Snubbed at the FDA, PTC files their three-time los­er for Duchenne MD over protest

PTC Ther­a­peu­tics $PTCT isn’t go­ing to let a slate of three con­sec­u­tive, failed stud­ies for ataluren block its re­view at the FDA. Us­ing the agency’s rules for ap­pli­ca­tions on drugs reg­u­la­tors have re­fused to file, the biotech says it filed over protest, us­ing reg­u­la­to­ry pro­ce­dures to force a PDU­FA date of Oc­to­ber 24.

PTC CEO Stu­art Peltz was frank about why the FDA wouldn’t ac­cept their ap­pli­ca­tion ear­ly last year. Reg­u­la­tors felt that the com­pa­ny had flat failed to make a case for the drug that was wor­thy of a re­view. And he’s been beat­ing on the FDA’s door ever since, de­spite the agency’s de­ci­sion to re­ject its first ap­peal, claim­ing once again that the “to­tal­i­ty” of the da­ta war­rant its ap­proval.

In a state­ment, the CEO said he would be “work­ing close­ly with the FDA and the DMD com­mu­ni­ty to bring this much-need­ed ther­a­py to pa­tients.”

Cur­rent­ly, the EU al­lows the drug to be sold af­ter grant­i­ng a con­di­tion­al ap­proval that now re­quires PTC to con­duct a new clin­i­cal tri­al over the next 5 years. PTC’s move to­day un­der­scores a dif­fer­ent set of stan­dards that reg­u­la­tors have ap­plied to drugs in the field.

While the FDA spurned PTC and re­ject­ed Bio­Marin’s dris­apersen, the FDA’s Janet Wood­cock over­rode the stren­u­ous ob­jec­tions of nu­mer­ous sub­or­di­nates who felt that Sarep­ta had nev­er made its case for eteplirsen. Pa­tients and fam­i­lies or­ga­nized a co­or­di­nat­ed cam­paign to win an ap­proval, though, and the drug is now sold as Ex­ondys 51 with a la­bel that says ef­fi­ca­cy of the drug has nev­er been es­tab­lished.

Jef­feries’ Gena Wang doesn’t give PTC very good odds.

We not­ed that the ‘fil­ing over protest’ has been rarely pur­sued and would be con­sid­ered as a new ap­pli­ca­tion pro­ce­du­ral­ly. We found two prece­dents – Phar­ma­cyclics’ Xcytrin in lung can­cer brain metas­tases (fil­ing over protest in Apr ’07, PDU­FA date of Dec 31 ’07 and non-ap­prov­able let­ter on Dec 21 ’07), and GSK’s Bexxar in Non-Hodgkin’s Lym­phoma (ini­tial fil­ing in 1999, and ac­cel­er­at­ed ap­proval af­ter fil­ing over protest in June ’03; with­drawn by GSK in 2013 due to in­com­plete post-mar­ket­ing tri­als).

“The bull ar­gu­ment for PTCT has been that eteplirsen’s ap­proval “opens the door” for ataluren,” not­ed RBC’s Simos Sime­oni­dis. “We def­i­nite­ly agree that both ap­pli­ca­tions are “less-than-per­fect” and, in our view, nei­ther one should have been ap­proved. But even though we dis­agreed with FDA on eteplirsen, we can un­der­stand their ra­tio­nale for ac­cel­er­at­ed ap­proval. The key and very sig­nif­i­cant dif­fer­ence be­tween the two ap­pli­ca­tions is that we would char­ac­ter­ize the eteplirsen dataset as in­com­plete and the ques­tion of whether eteplirsen “works” in DMD as still unan­swered. This can­not be said about ataluren, where mul­ti­ple large, well-con­trolled, ran­dom­ized tri­als have been con­duct­ed. ” They all failed.

PTC ac­knowl­edged just days ago that its drug failed a late-stage pro­gram for cys­tic fi­bro­sis, adding to the ev­i­dence that the drug doesn’t work. Ear­li­er it failed a Phase IIb for Duchenne MD fol­lowed by an­oth­er flop in Phase III, de­signed to over­come the prob­lems the biotech saw ear­li­er.

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Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Cedric Ververken, Confo Therapeutics CEO

Dai­ichi Sankyo inks $183M dis­cov­ery deal with GPCR biotech for CNS tar­get

Belgian biotech Confo Therapeutics has landed $183 million, plus potential royalties, in a drug-discovery deal with Daiichi Sankyo.

Early Thursday, Confo Therapeutics put out word of the deal that will be focused on small molecule antagonists to go after an undisclosed target that the company says is associated with CNS diseases.

Confo CEO Cedric Ververken told Endpoints News that Daiichi originally reached out to learn about the biotech’s technology. He added that Confo, founded in 2015, will use its platform to drug a GPCR target that Daiichi has struggled with internally.

Dif­fu­sion to hand Nas­daq spot to EIP Phar­ma for PhI­Ib de­men­tia study of ex-Ver­tex drug

One of the more than a dozen bidders for Diffusion Pharmaceuticals’ spot on Nasdaq has prevailed.

Boston biotech EIP Pharma will merge with Diffusion in an all-stock deal, with plans to start a Phase IIb clinical trial in the coming months in a common form of dementia with no approved treatments. The combined company will be renamed CervoMed.

The nine-year-old privately-held EIP is working on a former Vertex drug that it will test in a 160-person Phase IIb in patients with dementia with Lewy bodies, or DLB. The National Institute on Aging is expected to fund that trial with a $21 million grant. With the reverse merger, slated for closing in the middle of this year, EIP will be funded through that readout in the second half of 2024. EIP’s equity and debt holders will own about 77.25% of the combined company.

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CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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FDA ap­proves Nar­can opi­oid over­dose re­ver­sal spray for over-the-counter sale

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.