Weeks ago, the FDA en­dorsed a Lam­bert-Eaton myas­thenic syn­drome (LEMS) drug from fam­i­ly-run New Jer­sey-based com­pa­ny called Ja­cobus Phar­ma­ceu­ti­cals in pe­di­atric pa­tients, on the ba­sis of adult da­ta, to the sur­prise of Cat­a­lyst Phar­ma­ceu­ti­cals which on­ly last year got its pricey — yet sim­i­lar — treat­ment for the rare au­toim­mune dis­or­der across the fin­ish line in adults. It has now been re­vealed that Ja­cobus’ ver­sion car­ries a price that is less than half of Cat­a­lyst’s Fir­dapse — a move that could fu­el off-la­bel pre­scrip­tion in adults.

Be­fore Cat­a­lyst’s Fir­dapse was sanc­tioned for use 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.

Dos­ing for any of these treat­ments is based on the pa­tient’s weight and dis­ease sever­i­ty. Cat­a­lyst’s Fir­dapse, which is ap­proved with a max­i­mum dose of 80 mg, car­ries an av­er­age list price of $375,000 a year. The com­pa­ny does not dis­close a per pill num­ber, a Cat­a­lyst spokesper­son told End­points News. 

The list price for Ruzur­gi is $80 for each 10 mg tablet, and Ja­cobus’ treat­ment is ap­proved up to a max­i­mum dose of 100 mg, Lau­ra Ja­cobus, who runs the pri­vate­ly-held com­pa­ny, told End­points News. “As an ex­am­ple, the whole­sale cost for a 60 mg dos­ing reg­i­men would be $175,200.00 an­nu­al­ly.  The cost to sup­port a pa­tient re­quir­ing a dai­ly dose of 100 mg would be $292,000.00 an­nu­al­ly.”

Ver­mont Sen­a­tor Bernie Sanders — ahead of his an­nounce­ment to make a sec­ond at­tempt at the pres­i­den­cy — spot­light­ed Cat­a­lyst for the “im­moral ex­ploita­tion of pa­tients”. Oth­er crit­ics of Cat­a­lyst’s pric­ing strat­e­gy sug­gest­ed that de­pend­ing on how Ja­cobus prices its drug, Ruzur­gi, in­sur­ers could be per­suad­ed to fa­vor it over Cat­a­lyst’s prod­uct de­spite be­ing of­fi­cial­ly ap­proved for pe­di­atric use on­ly. 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.

Fir­dapse land­ed on the US mar­ket this Jan­u­ary, and Cat­a­lyst has not­ed it has en­coun­tered min­i­mal push­back from pay­ers, and in­di­cat­ed that typ­i­cal­ly, cov­ered pa­tients pay less than $10 per month out-of-pock­et.

In a note pub­lished last month, Sun­Trust Robin­son Humphrey’s Ed­ward Nash sug­gest­ed that de­spite sug­ges­tions to the con­trary, “(W)e have not seen any prece­dent where pay­ers cov­er an off-la­bel drug for use in un­ap­proved pa­tient pop­u­la­tion”.

Mean­while, HC Wain­wright’s An­drew Fein sug­gest­ed that Ja­cobus, de­spite the rep­u­ta­tion of a “mod­ern-day Robin Hood” is not equipped with the in­fra­struc­ture nor the ex­pe­ri­ence to sup­port a com­mer­cial push of Ruzur­gi, even with the ap­proval in hand.

“Hand­i­capped by le­gal rea­sons, we do not be­lieve that Ja­cobus can open­ly pro­mote off-la­bel use in LEMS adults…It is un­clear if and how Ja­cobus would be able to ex­pand com­mer­cial pen­e­tra­tion oth­er than through pro­mot­ing a pro­lif­er­a­tion of off-la­bel use at aca­d­e­m­ic cen­ters that were en­rolled in the com­pas­sion­ate pro­gram (which mounts to ap­prox­i­mate­ly 200 pa­tients),” he wrote in a May note.

In LEMS pa­tients, the body’s own im­mune sys­tem launch­es an at­tack on the neu­ro­mus­cu­lar junc­tion — which con­nects nerves and mus­cles. The con­di­tion can as­so­ci­at­ed with oth­er au­toim­mune dis­eases, but tends to oc­curs in pa­tients with can­cer. Its preva­lence in pe­di­atric pa­tients is not known, but glob­al­ly it is es­ti­mat­ed to af­fect three per mil­lion in­di­vid­u­als, ac­cord­ing to the FDA. In a re­cent in­vestor pre­sen­ta­tion Cat­a­lyst sug­gest­ed there are 3,000 LEMS US pa­tients, of which 300 are on Fir­dapse.

A STAT re­port pub­lished on Mon­day sug­gest­ed that some adult LEMS pa­tients have in­sin­u­at­ed that Fir­dapse is not ef­fec­tive enough and more tablets be­yond the ap­proved 80 mg dose must be tak­en for re­lief — these ex­tra pills sad­dle them with out-of-pock­et bills that could climb to thou­sands of dol­lars month­ly. The Cat­a­lyst spokesper­son did not pro­vide com­ment on this as­ser­tion.

Im­age: Shut­ter­stock

The rapid development of technology — and the resulting avalanche of data — are catalysts for significant change in the biopharmaceutical industry. This translates into urgent pressures for today’s biopharma, including a need to quickly and affordably develop products with proven therapeutic efficacy and value. This urgency is expedited by the growth of value-based contracting, where access to reimbursement and profit depends on these abilities.

On May 24, FLX Bio, a small cancer and inflammation biotech with backing from GV, changed its name to RAPT Therapeutics and filed confidentially for an IPO. On July 5th, they filed to raise up to $86 million. On July 22, they announced the IPO with a $75 million goal.  And on August 1, they abruptly and without explanation called it all off.

Now, without explanation, they’re reviving the bid, filing again for a $75 million IPO, this time with a new bookrunner and a new drug candidate in the clinic. The terms will be the same: 5 million shares at $14-$16 per share. It would give them a diluted market value of $351 million.

Ahead of its third-quarter results next week, Amgen on Tuesday disclosed the planned retirement of David Meline, who has served as the company’s chief financial officer since 2014.

Meline will be replaced by Ernst & Young vet, Peter Griffith, as CFO come January 1, 2020 — but until then Griffith will serve as executive vice president, finance.

“Over the last 5 years at Amgen, Meline instituted many major changes that led to operational efficiencies and margin expansion while successfully returning cash to shareholders. Now that Amgen is on solid footing, it was a good time to step away,” Cowen’s Yaron Werber wrote in a note. “We do not anticipate any major changes to strategy or operations immediately due to this transition as Amgen is on solid footing.”

Never underestimate the power of a good showing at a scientific conference.
In a presentation late last year, the researchers at Pliant Therapeutics launched a series of discussions about the preclinical data they were pulling together around their work on their small-molecule integrin inhibitor aimed at transforming growth factor beta, or TGF-β, a key pathway involved in fibrosis.
And they got some serious attention for the work.
“We got interest from pharma partners and at the end Novartis basically made it,” says Pliant CEO Bernard Coulie.