More bad news for Sarep­ta; No­var­tis, Mi­crosoft join forces in AI pact

Sarep­ta $SRPT is still feel­ing the heat. A lit­tle more than a month af­ter the FDA threw an un­ex­pect­ed sur­prise re­jec­tion for the com­pa­ny’s ap­pli­ca­tion for an ac­cel­er­at­ed ap­proval of its Duchenne MD drug, golodirsen, the com­pa­ny faced more bad news to­day. A ri­val com­pa­ny in Japan, NS Phar­ma, said that it had sub­mit­ted the nec­es­sary pa­per­work need­ed for ap­proval of their new drug, vil­to­larsen, to treat Duchenne MD for chil­dren born with the dis­or­der caused by a mu­ta­tion in the DNA se­quence ex­on 53. Ac­cord­ing to a STAT re­port, NS Phar­ma ex­pects vil­to­larsen to be ap­proved in the US by the mid­dle of 2020, if not soon­er — bar­ring any set­backs.

No­var­tis $NVS is “reimag­in­ing med­i­cine” by tap­ping in­to tech­nol­o­gy that is now firm­ly en­trenched across the bio­phar­ma in­dus­try: ar­ti­fi­cial in­tel­li­gence. Its part­ner of choice for its AI in­no­va­tion lab is the tech ti­tan Mi­crosoft $MS­FT — and the col­lab­o­ra­tion is broad, al­though sparse in de­tail.  The idea is to 1) “bring the pow­er of AI to the desk­top of every No­var­tis as­so­ciate…to take on the next wave of chal­lenges in med­i­cine” and 2) to “tack­le some of the hard­est com­pu­ta­tion­al chal­lenges with­in the life sci­ences,” in­clud­ing the op­ti­miza­tion of cell and gene ther­a­pies at scale. Re­call, No­var­tis has had some trou­ble with the man­u­fac­tur­ing of its CAR-T ther­a­py Kym­ri­ah.

→ An­oth­er win to add to the books for Mer­ck‘s $MRK block­buster, Keytru­da, which has been ap­proved as a monother­a­py in Chi­na for first-line treat­ment of cer­tain pa­tients with ad­vanced or metasta­t­ic non-small cell lung can­cer (NSCLC) whose tu­mors that ex­press PD-L1. The ap­proval was based up­on over­all sur­vival find­ings from the Phase III KEYNOTE-042 tri­al, in­clud­ing da­ta from an ex­ten­sion of the glob­al study in Chi­nese pa­tients. This ap­proval makes Keytru­da the “first an­ti-PD-1 ther­a­py ap­proved in Chi­na as both monother­a­py and in com­bi­na­tion with chemother­a­py for the first-line treat­ment of ap­pro­pri­ate pa­tients with NSCLC.”

In May, the FDA hand­ed Heron Ther­a­peu­tics $HRTX an un­ex­pect­ed re­jec­tion — re­lat­ing to man­u­fac­tur­ing con­cerns — for the com­pa­ny’s long-act­ing non-opi­oid painkiller, HTX-011, de­signed to treat post-sur­gi­cal anal­ge­sia. The com­pa­ny an­nounced to­day that it has re­sub­mit­ted its NDA for the drug to the FDA and that they ex­pect a 6-month re­view by the agency.

→ He­mo­phil­ia play­er Bio­Marin $BM­RN, has opened a new head­quar­ters in Dublin, Ire­land — dou­bling its cur­rent team of 500 in the re­gion. The new head­quar­ters is just one ex­pan­sion that the com­pa­ny has been work­ing on. The com­pa­ny is con­tin­u­ing to work on a €38 mil­lion ex­pan­sion of its plant at Shan­bal­ly in Cork, which is the com­pa­ny’s on­ly man­u­fac­tur­ing plant out­side of the Unit­ed States.

The Fi­nan­cial Times has re­port­ed that Chi­nese ven­ture cap­i­tal in­vest­ment in the US has hit its low­est lev­el since 2015. But as funds are trick­ling in at a slow pace in the US, Chi­na has turned to else­where such as In­dia and South-East Asia for its in­vest­ments. Ac­cord­ing to Re­fini­tiv da­ta, Chi­nese funds in­vest­ed just $4 bil­lion in US com­pa­nies in the first nine months of 2019, which is the small­est since 2015, down from near­ly $7 bil­lion in the same time frame last year and $9 bil­lion in the same 2017 pe­ri­od.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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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

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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President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

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.

Credit: AP Images

Covid-19 roundup: BAR­DA sup­ports Op­er­a­tion Warp Speed with big $628M con­tract to ser­vice Amer­i­ca's vac­cine pro­duc­tion needs

Another BARDA contract designed to service America’s Covid-19 vaccine needs has been deployed.

The White House-led initiative designed to bankroll development to bring a vaccine to the American public by this fall — Operation Warp Speed — has via BARDA handed a meaty contract to the maker of an FDA-licensed anthrax vaccine to open up its manufacturing apparatus to shore up production of Covid-19 vaccines.

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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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A low-pro­file biotech bests Re­gen­eron in high-pro­file patent suit

For nearly a decade now, the low-profile Cambridge biotech Kymab has been battling in US, UK, Japanese and Australian courts with the biotech behemoth Regeneron.

Regeneron has turned itself into a $70 billion company off of a platform of transgenically humanized mice they can use to make antibodies for anything from Ebola to colorectal cancer. The technology took decades and billions to build, 20 years from the company’s founding to the first approved drug. And the company guards and touts it zealously, breaking their production process down into various branded components — Velocimmune, Velocigene, Velocimouse and four other Velocis — and sometimes suing would-be copycats. In 2014, most notably, they sued two Pfizer-backed entities for patent infringement.

Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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