FDA ap­proves drug for rare con­di­tion that trig­gers in­tense pain from light ex­po­sure

Five years af­ter EU reg­u­la­tors ap­proved a ther­a­py to treat a rare in­her­it­ed meta­bol­ic con­di­tion that caus­es ex­treme sen­si­tiv­i­ty to light, the FDA has en­dorsed the treat­ment — mark­ing the first-ever ap­proval for pa­tients with ery­thro­poi­et­ic pro­to­por­phyr­ia (EPP) in the Unit­ed States.

The ther­a­py, which is ad­min­is­tered via an im­plant in­sert­ed sub­cu­ta­neous­ly, was de­vel­oped by Aus­tralian drug mak­er Clin­u­v­el Phar­ma­ceu­ti­cals and is sold as Sce­nesse.

In pa­tients with EPP, the ac­tiv­i­ty of fer­rochelatase — an en­zyme in­volved in the pro­duc­tion of a key com­po­nent in he­mo­glo­bin — is com­pro­mised. Re­duced fer­rochelatase ac­tiv­i­ty caus­es the ac­cu­mu­la­tion of pro­to­por­phyrin IX (PPIX) — and light ex­po­sure on the skin can re­act with PPIX, spark­ing in­tense pain, red­ness and thick­en­ing.

Sce­nesse, known chem­i­cal­ly as afame­lan­otide, binds with the melanocortin-1 re­cep­tor on the sur­face of melanocyte cells found in the epi­der­mis of the skin. This trig­gers a cas­cade of re­ac­tions that re­sult in the melanocyte fa­vor­ing the pro­duc­tion of eu­me­lanin (black/brown) over pheome­lanin (red/yel­low). The pro­duc­tion of eu­me­lanin in the skin in­de­pen­dent of ex­po­sure to sun­light or ar­ti­fi­cial light sources of­fers the pa­tient the abil­i­ty to cope with the mol­e­c­u­lar dam­age caused by light.

In Jan­u­ary, STAT pro­filed a Con­necti­cut woman who com­pared the pain she felt from light ex­po­sure to the ex­pe­ri­ence of hav­ing boil­ing oil poured on her hands and knees. On­ly around 600 Amer­i­cans suf­fer from EPP. Switzer­land and Italy had Clin­u­v­el agree to pay 80% of the drug’s cost be­fore al­low­ing it, ac­cord­ing to STAT.

“We are de­ter­min­ing our pric­ing strat­e­gy (in the Unit­ed States) but our ap­proach is go­ing to be far more trans­par­ent and fair than many of our peers. For this we re­fer you to our ap­proach to pric­ing in Eu­rope,” a Clin­u­v­el spokesper­son told End­points News.

Sce­nesse is priced in Eu­rope at €14,101 per in­jectable im­plant, or €56,404 per pa­tient based on four in­jectable im­plants per an­num (ex­clud­ing lo­cal tax­es), the spokesper­son added.

The US ap­proval for Sce­nesse was based on twin place­bo-con­trolled clin­i­cal tri­als. In the first 93-pa­tient tri­al, 48 sub­jects were giv­en Sce­nesse, while the rest were on place­bo.  Af­ter 180 days of ex­po­sure to di­rect sun­light be­tween 10 am and 6 pm, pa­tients on the ther­a­py were pain-free for a me­di­an 64 hours, ver­sus 41 hours for those in the place­bo arm.

In the oth­er 74-pa­tient study, 38 re­ceived Sce­nesse, while the rest got place­bo. Pa­tients were fol­lowed for 270 days which they spent out­doors be­tween 10 am and 3 pm and were as­sessed for pain lev­els — al­though the analy­sis did not in­clude sun ex­po­sure on days pa­tients re­port­ed spend­ing time in a com­bi­na­tion of both di­rect sun­light and shade. The me­di­an to­tal num­ber of hours un­der these con­di­tions in which pa­tients felt ‘no pain’ was six hours for pa­tients re­ceiv­ing Sce­nesse and 0.75 hours for pa­tients re­ceiv­ing place­bo.

Clin­u­v­el formed in 1987 af­ter re­searchers start­ed work­ing on the idea of syn­the­siz­ing hu­man hor­mones to pro­tect the skin.

Nick Galakatos, Blackstone global head of life sciences

Nick Galakatos and the Black­stone team now have a record $4.6B to in­vest in bio­phar­ma, with a big fo­cus on push­ing com­pa­nies over the top

Nick Galakatos and his team at Blackstone Life Sciences have seen their biggest opportunities swell up in mostly established players who don’t have all the money they need to accomplish everything on the to-do list. And right now, with the industry booming, that’s a long list with some hefty needs.

