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.

Jude Samulski, Marianne De Backer

Bay­er buys a biotech ‘race horse’ with a $4B deal — $2B in cash — aimed at go­ing big in­to gene ther­a­py

In the latest sign that Big Pharma wants a leading place in the push to develop a new generation of cell and gene therapies, Bayer is stepping up today with a $2 billion cash deal to buy out one of the fast-moving pioneers in the field, while adding up to $2 billion more in milestones if the new pharma subsidiary can deliver the goods.

As part of a continuing series of deals engineered by Bayer BD chief Marianne De Backer, the pharma player has snapped up Asklepios, more commonly referred to in more casual fashion as AskBio. And they are paying top dollar for a Research Triangle Park-based company that raised $225 million a little more than a year ago to back the brainchild of Jude Samulski, the gene therapy pioneer out of the University of North Carolina Gene Therapy Center.

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Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

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News brief­ing: No­var­tis' Zol­gens­ma hits block­buster sta­tus, trig­ger­ing $80M mile­stone; Caris rais­es $310M for ge­nom­ic pro­fil­ing ex­pan­sion

Novartis reported that Zolgensma earned $291 million in Q3, which puts the SMA gene therapy squarely in the blockbuster category. That, in turn, is paying benefits to Regenxbio, which provided the NAV AAV9 vector used by Zolgensma.

The biotech $RGNX reported Tuesday morning that it is getting an $80 million milestone payment from Novartis, which now has earned more than $1 billion cumulatively from the new gene therapy franchise. And with large percentages of newborns now being screened for the rare, lethal ailment, the franchise can continue to grow.

Cedric Francois, Apellis CEO (Optum via YouTube)

UP­DAT­ED: So­bi bets $250M cash, about $1B in mile­stones for rights to a C3 ther­a­py be­ing pushed through 5 piv­otal tri­als

A couple years after licensing Novimmune’s emapalumab and turning around a quick FDA OK, Stockholm-based Sobi is betting up to $1.2 billion for rights to another rare disease drug.

The company is shelling out $250 million upfront and adding up to $915 million in milestones for rights to develop and commercialize Apellis Pharmaceuticals’ drug pegcetacoplan outside the US. Together, the companies will see the systemic C3 therapy through five registrational trials in hematology, nephrology and neurology.

Christian Rommel (via Roche)

Bay­er fol­lows R&D deal spree by raid­ing Roche's can­cer group for its new re­search chief

The day after Bayer signed off on a $4 billion deal designed to put the company among the leaders in gene therapy development, the pharma giant has recruited a new chief for its R&D division. And they opted for an expert in the cancer field.

Christian Rommel, Roche’s head of discovery and early-stage oncology development, has been tapped to take over the job. Joerg Moeller, who got the top research post after early and late-stage development roles were combined 2 years ago, is hitting the exit “to pursue other career opportunities.”

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Albert Bourla, AP

UP­DAT­ED: Where's the Pfiz­er ef­fi­ca­cy read­out? CEO Bourla says 'soon,' but you're go­ing to have to wait for it

Pfizer CEO Albert Bourla had promised repeatedly that the pharma giant would know if its leading Covid-19 vaccine is effective by the end of this month — now just a few days away.

Instead, the company reported early Tuesday that it has yet to conduct any interim efficacy analyses. And it won’t now until sometime next month.

The news was included in a slide for their Q3 report.

In the morning Q3 call with analysts, Bourla says that they expect efficacy data “soon,” but noted that they wouldn’t be able to say anything until all the administrative work was done on the interim, which would take about a week. And he added that Pfizer isn’t going to say anything else about that hot topic until they have the data in hand.

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Charles Baum, Mirati CEO

UP­DAT­ED: Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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Yung Chyung, Scholar Rock CMO (Business Wire)

A dark horse en­trant in­to the spinal mus­cu­lar at­ro­phy field dou­bles its val­ue on some PhII da­ta

The last four years have seen a sudden explosion in treatments for spinal muscular atrophy, a neurodegenerative condition that once led patients — often young ones — with a grim prognosis and no options. The prognosis still isn’t rosy, but now there are three FDA-approved options, enough to make the choice of one difficult.

Now a fourth potential option has entered the mix. Today, Scholar Rock announced the results from a proof-of-concept testing their SMA drug by itself and in combination with Ionis’ Spinraza, showing that all patient cohorts improved on standard scales used for measuring motor function in people with SMA.

Kevin Conroy (Exact Sciences)

UP­DAT­ED: A month af­ter Il­lu­mi­na's big Grail buy­out, Ex­act Sci­ences scoops up liq­uid biop­sy ri­val Thrive for a rel­a­tive bar­gain

Illumina is going to have a lot of work to do to prove Grail was worth those $8 billion.

Today, Exact Sciences announced that it will acquire Thrive, Grail’s chief rival among the early cancer detection startups, for a sizeable but relatively moderate $2.15 billion. The yawning gap in part reflects the vast differences in capital that have been invested to date in each company.  But both have gone toe-to-toe over the last year and a half, with Grail having published data in over 50 cancers but Thrive recently beating them to a key test for liquid biopsy companies.