Months af­ter Fir­dapse mak­er Cat­a­lyst Phar­ma­ceu­ti­cals filed a law­suit against the FDA, the Flori­da com­pa­ny on Wednes­day said the drug had failed a piv­otal tri­al in pa­tients af­flict­ed with con­gen­i­tal myas­thenic syn­dromes (CMS), an um­brel­la term for rare neu­ro­mus­cu­lar dis­or­ders com­pris­ing a spec­trum of more than 50 ge­net­ic de­fects.

The drug, known chem­i­cal­ly as am­i­fam­pri­dine, was sanc­tioned for use in adult Lam­bert-Eaton myas­thenic syn­drome (LEMS) pa­tients in No­vem­ber 2018. The com­pa­ny is work­ing on ex­pand­ing the la­bel to in­clude pa­tients with CMS, MuSK-pos­i­tive myas­the­nia gravis (MuSK-MG) and spinal mus­cu­lar at­ro­phy (SMA).

Cat­a­lyst es­ti­mates that there are be­tween 1,000 and 1,500 CMS pa­tients in the Unit­ed States — there are at least 600 fam­i­lies with af­fect­ed in­di­vid­u­als who have been rep­re­sent­ed in sci­en­tif­ic lit­er­a­ture, ac­cord­ing to the NIH. Pa­tients with CMS car­ry mu­ta­tions in genes en­cod­ing pro­teins es­sen­tial for neu­ro­mus­cu­lar trans­mis­sion.

Cat­a­lyst’s late-stage study test­ed the drug against a place­bo in ge­net­i­cal­ly con­firmed CMS pa­tients. 20 pa­tients aged two and above were en­rolled in the tri­al, and 16 were ran­dom­ized. The drug failed to meet the main goal of sub­ject glob­al im­pres­sion (SGI) scale, a mea­sure used by clin­i­cians to rate the sever­i­ty of the ill­ness at the time of as­sess­ment, rel­a­tive to the clin­i­cian’s past ex­pe­ri­ence with pa­tients with the same di­ag­no­sis.

The com­pa­ny’s shares $CPRX slipped about 12.3% to $5 in pre­mar­ket trad­ing.

The sec­ondary end­point of mus­cle func­tion mea­sure (MFM) across all test­ed sub­types was al­so not met, al­though in­di­vid­ual pa­tient im­prove­ments were ob­served in some pa­tient sub-groups, the com­pa­ny said.

“Due to the small pa­tient preva­lence, the low num­ber of pa­tients test­ed, and het­ero­gene­ity of the dis­ease with a wide range of vari­a­tion in clin­i­cal pre­sen­ta­tion across its more than 50 sub­types, it was chal­leng­ing to demon­strate a sta­tis­ti­cal­ly sig­nif­i­cant ben­e­fit across mul­ti­ple sub­types,” Steven Miller, Cat­a­lyst’s COO and CSO said in a state­ment.

The com­pa­ny will meet the FDA be­fore the end of 2019 to fig­ure out the next steps for the CMA pro­gram. Mean­while, the Cat­a­lyst ex­pects to re­port da­ta from the MuSK-MG tri­al as well as re­sults from its SMA proof of con­cept study in the first half of next year.

Hav­ing launched in Jan­u­ary, Fir­dapse gen­er­at­ed about $41.3 mil­lion in sales in the first half of this year. Be­fore the drug (which car­ries an av­er­age an­nu­al list price of $375,000) was ap­proved by the FDA, hun­dreds of pa­tients had been able to ac­cess a sim­i­lar drug from com­pound­ing phar­ma­cies for a frac­tion of the cost, or Ja­cobus’ for free, as part of an FDA-rat­i­fied com­pas­sion­ate use pro­gram.

But the ap­proval of the Cat­a­lyst drug — ac­com­pa­nied by mar­ket ex­clu­siv­i­ty span­ning sev­en years — ef­fec­tive­ly pre­clud­ed Ja­cobus and com­pound­ing phar­ma­cies from sell­ing their ver­sions.

Then, in an un­ex­pect­ed twist, the FDA en­dorsed New Jer­sey-based Ja­cobus’ ver­sion in pe­di­atric pa­tients, on the ba­sis of adult da­ta — a move that could spark off-la­bel pre­scrip­tion in adults (As far as the FDA is con­cerned, doc­tors can pre­scribe drugs for off-la­bel use when they judge that it is med­ical­ly ap­pro­pri­ate for their pa­tient). Adding fu­el to the fire, Ja­cobus’s drug, Ruzur­gi, car­ries a list price that is less than half of Fir­dapse’s. Cat­a­lyst main­tains that typ­i­cal­ly, cov­ered pa­tients pay less than $10 per month out-of-pock­et.

In an in­ter­view with End­points News ahead of Cat­a­lyst’s third-quar­ter re­sults ex­pect­ed mid-No­vem­ber, chief Patrick McE­nany said that the Ruzur­gi ap­proval has trig­gered a “trick­le of ero­sion” on its LEMS pa­tient base. “It was not un­ex­pect­ed,” he said.

In June, Cat­a­lyst filed a law­suit against the health reg­u­la­tor — ef­fec­tive­ly ac­cus­ing the agency of bow­ing to po­lit­i­cal pres­sure sur­round­ing sky­rock­et­ing drug prices. Un­der fed­er­al law, the agency is meant to treat all com­pa­nies in the same man­ner. Cat­a­lyst has as­sert­ed the agency un­der­mined its or­phan drug ex­clu­siv­i­ty, and vi­o­lat­ed fed­er­al law by play­ing fa­vorites in the con­text of a hy­per­vig­i­lant pric­ing en­vi­ron­ment.

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Every few years, a public health crisis (think Ebola, Zika) spurred by a rogue pathogen triggers a small-biotech rally, as drugmakers emerge from the woodwork with ambitious plans to treat the mounting outbreak. In most cases, that enthusiasm never quite delivers.

Things are no different, as the coronavirus outbreak in Wuhan, China takes hold. There have been close to 300 confirmed human infections in China, and at least four deaths. Coronaviruses are a large family of viruses, which include MERS and SARS. On Tuesday, the CDC reported the virus was detected in a US traveler returning from Wuhan.

Update: Nominations open through end of day, Monday, January 27

Two years ago, when we did our first Endpoints 20-under-40, we profiled a set of up-and-comers who promised to help reshape the industry as we know it. Now we’re back and once again looking for the top 20 biopharma professionals under the age of 40. We’ll be profiling folks who have accomplished a lot at a young age but seem on the verge of accomplishing so much more.

The venture tide might have subsided, the IPO window may be closing and certain listed biotechs may be having a tough time amid Neil Woodford’s well-publicized demised, but there’s still plenty to celebrate in the UK BioIndustry Association’s eyes.

Overall investment in UK biotech last year fell from the record-breaking £2.2 billion levels of 2018 to £1.3 billion — including £679 million in venture capital, a meager £64 million in IPOs plus £596 million when you add up all public financings, according to a new report from the BIA.

→ Blueprint Medicines filed an amendment to its application to get the gastrointestinal stromal tumor (GIST) drug Ayvakit approved in fourth-line GIST, the company disclosed in the prospectus for a new $325 million public offering.  Blueprint got a big accelerated OK on the drug this month in a particular mutation, but because the FDA decided to split their review in two, they didn’t hear on fourth-line GIST. They were supposed to hear before February 14, but this amendment could push that date back by 3 months. Blueprint wrote that the amendment is designed to allow the company to comply with the FDA’s request for data from the Phase III VOYAGER before they give a judgment.