data on a secondary endpoint.

In what study investigator Charles Wykoff called the “first direct evidence of function preservation by slowing GA growth” in an investigational treatment, Apellis Pharmaceuticals’ drug pegcetacoplan led to less loss of retinal sensitivity versus sham  within 250 microns on either side of the GA lesion over 24 months.

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BioCryst's new website for its HAE unbranded campaign encourages patients to take charge of treatment decisions.

BioCryst launch­es aware­ness cam­paign around man­age­ment of rare vas­cu­lar dis­ease

While hereditary angioedema (HAE) is rare, treatment options for the condition have become much more common. So BioCryst Pharmaceuticals is taking a new angle in its recently launched HAE awareness campaign encouraging patients to take control of their disease management.

“Hereditary angioedema (HAE) isn’t the author of your story — you are … #cHAEngetheplan. Not the goal,” the US campaign website advises.

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EMA makes new rec­om­men­da­tions for Im­bru­vi­ca, med­i­cines with ter­li­pressin over res­pi­ra­to­ry and car­dio­vas­cu­lar risks

The EMA is handing out a new recommendation surrounding J&J and AbbVie’s cancer drug Imbruvica and some medicines containing terlipressin.

On Friday, the EMA’s safety committee (PRAC) stated that it has taken new measures to reduce the risk of respiratory failure and sepsis when medicines containing terlipressin that are used to treat people with hepatorenal syndrome (HRS-1), particularly for patients with advanced acute-on-chronic liver disease or advanced kidney failure.