Cat­a­lyst Phar­ma's LEMS drug Fir­dapse wins FDA nod, as reg­u­la­tors keep the ap­proval en­gine run­ning at record speed

Turns out, a web er­ror by a com­pa­ny con­trac­tor that tipped off in­vestors Cat­a­lyst Phar­ma­ceu­ti­cals’ $CPRX Fir­dapse had won ap­proval ear­li­er in the week of­fered alert traders a quick score. The for­mal no­tice that the FDA green-light­ed the treat­ment for the rare neu­ro­mus­cu­lar dis­ease came through Wednes­day evening.

The drug is to be used in pa­tients with Lam­bert-Eaton Myas­thenic Syn­drome (LEMS), a rare au­toim­mune dis­ease that af­fects about 1 in every 300,000 peo­ple. In pa­tients with LEMS, the body’s im­mune sys­tem at­tacks the neu­ro­mus­cu­lar junc­tion (the con­nec­tion be­tween nerves and mus­cles) and dis­rupts the abil­i­ty of nerve cells to send sig­nals to mus­cle cells, caus­ing weak­ness and fa­tigue that typ­i­cal­ly cul­mi­nates in dif­fi­cul­ties with dai­ly func­tion­ing. LEMS is of­ten as­so­ci­at­ed with oth­er au­toim­mune dis­eases, and com­mon­ly oc­curs in pa­tients with small cell lung can­cer.

Un­til Fir­dapse, there was no spe­cif­ic FDA-ap­proved treat­ment for LEMS, the agency said. Cat­a­lyst ex­pects to make the drug com­mer­cial­ly avail­able ear­ly in the first quar­ter of 2019.

On Tues­day, the com­pa­ny is­sued a state­ment say­ing it had not yet re­ceived word from the FDA on the sta­tus of its mar­ket­ing ap­pli­ca­tion, even though “a con­trac­tor work­ing on a web page un­in­ten­tion­al­ly al­lowed a live screen to be seen that re­flect­ed that the pos­si­ble ap­proval of the com­pa­ny’s NDA had al­ready been re­ceived.”

Ed­ward Nash

The drug, which is sold by Bio­Marin $BM­RN in Eu­rope where it has been ap­proved since 2009, was giv­en all the bells and whis­tles by the FDA — break­through ther­a­py sta­tus, or­phan drug des­ig­na­tion and pri­or­i­ty re­view — to has­ten its path to­ward ap­proval. But the Coral Gables, Flori­da-based biotech has not had an easy time push­ing for ap­proval – the com­pa­ny was forced to con­duct an ad­di­tion­al tri­al and  shrink its work­force by about third in 2016, af­ter the FDA deemed its mar­ket­ing ap­pli­ca­tion in­com­plete.

Fir­dapse brought in 18.8 mil­lion in Eu­ro­pean sales for Bio­Marin last year. But in the Unit­ed States, it is es­ti­mat­ed to bring in $375 mil­lion in 2025 for Cat­a­lyst, Sun­Trust Robin­son Humphrey’s Ed­ward Nash wrote in a note.

Nash es­ti­mat­ed the drug will car­ry a whole­sale price of $300,000 per pa­tient per year — in-line with oth­er or­phan drugs such as Pro­cys­bi, Gat­tex and Rav­ic­ti — and fore­cast­ed 5% an­nu­al price hikes. Cat­a­lyst will like­ly gen­er­ate Fir­dapse sales of about $17 mil­lion next year, he added.

Fir­dapse is al­so be­ing in­ves­ti­gat­ed for use in oth­er au­toim­mune con­di­tions in two late-stage tri­als — in pa­tients with con­gen­i­tal myas­thenic syn­dromes (CMS) and MuSK an­ti­body pos­i­tive Myas­the­nia Gravis (MuSK-MG), re­spec­tive­ly. If ap­proved in these two con­di­tions, Nash es­ti­mates Cat­a­lyst could fur­ther rake in a com­bined $491 mil­lion in 2025.

The ap­proval marks the FDA’s 55th of the year, sur­pass­ing fur­ther its record set in 1996 of 53 ap­provals.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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Joseph Kim, Inovio CEO (Andrew Harnik, AP Images)

Caught in a stand­off with its con­tract man­u­fac­tur­er over Covid-19 vac­cine, In­ovio files suit in an at­tempt to break free while ri­vals race ahead

Inovio was one of the first vaccine developers to snag attention for a jab that their execs said promised to end the Covid-19 pandemic. Using their own unique DNA tech, CEO Joseph Kim said it took just 3 hours to work it out.

But while rivals are racing to the finish line with ambitious plans to make vast quantities of their vaccines with billions of dollars of deals, Inovio is still stuck at the starting line on manufacturing.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

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