Sarep­ta CEO dis­putes FDA re­venge nar­ra­tive; Take­da posts long-term da­ta on key Shire drug

Sarep­ta hasn’t been hav­ing its best days with the FDA, but the biotech is keen to stay on the agency’s good side. Push­ing against a per­cep­tion that its sec­ond Duchenne mus­cu­lar dy­s­tro­phy ther­a­py, Vyondys 53, was re­ject­ed in an act of reg­u­la­to­ry re­venge, CEO Doug In­gram had this to say: “To those may be­lieve that the CRL sug­gest some sort of bias on be­half of the di­vi­sion of neu­rol­o­gy to­wards Sarep­ta, I would un­equiv­o­cal­ly and em­phat­i­cal­ly dis­agree. Let me re­it­er­ate that I re­main con­vinced that we were treat­ed very fair­ly and pro­fes­sion­al­ly by the di­vi­sion of neu­rol­o­gy.”

Take­da has un­veiled new long-term da­ta on Takhzy­ro, the hered­i­tary an­gioede­ma drug ac­quired from Shire. Da­ta from the Phase III HELP study, in­volv­ing pa­tients 12 years old or above, sug­gest­ed that the drug con­tin­ues to pre­vent HAE at­tacks “at a rate sim­i­lar to that ob­served in the piv­otal HELP Study, in pa­tients who re­ceived treat­ment for a mean du­ra­tion of 19.7 (0-26.1) months.”

Ab­b­Vie has sub­mit­ted an sN­DA to the FDA for Im­bru­vi­ca in com­bi­na­tion with rit­ux­imab for the first-line treat­ment of pre­vi­ous­ly un­treat­ed pa­tients (70 years old or younger) with chron­ic lym­pho­cyt­ic leukemia (CLL) or small lym­pho­cyt­ic lym­phoma (SLL). In the Phase III E1912 study showed, the drug im­proved both PFS and OS com­pared to rit­ux­imab plus chemother­a­py.

→ Trans-Pa­cif­ic biotech Phanes has teamed up with Fo­s­un Kite Biotech­nol­o­gy, the cell ther­a­py joint ven­ture launched by Fo­s­un and Kite Phar­ma, to boost their CAR-T. Un­der the agree­ment, Phanes will pro­vide a col­lec­tion of hu­man­ized an­ti­bod­ies tar­get­ing two can­cer anti­gens to arm CAR-T prod­ucts for sol­id tu­mor treat­ments in Chi­na (in­clud­ing Hong Kong SAR, Macau SAR and Tai­wan). Phanes is el­i­gi­ble for un­spec­i­fied up­front and mile­stones in ad­di­tion to roy­al­ties, while Fo­s­un Kite owns the right of first ne­go­ti­a­tion for the use of these an­ti­bod­ies out­side of Chi­na.

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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FDA Vas­cepa re­view spot­lights new safe­ty sig­nals, pos­si­ble min­er­al oil spoil­er as Amarin hunts a block­buster ap­proval

An in-house FDA review of Amarin’s Vascepa raises a set of hurdles the biotech will have to clear if the biotech expects to get the long-awaited FDA approval that could set it on a path to superstar status. But it appears that Amarin has survived another potential setback without introducing a major new threat to its prospects.
The stakes don’t get much higher, with analysts saying a win this week for Amarin could lead to billions in new sales — provided the agency stamps it with an OK. And investors liked what they say in the FDA review this morning, bumping the stock $AMRN 17%.
The insider take at the agency includes a note on two new safety signals seen in the big cardio outcomes study of the omega-3 fatty acid drug that shocked many analysts with a solid set of efficacy data. There’s a key concern over whether the use of mineral oil in the placebo skewed LDL levels in such a way that tilted the data in Amarin’s favor.
The FDA overview was written by John Sharretts, the acting deputy director in the Division of Metabolism and Endocrinology Products. 
On the safety side, the internal review focused on a 3.1% versus 2.1% rate of adjudicated events of atrial fibrillation or atrial flutter requiring hospitalization. But they also say a-fib shouldn’t confound the benefit-safety of the drug — given the improvement on MACE — or prevent its use. And then there was also a higher rate of bleeding events in the drug arm.

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FDA puts Sol­id Bio’s lead gene ther­a­py pro­gram on hold — again — af­ter an­oth­er pa­tient is hurt by SGT-001

Solid Biosciences continues to be plagued by safety issues.

Close to 18 months after the gene therapy biotech was able to quickly shed an FDA hold on their lead Duchenne muscular dystrophy program for SGT-001, regulators have stepped back in to force another halt after another patient was hit hard by a set of serious adverse events remarkably similar to the first set.

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Bill Haney, Skyhawk

Cel­gene ex­ecs shell out $92M cash for a pair of R&D deals that will fit per­fect­ly in their new home at Bris­tol-My­ers

With Bristol-Myers Squibb’s Celgene buyout all but complete, the BD teams are working in perfect synchrony now. The Celgene side is going back to Skyhawk, a darling of the crowd that set out to drug RNA, and they’re adding a suite of new programs that mesh perfectly with the new regime in charge.

Celgene is shelling out $80 million in a cash upfront to add oncology, immuno-oncology and autoimmune diseases to the initial roundup of neurological targets mapped early in Skyhawk’s existence.

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Reata's bar­dox­olone of­fers promise in pa­tients with rare kid­ney dis­or­der

After surprising Wall Street with positive data on its drug, omaveloxolone, in patients suffering from a notoriously hard-to-treat degenerative neuromuscular disorder last month, Reata Pharma on Monday unveiled pivotal results from a trial testing another drug, bardoxolone, in patients with a rare, genetic form of chronic kidney disease for which there exist no approved therapies.

Bardoxolone, like Reata’s other lead drug — omaveloxolone — is a small molecule engineered to bind to a gene called Keap1 to enhance the activity of the protein Nrf2 in order to defuse inflammation.

Kad­mon wax­es rhap­sod­ic on cGVHD re­sults as race with Jakafi heats up

A year ago, Kadmon piqued cautious interest and sent its stock up 20% when it announced positive results from a tiny proof-of-concept study on a new, chronic graft-versus-host-disease treatment. Now interim results are out on the pivotal, and not just the biotech’s executives are gushing about it.

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ARCH-backed Chi­nese biotech scores PhI­II win for glu­cok­i­nase-mod­u­lat­ing di­a­betes drug

When ARCH Venture Partners helped launch Hua Medicine with former Roche exec Li Chen, it marked one of the first bets on a Chinese biotech to deliver a world-class diabetes drug. Eight years later, their lead drug appears to have turned the corner and entered the final stretch.

Late on Monday night Shanghai time — where it’s based — Hua unveiled positive data from the first part of its pivotal study of dorzagliatin, celebrating a win on the primary endpoint as well as decent safety results.

US Capitol (Arsalan Arif for Endpoints News)

Vot­ers say Con­gress needs to curb drug prices. So why do the odds look grim for ma­jor leg­is­la­tion this year?

House Democrats are poised to pass sweeping legislation to lower drug prices using strategies President Donald Trump has endorsed. A Trump aide urged the Republican-controlled Senate to vote on a different package curbing drug prices that was drafted by a senior Republican.

But at least right now, neither measure appears likely to attract enough bipartisan support to become law.

Nearly 8 in 10 Americans say the cost of prescription drugs is unreasonable, with voters from both parties agreeing that reducing the cost of prescription drugs should be one of Congress’ top priorities, according to a poll last month by the Kaiser Family Foundation. (KHN is an editorially independent program of the foundation.)