Ipsen re­cruits Steven Hilde­mann as CMO; Eli Lil­ly of­fers two more low-cost in­sulin op­tions

→ Af­ter los­ing their CEO last month, Ipsen — who’s grap­pling with an FDA hold af­ter safe­ty con­cerns emerged from late-stage stud­ies of its lead rare dis­ease drug palo­varotenehas tapped for­mer Mer­ck Serono ex­ec Steven Hilde­mann as EVP, chief med­ical of­fi­cer, head of glob­al med­ical af­fairs and phar­ma­covig­i­lance. Dur­ing his time at Mer­ck, Hilde­mann served as CMO and SVP and head of glob­al med­ical af­fairs and pa­tient safe­ty. His pre­vi­ous ex­pe­ri­ence spans Mer­ck/Scher­ing Plough, Am­gen and Pfiz­er/Phar­ma­cia.

→ In a new at­tempt to ad­dress crit­i­cism on the high cost of in­sulin, Eli Lil­ly is rolling out two ad­di­tion­al cost-sav­ing op­tions to Hu­ma­log Mix75/25 KwikPen (in­sulin lispro pro­t­a­mine and in­sulin lispro in­jectable sus­pen­sion 100 units/mL) and Hu­ma­log Ju­nior KwikPen (in­sulin lispro in­jec­tion 100 units/mL). The list prices of both will be 50% low­er com­pared to the brand­ed ver­sions and will be avail­able by mid-April. The com­pa­ny of­fered its first half-price al­ter­na­tive to Hu­ma­log, In­sulin Lispro in­jec­tion (100 units/mL), in May 2019, though sen­a­tors have since slammed Lil­ly for not liv­ing up to its promise as the in­jec­tion is still not wide­ly avail­able (an ac­cu­sa­tion that CEO Dave Ricks called “non­sense“).

→ With ri­vals gun­ning at their star block­buster Soliris, Alex­ion is fo­cus­ing on ini­ti­at­ing a Phase III study of their fol­low-on drug Ul­tomiris (ravulizum­ab) in amy­otroph­ic lat­er­al scle­ro­sis. CHAM­PI­ON-ALS will be a 50-week glob­al study with ap­prox­i­mate­ly 350 adult pa­tients. The pri­ma­ry end­point of the study, which the biote­hc ex­pects to launch this quar­ter, will be change in the ALS func­tion­al rat­ing scale-re­vised (ALS­FRS-R) score.

→ Pfiz­er is team­ing up with Alex Zha­voronkov’s AI-shop In­sil­i­co Med­i­cine to uti­lize In­sil­i­co’s tech and pan­domics dis­cov­ery plat­form. The goal of the col­lab­o­ra­tion is to iden­ti­fy re­al-world ev­i­dence for po­ten­tial ther­a­peu­tic tar­gets im­pli­cat­ed in a va­ri­ety of dis­eases.

→ DNA se­quenc­ing gi­ant Il­lu­mi­na — who just tore up a $1.2 bil­lion buy­out of Pa­cif­ic Bio­sciences due to FTC “mo­nop­o­list” ac­cu­sa­tions — has forged a 15 year, non-ex­clu­sive col­lab­o­ra­tion agree­ment with Roche. The deal will help “ac­cel­er­ate the avail­abil­i­ty of dis­trib­utable NGS-based in-vit­ro di­ag­nos­tic (IVD) tests on Il­lu­mi­na’s di­ag­nos­tic (Dx) se­quenc­ing sys­tems.”

Oys­ter Point Phar­ma has rolled out pos­i­tive da­ta from its Phase II MYS­TIC study in test­ing their nasal spray against dry eye dis­ease. The re­sults showed a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in Schirmer’s score from base­line at Day 84 in both dos­es as com­pared to the con­trol group. The study demon­strat­ed that OC-01 was well-tol­er­at­ed at the two dos­es test­ed and there were no re­ports of se­ri­ous treat­ment-emer­gent ad­verse events.

Adimab is ex­tend­ing its li­cense agree­ment with No­vo Nordisk that it start­ed in 2013. Un­der the terms of agree­ment, the Dan­ish drug­mak­er will have con­tin­ued ac­cess to a hu­man an­ti­body li­brary and will re­ceive a li­cense to the Adimab plat­form for use on an un­lim­it­ed num­ber of tar­gets. Adimab is re­ceiv­ing an undis­closed ex­ten­sion fee as well as de­vel­op­ment and sales mile­stones plus tiered roy­al­ties on the an­nu­al net sales.

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.

Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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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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Gilead dusts off a failed Ebo­la drug as coro­n­avirus spreads; Ex­elix­is boasts pos­i­tive Ph I/II da­ta

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

Alex Karnal (Deerfield)

Deer­field vaults to the top of cell and gene ther­a­py CD­MO game with $1.1B fa­cil­i­ty at Philadel­phi­a's newest bio­phar­ma hub

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.

Tri­fec­ta of sick­le cell dis­ease ther­a­pies ex­tend life ex­pectan­cy, but are not cost-ef­fec­tive — ICER

Different therapeutic traits brandished by the three approved therapies for sickle cell disease all extend life expectancy, but their impact on quality of life is uncertain and their long-term cost-effectiveness is not up to scratch according to the thresholds considered reasonable by ICER, the non-profit concluded in a draft guidance report on Thursday.

Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.

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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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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