Kym­ri­ah wins sec­ond FDA OK, boost­ing hope for No­var­tis' low CAR-T sales

Af­ter dis­ap­point­ing in­vestors this year with slug­gish sales, the prospects for No­var­tis’ CAR-T ther­a­py Kym­ri­ah may be im­prov­ing. The FDA has hand­ed the phar­ma gi­ant its sec­ond ap­proval for the drug, the com­pa­ny said Tues­day af­ter­noon.

The drug, which was first ap­proved last Au­gust for pa­tients un­der 25 with B-cell pre­cur­sor acute lym­phoblas­tic leukemia, is now OK’ed to treat large B-cell lym­phoma. The can­cer must be re­lapsed or re­frac­to­ry, and the pa­tient must have gone through two or more lines of sys­temic ther­a­py first.

Kym­ri­ah — the first CAR-T ever to get ap­proval — is now al­so the first CAR-T to get ap­proval for two dis­tinct in­di­ca­tions in non-Hodgkin lym­phoma (NHL) and B-cell ALL.

Liz Bar­rett

“To­day’s FDA ap­proval of Kym­ri­ah pro­vides an­oth­er op­por­tu­ni­ty for No­var­tis to build on its lead­er­ship in CAR-T de­vel­op­ment, de­liv­er­ing a po­ten­tial­ly trans­for­ma­tive ther­a­py with durable and sus­tained re­sponse rates and a well-char­ac­ter­ized safe­ty pro­file to help pa­tients in dire need of new treat­ment op­tions,” said Liz Bar­rett, CEO of No­var­tis On­col­o­gy, in a state­ment. “We look for­ward to lever­ag­ing all of our learn­ings and new ca­pa­bil­i­ties from the ini­tial launch of Kym­ri­ah in pe­di­atric and young adult B-cell ALL for this larg­er group of pa­tients.”

Kym­ri­ah was a bit of a dis­ap­point­ment to $NVS in­vestors when Q1 re­sults came out. In the first quar­ter of 2018, the treat­ment brought in $12 mil­lion, which is rough­ly four times be­low the sales need­ed to meet an­a­lyst ex­pec­ta­tions of around $159 mil­lion for the year.

The cost of Kym­ri­ah ($475,000) could be part of the prob­lem, but there’s al­so the small pa­tient group to con­sid­er. Its first in­di­ca­tion is a rare can­cer that af­fects on­ly 3,100 new pa­tients each year, and the drug on­ly tar­gets rough­ly 30% of those pa­tients who do not re­spond to stan­dard ther­a­py.

The new ap­proval will add to that pa­tient pool and give in­vestors rea­son to hope for bet­ter sales fig­ures down the road.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Sen­ate Fi­nance Com­mit­tee lobs more bi­par­ti­san pres­sure on­to PBMs

Congress is honing in on how it wants to overhaul the rules of the road for pharmacy benefit managers, with a Senate Finance Committee hearing Thursday serving as the latest example of the Hill’s readiness to make changes to how pharma middlemen operate.

While pledging to ensure patients and pharmacies “don’t get a raw deal,” Finance Committee Chair Ron Wyden (D-OR) laid out the beginning of what looks like a major bipartisan effort — moves the PBM industry is likely to challenge vigorously.

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Nicklas Westerholm, Egetis Therapeutics CEO

Ac­qui­si­tion talks on­go­ing for Swedish rare dis­ease biotech Egetis, shares up al­most 40%

Shares of the Sweden-based rare disease biotech Egetis Therapeutics skyrocketed on Thursday afternoon as the company said it’s engaged in “ongoing discussion” with external parties regarding a “potential acquisition.”

Egetis confirmed rumors with a statement on Thursday while noting that there is no certainty that a takeover offer will be made.

Nonetheless, the possibility of an acquisition has shot up Egetis’ share price. By the afternoon on Thursday, its stock price was {$EGTX.ST} up over 38%. An Egetis spokesperson told Endpoints News in an email that it has no further comments.

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Lu­pus drug de­vel­op­ment mar­ket heat­ing up, while FDA links with ad­vo­ca­cy group to fur­ther ac­cel­er­ate re­search

The long-underserved systemic lupus erythematosus (SLE) market is suddenly buzzing with treatment possibilities. Less than two years after AstraZeneca’s approval for Saphnelo — the first new SLE drug in a decade and joining just one other approved in GSK’s Benlysta – the pipeline of potential drugs numbers in the dozens.

Although most are very early stage — Spherix Global Insights estimates five in Phase II/III — the pharma R&D enthusiasm is catching on among doctors, patients and advocacy groups. On Wednesday, the Lupus Research Alliance and the FDA formed a novel private-public partnership called Lupus Accelerating Breakthroughs Consortium (Lupus ABC) to help advance lupus clinical trial success.

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Stéphane Bancel, Moderna CEO (AP Photo/Markus Schreiber)

Mod­er­na so­lid­i­fies deal with Kenya to build mR­NA man­u­fac­tur­ing fa­cil­i­ty

The mRNA player Moderna is further cementing its presence on the African continent.

Moderna announced on Thursday that it has finalized an agreement with Kenya’s government to partner up and bring an mRNA manufacturing facility to the east African nation. The new facility aims to manufacture up to 500 million doses of vaccines annually. Moderna also said the new facility will have the ability to spike its production capabilities to respond to public health emergencies on the continent or globally.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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J&J bows out of RSV vac­cine race, end­ing PhI­II study and ced­ing to Pfiz­er, GSK

Johnson & Johnson announced Wednesday morning it is ending development of its adult RSV vaccine that was in the middle of a 27,200-patient trial, giving up a big slice of what’s expected to be the next multibillion-dollar pharma market.

The decision came down to the shifting RSV “competitive landscape,” a company spokesperson tells Endpoints News, adding the “breadth of options” was much different than when J&J first started its pivotal study. The spokesperson declined to comment on the Phase III data, saying only the shot is undergoing an “ongoing assessment.”

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Deborah Waterhouse, ViiV Healthcare CEO

MPP re­cruits three gener­ic man­u­fac­tur­ers to ex­pand use of Vi­iV's in­jectable PrEP drug

ViiV Healthcare has teamed up with the UN-backed Medicines Patent Pool and three generic manufacturers to expand access to medicine that can prevent HIV.

ViiV and the Medicines Patent Pool jointly announced Thursday that the MPP signed sub-licensing agreements with Aurobindo, Cipla and Viatris to manufacture generics of a long-acting form of cabotegravir for HIV pre-exposure prophylaxis (PrEP). As a result of the agreement, the manufacturers will be able to develop, manufacture and supply generic versions of cabotegravir LA in 90 countries — subject to regulatory approvals.