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.

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

Sanofi and Regeneron can boast of another inflammatory disease where Dupixent has proven effective.

The best-selling drug, which targets both IL-4 and IL-13, has delivered a clean sweep in a Phase III trial for prurigo nodularis, a chronic disease characterized by itch so intense that it can affect patients’ sleep and psychology. Thick skin lesions can cover most of the body.

On the primary endpoint, 37% of patients taking Dupixent saw a clinically meaningful reduction in itch compared to 22% of those on placebo (p=0.0216) at week 12. All secondary endpoints were also met, including clearance of skin lesions and improvement in quality of life.

Ocular Therapeutix $OCUL has had its ups and downs in the 7 years since it went public. Friday was one of those down days.

The Bedford, MA-based biotech reported that its lead experimental eye drug, OTX-CSI (cyclosporine intracanalicular insert), failed a Phase II trial for dry eye disease. And the stock experienced one of its periodic meltdowns, dropping more than 30% ahead of the bell.

The therapy flat failed the primary endpoint: increased tear production at 12 weeks as measured by the Schirmer’s Test compared to the vehicle control group. And while investigators called out an improvement from baseline in “signs of dry eye disease as measured by total corneal fluorescein staining (CFS) and symptoms of dry eye disease as measured by the visual analogue scale (VAS) eye dryness in subjects treated with the OTX-CSI insert,” it wasn’t statistically significant.

Pfizer will present its case to the FDA’s vaccine adcomm next week, seeking authorization for a lower-dose version of its Covid-19 vaccine for kids ages 5 through 12, which the Biden administration said will likely begin rolling out early next month.

Two primary doses of the 10 µg vaccine (the dose for those ages 12 and up is 30 μg) given 3 weeks apart in this group of children “have shown a favorable safety and tolerability profile, robust immune responses against all variants of concern including Delta, and vaccine efficacy of 90.7% against laboratory-confirmed symptomatic COVID-19,” the company said in briefing documents ahead of next Tuesday’s meeting of the FDA’s Vaccines and Related Biological Products Advisory Committee.