The Blackstone team has neatly tied up the largest private fund ever raised in life sciences for making big dreams come true in biopharma. Late Thursday, Blackstone put out word that they had closed their highly anticipated fund with the projected $4.6 billion all in.

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UP­DAT­ED: Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Gilead boasts of pos­i­tive remde­sivir da­ta on mor­tal­i­ty — but their analy­sis pro­vokes the skep­tics

Gilead is surging again off data that suggest its antiviral remdesivir might improve survival.

The new data come from an analysis Gilead conducted comparing the death rate and recovery time of patients in one of its remdesivir trials to a group of 800 patients “with similar baseline characteristics and disease severity” who received only standard-of-care around the same time. The result, they said, suggested that patients who received remdesivir had a 62% better chance at surviving than those who did not.

Hal Barron, GSK

Win or lose on the mar­ket­ing OK, the FDA just gunned down GSK’s bright hopes for their BC­MA ther­a­py

The FDA’s ODAC — the Oncologic Drugs Advisory Committee — has a well-known bias in favor of adding new cancer drugs to the market, even if efficacy is at best marginal and serious safety issues demand careful management.

Doctors want as many arrows in their quiver as they can get. And when patients are dying after failing multiple drugs, why not give it a go one more time?

GlaxoSmithKline, though, is about to test out how their new BCMA antibody drug conjugate belantamab mafodotin can do after being mauled in an in-house FDA review, ahead of the Tuesday expert panel discussion. Even if the agency goes ahead with an expected green light, this drug will likely be constrained to a small niche — icing any plans they may have for making waves in oncology anytime soon.

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Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Covid-19 roundup: BioN­Tech go­ing head-to-head with Mod­er­na as PhI­II mR­NA launch looms; Tri­al on Shin­zo Abe’s once-fa­vorite an­tivi­ral is in­con­clu­sive

It’s a race to the Phase III finish line now for the 2 leading mRNA vaccines in the pipeline for Covid-19.

BioNTech chief Ugur Sahin told the Wall Street Journal that his company will start Phase III testing of their vaccine later this month, setting them up to lateral the data to regulators before the end of this year.

That puts them essentially on the exact same schedule as Moderna is dedicated to. The Massachusetts rival to BioNTech also expects to launch Phase III this month. Lots of rumors have circulated about delays and conflict among the scientists advancing the Moderna jab, but the biotech has consistently stuck to its plan to start a late-stage pivotal this month.

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Andrew Kruegel, Kures president and co-founder (Columbia Tech Ventures via Vimeo)

Af­ter psilo­cy­bin and ke­t­a­mine, a new biotech comes along de­vel­op­ing a drug Scott Got­tlieb fought

Andrew Kruegel was six years into his chemistry work at Columbia University, when, one day in August 2016, he learned he might have only 30 days before the government made him destroy his research.

Kruegel had been studying kratom, a leaf long used in Southeast Asia as a stimulant or for pain. It had opioid-like properties, he found, but seemed to offer pain relief without the addictive potential or respiratory side effects of traditional opioids — a riddle that might help illuminate how human opioid receptors work.

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The home run count: The $100M+ mega-round boom in biotech in­spired a $7.3B feed­ing fren­zy — so far this year

Over the last 6 months there’s been a blizzard of money piling up drifts of the green stuff through the biotech landscape. And the forecast calls for more cash windfalls ahead.

Even as a global pandemic has killed more than half a million people, blighted economies and divided nations over the proper response, it’s also helped ignite an unprecedented burst of big-time investing. And not just in Covid-19 deals, as we’ve looked at before.

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Atul Deshpande, Harbour BioMed chief strategy officer & head, US operations (Harbour BioMed)

An­oth­er biotech IPO set-up? Multi­na­tion­al biotech leaps from round to round, scoop­ing up cash at a blis­ter­ing pace

A short four months after announcing a $75 million haul in Series B+ fundraising, the multinational biotech Harbour BioMed pulled in another round of investments and eclipsed the nine-digit mark in the process.

Harbour completed its Series C financing, the company announced Thursday morning, raising $102.8 million and bringing its total investment sum to over $300 million since its founding in late 2016. The biotech plans to use the money to transition early-stage candidates from the discovery phase, fund candidates already in the clinic, and prep late-stage candidates for commercialization.

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