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.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology

ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development

CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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SEC calls out lit­tle Ther­a­peu­tic­sMD for its in­sid­er con­tacts with an­a­lysts to boost share price, then halt rout

Back in May 2017, following an FDA rejection, TherapeuticsMD saw its share price plummet to the lowest levels in two years. The little Florida biotech eventually found its way back to the good side of regulators, scoring a curious OK a year later for its therapy preventing vaginal pain during sex. But the SEC is now accusing it of selectively disclosing nonpublic information in attempts to manipulate its stock.

In two instances in June and July of 2017, TherapeuticsMD allegedly violated the Regulation Fair Disclosure rule by sharing material information with certain sell-side analysts and not the public, resulting in a more favorable stock move than otherwise would be expected.

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Therapists Marcela Ot'alora and Bruce Poulter are trained to conduct MDMA-assisted psychotherapy. In this reenactment, they demonstrate how they help guide and watch over a patient who is revisiting traumatic memories while under the influence of MDMA. (Photo: Multidisciplinary Association for Psychedelic Studies)

MD­MA, now in Phase III, shows promise as a PTSD treat­ment

The first time Lori Tipton tried MDMA, she was skeptical it would make a difference.

“I really was, at the beginning, very nervous,” Tipton said.

MDMA is the main ingredient in the club drug known as ecstasy or molly. But Tipton wasn’t taking pills sold on the street to get high. She was trying to treat her post-traumatic stress disorder by participating in a clinical trial.

After taking a dose of pure MDMA, Tipton lay in a quiet room with two specially trained psychotherapists. They sat next to her as she recalled some of her deepest traumas, such as discovering her mother’s body after Tipton’s mother killed two people and then herself in a murder-suicide.

Ted Ashburn. Oncorus

Cowen, Per­cep­tive lead $79.5M Se­ries B for 's­tand­out' biotech shep­herd­ing on­colyt­ic virus to clin­ic

As several Big Pharma players secure biotech partners in the oncolytic virus space for new immuno-oncology combos, Cowen and Perceptive Advisors have come out with their own bet on a startup that promises to shine.

The marquee investors are joining MPM, Deerfield, Celgene, Astellas, Arkin Bio Ventures and UBS Oncology Impact Fund in backing the drug developer, Oncorus, which will now deploy the $79.5 million in Series B cash toward clinical development of its lead program. Other new investors include Surveyor Capital, Sphera Funds, IMM Investment, QUAD Investment Management, UTC Investment, SV Investment Corp and Shinhan Investment-Private Equity, the last five of which are Korean-based funds.

Fu­til­i­ty analy­sis au­gurs de­feat in piv­otal tri­al test­ing of Nu­Cana's lead drug in metasta­t­ic pan­cre­at­ic can­cer

Nearly two years after making its public debut, UK-based NuCana’s mission to make chemotherapies more potent and safer was dealt a blow, after a pivotal study testing its lead experimental drug halted enrollment in a hard-to-treat advanced form of cancer, following a futility analysis.

The drug, Acelarin, is being evaluated for use in metastatic pancreatic cancer patients who were not considered suitable for combination chemotherapy. In the late-stage ACELARATE study — which compared the experimental drug against the chemotherapy gemcitabine — 200 patients had been enrolled by the sponsor, Clatterbridge Cancer Centre, before an analysis from an independent safety and data monitoring panel suggested the study’s main goal would not be met.