Joe Todisco, CorMedix CEO

CorMedix hit with sec­ond CRL for an­ti­fun­gal treat­ment, cit­ing man­u­fac­tur­ing and API is­sues

Fol­low­ing a C-suite shuf­fle, a shut­ter­ing of its Eu­ro­pean op­er­a­tions and a CRL for its treat­ment to pre­vent blood­stream in­fec­tions in pa­tients un­der­go­ing chron­ic he­modial­y­sis, CorMedix is still not im­press­ing the FDA.

The biotech has now been giv­en a sec­ond re­jec­tion let­ter, as the NDA sur­round­ing its an­ti­fun­gal so­lu­tion, De­fen­Cath, can­not be ap­proved un­til is­sues sur­round­ing its con­tract man­u­fac­tur­ing or­ga­ni­za­tion and the sup­pli­er of the API he­parin dur­ing in­spec­tions are re­solved, CorMedix said Mon­day af­ter­noon.

While CorMedix has been work­ing with its third-par­ty man­u­fac­tur­er to cor­rect any prob­lems, it has still not been cleared to go ahead with the NDA.

Sep­a­rate­ly, the FDA al­so in­spect­ed the fa­cil­i­ty of the com­pa­ny’s he­parin sup­pli­er. This led to a warn­ing let­ter for the API sup­pli­er for hav­ing man­u­fac­tur­ing de­fi­cien­cies for a non-he­parin API. Though the sup­pli­er has re­tained an in­de­pen­dent con­sul­tant to cor­rect the prob­lems, they could not be re­solved be­fore the PDU­FA date.

As a re­sult of these is­sues, CorMedix has now sought an­oth­er CMO in Al­ca­mi Cor­po­ra­tion and is in the process of hav­ing its tech trans­ferred over. CorMedix is al­so get­ting its sup­ply of he­parin from an­oth­er al­ter­na­tive source as well. CorMedix now ex­pects to send its NDA back around the first quar­ter of next year.

“While I am dis­ap­point­ed that we will not re­ceive FDA ap­proval for the NDA on our PDU­FA date, I am en­cour­aged to have sub­stan­tive la­bel­ing and clin­i­cal re­view com­plet­ed by FDA and I am con­fi­dent there is a line of sight to FDA ap­proval of the NDA, once ei­ther our ex­ist­ing CMO and API sup­pli­er ob­tain com­pli­ance clear­ance, or we are able to sub­mit and ob­tain reg­u­la­to­ry ap­proval for man­u­fac­tur­ing at Al­ca­mi,” said CorMedix CEO Joe Todis­co in a state­ment.

This is not the first time the com­pa­ny has faced out­side man­u­fac­tur­ing prob­lems. Last year’s CRL al­so stemmed from de­lays from a con­tract­ed man­u­fac­tur­er, prompt­ing the com­pa­ny to go through the re­sub­mis­sion process a sec­ond time.

Right af­ter this, the com­pa­ny end­ed its Eu­ro­pean busi­ness and dis­con­tin­ued the dis­tri­b­u­tion of Neu­trolin, which is used to pre­vent catheter-re­lat­ed blood in­fec­tions, de­vot­ing its full fo­cus to De­fen­Cath.

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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Elisabeth Stampa, Medicines for Europe president

As win­ter ap­proach­es, a Eu­ro­pean gener­ics group rais­es alarm over en­er­gy prices for man­u­fac­tur­ers

While colder temperatures are fast approaching, the situation surrounding the rise of energy prices in Europe is hitting businesses of every kind, including generic drug manufacturers.

Medicines for Europe, a group that represents the generic industry on the continent, sent a letter addressed to energy ministers and commissioners concerning inflation and the costs of energy on the supply of generic medicines.

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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