William Heiden, AMAG

Cred­i­bil­i­ty shred­ded, AM­AG jet­ti­sons CEO and 2 drugs as it looks to re­or­ga­nize around a sal­vage plan

With its rev­enue num­bers in a tail­spin and a top drug un­der crit­i­cal re­view at the FDA fol­low­ing a failed con­fir­ma­to­ry study, AM­AG is start­ing the new year with a new plan.

Out go two drugs for women’s health: the con­tro­ver­sial Vyleesi ap­proved last year to stim­u­late women’s sex­u­al de­sire and In­trarosa for the re­lief of pain dur­ing sex. The biotech $AM­AG plans to auc­tion off those drugs to any­one game to try to mar­ket them. Al­so out: CEO William Hei­den, once they find a suc­ces­sor for the helm.

Hei­den is fol­low­ing in the re­treat­ing foot­steps of 110 sales staffers who were cut last year dur­ing an ear­li­er re­or­ga­ni­za­tion.

The CEO had this to say in a pre­pared state­ment:

We con­tin­ue to be­lieve in the sig­nif­i­cant long-term po­ten­tial of In­trarosa and Vyleesi. How­ev­er, the un­cer­tain­ty around the long-term dura­bil­i­ty of Mak­e­na rev­enues makes it chal­leng­ing to in­vest in both our promis­ing pipeline and in the physi­cian and con­sumer mar­ket­ing re­quired to sup­port these two new prod­ucts.

AM­AG has been un­der con­sid­er­able scruti­ny since a ma­jor­i­ty of FDA ad­vis­ers vot­ed to sug­gest that the agency should yank their ap­proval for Mak­e­na. The drug flopped bad­ly in the con­fir­ma­to­ry study the FDA had de­mand­ed as it pro­vid­ed an ac­cel­er­at­ed OK on the drug — way back in 2011.

Now per­suad­ing the FDA to keep the preterm birth drug Mak­e­na on the mar­ket is a top pri­or­i­ty, along with the de­vel­op­ment of cira­paran­tag and AM­AG-423 while “dri­ving the con­tin­ued growth of Fer­a­heme, which funds our two pipeline as­sets.”

An­a­lysts re­mained skep­ti­cal af­ter the an­nounce­ment Thurs­day.

Eun Yang at Jef­feries says sell­ing off the two un­der-per­form­ing as­sets made sense, but didn’t see any quick up­side in terms of rev­enue. The biotech has lost cred­i­bil­i­ty in re­cent years.

Note that in ear­ly-2017, when AM­AG ac­quired Vyleesi ($60M up­front) & In­trarosa ($50M in cash + $13.5M in stock up­front), the Street was gen­er­al­ly skep­ti­cal of the com­mer­cial po­ten­tial and the shares sold off >30% dur­ing that pe­ri­od. From 2016-2019, to­tal rev­enues de­clined by ~40% to ~$325M (our es­ti­mate) & non-GAAP OpEx in­creased by 37% to ~$387M (by our es­ti­mate).

The new CEO is go­ing to have quite a chal­lenge win­ning an­a­lysts back.

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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Ku­ra co-founder heads to Asian mul­ti-na­tion­al as biotech eyes the goal posts for lead drug

Six years after Kura Oncology snagged a farnesyl transferase inhibitor from J&J and leapt straight into clinical development, one of the biotech’s founders is leaving to start a new chapter in his career.

CMO and development chief Antonio Gualberto is exiting the company, and Kura — led by longtime biotech entrepreneur Troy Wilson — is on the hunt for a replacement. Wilson credited the CMO for some key biomarker work, including the discovery of the CXCL12 pathway as a target of their lead drug tipifarnib. Those biomarkers are being relied on to define the patient population most likely to benefit from the drug.

FDA waves Epizyme's $186K rare can­cer drug through to mar­ket — now get ready for the sec­ond act

After winning the hearts of the expert panel convened by the FDA despite a bleak in-house review and a checkered development history, Robert Bazemore has steered Epizyme to its first-ever OK for a rare cancer drug.

The approval in epithelioid sarcoma sets tazemetostat, now Tazverik, up nicely for a quick expansion to follicular lymphoma — a much bigger indication for which the biotech has just submitted an NDA.

UP­DAT­ED: Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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2019 a 'trans­for­ma­tive year' for phar­ma M&A. Is that a good thing?

Big Pharma keeps getting bigger.

Fueled by the mega-mergers between Bristol-Myers Squibb and Celgene and between Allergan and AbbVie, the industry last year saw $350 billion worth of M&A, according to the new year-end report from the consultants at PwC.  That’s a more than 50% increase on 2018.

“I kind of look at 2019 as a transformational year,” report author Glen Hunzinger told Endpoints News. 

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Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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