As­traZeneca, Mer­ck score Chi­na OK for Lyn­parza; Re­gen­eron spot­lights mid-stage da­ta for rare dis­ease drug

→ As they con­tin­ue to scoop up wins for their PARP jug­ger­naut Lyn­parza, As­traZeneca — along with their part­ners at Mer­ck — have scored ap­proval in Chi­na as a first-line main­te­nance treat­ment for a form of ovar­i­an can­cer. Reg­u­la­tors based their de­ci­sion on re­sults from the SO­LO-1 tri­al, in which the drug low­ered the risk of dis­ease pro­gres­sion or death by 70% when com­pared to place­bo. It marks a ma­jor ad­di­tion to the list of 65 coun­tries in which Lyn­parza is ap­proved to treat plat­inum-sen­si­tive re­lapsed ovar­i­an can­cer, the com­pa­nies said.

Re­gen­eron re­leased pos­i­tive ear­ly da­ta from a small, Phase II tri­al on a rare blood dis­or­der. Six pa­tients were giv­en Re­gen­eron’s an­ti-C5 an­ti­body poze­limab for parox­ys­mal noc­tur­nal he­mo­glo­bin­uria, or PNS, a chron­ic and life-threat­en­ing con­di­tion where red blood cells rou­tine­ly rup­ture and spill their con­tents in­to the blood, caus­ing fa­tigue, short­ness of breath and blood clots. The pa­tients giv­en a week­ly 800 mg sub­cu­ta­neous in­jec­tion of the an­ti­body saw their lev­els of lac­tate de­hy­dro­ge­nase — an en­zyme that falls in­to the blood when red blood cells break — re­turn to nor­mal.

Glax­o­SmithK­line’s HIV-fo­cused sub­sidiary Vi­iV has com­plet­ed its pitch to the FDA for what it hopes to be the lat­est ad­di­tion to an ar­se­nal of an­ti­retro­vi­ral agents. The drug, fos­tem­savir, is a pro­drug of tem­savir and works by bind­ing di­rect­ly to the sur­face of the virus. In a Phase III tri­al in­volv­ing heav­i­ly pre-treat­ed pa­tients with mul­tidrug re­sis­tance, fos­tem­savir per­formed bet­ter than place­bo as mea­sured by a de­cline in HIV-1 RNA.

→ For its next for­ay in­to AI-pow­ered drug dis­cov­ery, As­traZeneca has turned to Gate­house Bio — a port­fo­lio com­pa­ny in its Boston Bio Hub in­cu­ba­tor har­ness­ing small RNA sig­na­tures to find mol­e­c­u­lar path­ways as­so­ci­at­ed with dis­ease. The start­up will ap­ply its plat­form to iden­ti­fy new tar­gets for res­pi­ra­to­ry and car­dio­vas­cu­lar dis­eases.

Back in May, mi­cro­bio­me up­start Vedan­ta Bio­sciences got an ex­tra $18.5 mil­lion in the bank as part of a $45.5 mil­lion Se­ries C round fi­nanc­ing. To­day, the biotech se­cured more cash through a re­search grant from CARB-X of $5.8 mil­lion — the sec­ond it’s re­ceived from the non-prof­it. In ad­di­tion, the com­pa­ny is el­i­gi­ble for $3.5 mil­lion in fur­ther fund­ing up­on com­ple­tion of spe­cif­ic mile­stones. The new in­fu­sion will help push VE707 — Vedan­ta’s pre­clin­i­cal hu­man mi­cro­bio­me-de­rived dis­cov­ery pro­gram de­signed to pre­vent in­fec­tion and col­o­niza­tion of mul­ti-drug re­sis­tant in­fec­tions by restor­ing a healthy mi­cro­bio­ta.

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.

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.

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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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.

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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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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