FDA re­buffs lit­tle As­ser­tio Ther­a­peu­tic­s' long-act­ing ACTH for­mu­la­tion, shares sink

Tiny As­ser­tio Ther­a­peu­tics’ shares plunged pre-mar­ket on Tues­day, af­ter the FDA has spurned its man-made ver­sion of the hor­mone ACTH, which was be­ing re­viewed as a di­ag­nos­tic for pa­tients pre­sumed to have adreno­cor­ti­cal in­suf­fi­cien­cy.

The Lake For­est, Illi­nois-based drug­mak­er said its de­vel­op­ment part­ner West Ther­a­peu­tic De­vel­op­ment had re­ceived a com­plete re­sponse let­ter from the US reg­u­la­tor, which in­di­cat­ed that that cer­tain “phar­ma­co­dy­nam­ic pa­ra­me­ters were not ad­e­quate­ly achieved” for the prod­uct.

As­ser­tio’s shares $AS­RT were down more than 28% at 84 cents pre-mar­ket on Tues­day.

The prod­uct is an in­jectable for­mu­la­tion of long-act­ing cosyn­tropin (syn­thet­ic adreno­cor­ti­cotrop­ic hor­mone, or ACTH). The ACTH stim­u­la­tion test is the most spe­cif­ic test for di­ag­nos­ing adren­al in­suf­fi­cien­cy. Blood cor­ti­sol lev­els are mea­sured be­fore and af­ter a syn­thet­ic form of ACTH is giv­en by in­jec­tion. The nor­mal re­sponse af­ter the in­jec­tion is a rise in blood cor­ti­sol lev­els. Pa­tients with adren­al in­suf­fi­cien­cy see lit­tle or no in­crease in cor­ti­sol lev­els.

Adren­al in­suf­fi­cien­cy, in­clud­ing Ad­di­son’s dis­ease, is a dis­or­der that oc­curs when the adren­al glands don’t pro­duce ad­e­quate lev­els of cer­tain hor­mones, in­clud­ing cor­ti­sol.

As­ser­tio, which re­cent­ly wooed for­mer As­traZeneca ex­ec­u­tive David Whead­on to its board, al­so has a painkiller un­der FDA re­view and an acute mi­graine ther­a­py in de­vel­op­ment.

The com­pa­ny does not de­vel­op new chem­i­cal en­ti­ties, but rather its tech­nol­o­gy is de­signed to al­low oral drugs to be re­tained in the stom­ach for a pe­ri­od that eclipses the im­me­di­ate-re­lease and some ex­tend­ed-re­lease for­mu­la­tions that cur­rent­ly ex­ist — there­by the­o­ret­i­cal­ly al­low­ing for greater ef­fi­ca­cy, in­creased tol­er­a­bil­i­ty, and the con­ve­nience of once- or twice-dai­ly dos­ing. It has three FDA ap­proved prod­ucts for neu­ro­log­i­cal con­di­tions.

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 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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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.)

Safe­ty qualms in MyoKar­dia mid-stage study cause in­vestors to pause

Ahead of the pivotal data readout of MyoKardia’s lead drug, mavacamten, in patients with obstructive hypertrophic cardiomyopathy (HCM), investors took issue with the drug’s safety profile in a separate Phase II non-obstructive HCM study.

Mavacamten is expected to break new ground in heart disease — a field monopolized by large pharmaceutical companies largely due to the long, arduous and expensive trials that are commonplace in heart drug development. Unlike other developers focusing on common heart disorders, MyoKardia’s treatment is also targeting a so-far untapped disease — hypertrophic cardiomyopathy — a genetic condition in which heart muscle thickens, making it harder for the organ to pump blood to the rest of the body.

The lat­est Cin­derel­la sto­ry in on­col­o­gy ends with a sud­den rout as up­dat­ed da­ta dis­play spooks in­vestors

NextCure’s turn as the Cinderella of cancer-focused biotechs was short-lived.
Just a few days after its shares $NXTC zoomed up more than 250% on some very early stage results in a SITC abstract, a more complete analysis over the weekend spiked the hype and left investors in high dudgeon as the stock price collapsed back towards earth Monday.
The focus at NextCure is centered on NC318, an antibody that is intended to shut down the immunosuppressive Siglec-15 — or S-15 — target. After adding a small group of patients to the readout, investigators circled 2 clinical responses, a complete and partial response, along with 4 stable disease cases in non-small cell lung cancer.

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Te­va spin­out rais­es $85M in IPO; No­var­tis beefs up gener­ics unit with $440M deal

→ After Teva spinout 89bio recently announced that its IPO was being held up, the company is back in the game offering 5,304,687 shares at a price of $16 per share. The company has raised $84.9 million IPO in gross proceeds and will be listed under the ticker symbol $ETNB. BofA Securities, SVB Leerink and RBC Capital Markets are the joint book-running managers for the offering. Oppenheimer & Co is the co-manager for the offering.
→ Looking to amp up its presence in Japan’s hospitals, Novartis has struck a deal to buy out Aspen’s portfolio of generics in the world’s third largest healthcare market. The pharma giant is paying $440 million for Aspen’s Japanese subsidiary.
→ Novartis said tropifexor, a non-bile acid FXR agonist, has scored on several key biomarkers of NASH in a Phase IIb trial, including reductions in hepatic fat, alanine aminotransferase and body weight compared to a placebo at 12 weeks.