Two CRLs lat­er, FDA en­dors­es drug to pre­vent hear­ing loss as­so­ci­at­ed with chemo

Af­ter re­ject­ing it twice, the FDA has fi­nal­ly ap­proved a new drug for low­er­ing the risk of hear­ing loss among chil­dren be­ing treat­ed for can­cer with chemother­a­py.

Ped­mark, as Fen­nec Phar­ma calls its drug, is a for­mu­la­tion of sodi­um thio­sul­fate. Giv­en that oto­tox­i­c­i­ty is a ma­jor dose-lim­it­ing side ef­fect of cis­platin chemother­a­py and there were no avail­able treat­ments for it, the FDA grant­ed North Car­oli­na-based Fen­nec both fast track and break­through ther­a­py des­ig­na­tions back in 2018, and put the drug on pri­or­i­ty re­view two years lat­er.

But man­u­fac­tur­ing is­sues that turned up at a pre-ap­proval in­spec­tion and were doc­u­ment­ed in a Form 483 ul­ti­mate­ly led to a com­plete re­sponse let­ter from the FDA. A re­sub­mis­sion one year lat­er failed to change the FDA’s minds.

Then this April, Fen­nec filed for ap­proval a third time.

In its re­cent quar­ter­ly re­port, the com­pa­ny not­ed that it’s built out the com­mer­cial or­ga­ni­za­tion over the past months with key hires in sales, mar­ket ac­cess and field-based med­ical teams.

Ped­mark is in­di­cat­ed for pe­di­atric pa­tients be­tween 1 month to 18 years old who have lo­cal­ized, non-metasta­t­ic sol­id tu­mors and have been treat­ed with cis­platin in­fu­sions for no longer than six hours. For this group of pa­tients, hear­ing loss stem­ming from such chemother­a­pies is of­ten long-last­ing and per­ma­nent, and cur­rent­ly they would need to re­sort to cochlear im­plants.

Com­mon side ef­fects in­clude hy­per­sen­si­tiv­i­ty, hy­per­na­trem­ia (hav­ing too much sodi­um in the blood), hy­pokalemia (hav­ing too lit­tle potas­si­um), nau­sea and vom­it­ing.

Shares $FENC rose al­most 16% to $7.63.

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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#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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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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Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

#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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