Far from the big hubs, Apel­lis is steer­ing its ri­val to Alex­ion's Soliris in­to a PhI­II pro­gram

A Ken­tucky biotech says they’re lay­ing the foun­da­tion for a piv­otal pro­gram for their C3 in­hi­bi­tion ther­a­py, which ex­ecs be­lieve can re­place Soliris in treat­ing PNH.

Louisville-based Apel­lis has been mak­ing progress on its lead drug far away from the spot­light that con­cen­trates at­ten­tion on the big biotech hubs. But it’s been well fund­ed, with a $47 mil­lion D round that dropped ear­ly last year af­ter they gave up on an IPO in chilly mar­ket wa­ters. And the com­pa­ny says they’ve been nail­ing down hard hu­man ev­i­dence that by mov­ing up­stream from C5 in­hi­bi­tion, where Soliris hits, they can do a bet­ter job in con­trol­ling ane­mia and trans­fu­sion de­pen­dence among pa­tients with this ex­treme­ly rare con­di­tion.

This week, Apel­lis is re­port­ing on two tiny stud­ies of 3 and 6 pa­tients. In 3 pa­tients nev­er treat­ed with Soliris, in­ves­ti­ga­tors re­port­ed that all of them ex­pe­ri­enced a quick cor­rec­tion on a key bio­mark­er for lac­tate de­hy­dro­ge­nase, or LDH. In 6 pa­tients not re­spond­ing well to Soliris, the av­er­age he­mo­glo­bin lev­el was brought up an av­er­age of 36%, LDH was cor­rect­ed and trans­fu­sions dropped from 3.4/month on eculizum­ab monother­a­py to 0.3/month when APL-2 was added to eculizum­ab. And the biotech raised no un­usu­al red flags on the safe­ty side.

As one of the world’s most ex­pen­sive ther­a­pies, Soliris has in­spired a range of ri­vals all look­ing to re­place it with their own drug. Com­pa­nies like Ra Phar­ma­ceu­ti­cals and Akari have been on the trail, while Soliris’ man­u­fac­tur­er, Alex­ion, has been mak­ing ad­vances with a sec­ond-gen prod­uct for their key mon­ey­mak­er. ALXN1210 — an an­ti-C5 an­ti­body that in­hibits ter­mi­nal com­ple­ment for pa­tients with parox­ys­mal noc­tur­nal he­mo­glo­bin­uria (PNH) — was about the on­ly ex­per­i­men­tal prod­uct that earned much re­spect from new CEO Lud­wig Hantson when he took over ear­li­er this year.

Cedric Fran­cois

Apel­lis has at­tract­ed con­sid­er­able fi­nan­cial sup­port for its work. At the time it filed its S-1, the biotech re­port­ed that Morn­ing­side Ven­ture In­vest­ments owned 32.6% of the com­pa­ny, mak­ing the VC their biggest in­vestor. And they say that they’re just get­ting start­ed with a lead fo­cus on PNH, with a range of oth­er dis­eases that they be­lieve can be treat­ed through the same path­way.

CEO Cedric Fran­cois said he found the da­ta en­cour­ag­ing as he steers the com­pa­ny to a Phase III study in a few months.

We be­lieve that C3-in­hibitor APL-2 can be the next gen­er­a­tion PNH treat­ment of­fer­ing pa­tients a pow­er­ful so­lu­tion to mean­ing­ful­ly im­prove their qual­i­ty of life.


Im­age: Down­town Louisville, Ken­tucky Shut­ter­stock

The Advance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Af­ter de­cou­pling from Re­gen­eron, Sanofi says it’s time to sell the $13B stake picked up in the mar­riage

With Regeneron shares going for a peak price — after doubling from last fall — Sanofi is putting a $13 billion stake in their longtime partner on the auction block. And Regeneron is taking $5 billion of that action for themselves.

Sanofi — which has been decoupling from Regeneron for more than a year now — bought in big in early 2013, back when Regeneron’s stock was going for around $165 a share. Small investors flocked to the deal, buzzing about an imminent takeover. The buyout chatter wound down long ago.

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Covid-19 roundup: Janet Wood­cock steps aside — for now — as FDA drug czar; WHO hits the brakes on hy­droxy study af­ter lat­est safe­ty alarm

The biopharma industry will soon get a look at what the FDA will look like once CDER’s powerful chief Janet Woodcock retires from her post.

Long considered one of the most influential regulators in the agency, if not its single most powerful official when it counts, Woodcock is being detached to devote herself full time to the White House’s special project to fast-forward new drugs and vaccines for the pandemic. The move comes a week after some quick reshuffling as Woodcock and CBER chief Peter Marks joined Operation Warp Speed. Initially they opted to recuse themselves from any FDA decisions on pandemic treatments and vaccines, after consumer advocates criticized the move as a clear conflict of interest in how the agency exercises oversight on new approvals.

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Janet Woodcock, director of the Center for Drug Evaluation and Research (AP Images)

Covid-19 roundup: Hit with new con­flict ac­cu­sa­tions, Janet Wood­cock steps out of the agen­cy's Covid-19 chain of com­mand

Two weeks ago, FDA drug chieftain Janet Woodcock was assuring a top Wall Street analyst that any vaccine approved for combating Covid-19 would have to meet high agency standards on safety and efficacy before it’s approved. But over the weekend, after she and Peter Marks took top positions with the public-private operation meant to speed a new vaccine to lightning-fast approvals — they both recused themselves from the review process after an advocacy group argued their roles close to the White House could pose a conflict of interest.

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An­oth­er NASH de­lay for In­ter­cept frus­trates in­vestors, shares wilt

A previous FDA advisory committee delay for Intercept’s NASH drug may have dampened spirits, but investors perked up after French rival Genfit recently failed to best a placebo with its offering in a keenly anticipated pivotal study. In yet another twist on Friday, the New York drugmaker said the FDA is postponing its adcom again to accommodate the review of additional data it has asked the company to furnish.

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Eric Edwards, Phlow president and CEO (PR Newswire)

BAR­DA of­fers a tiny start­up up to $812M to cre­ate a US-based drug man­u­fac­tur­er — and the CEO comes with a price goug­ing con­tro­ver­sy on his ré­sumé

BARDA has tapped a largely unknown startup to ramp up production of a list of drugs that may be at risk of running short in the US. And the deal, which comes with up to $812 million in federal funds, was inked by a CEO who found himself in the middle of an ugly price gouging controversy a few years ago.

The feds’ new partner — called Phlow — won a 4-year “base” contract of $354 million, with another $458 million that’s on the table in potential options to sustain the outfit. That would make it one of the largest awards in BARDA’s history.

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Julie Grant, Day One CEO

A new biotech from a long­time de­vel­op­er wants to bring the tar­get­ed can­cer rev­o­lu­tion to the kids left be­hind

Daphne Haas-Kogan was treating and studying children’s brain tumors at the University of California San-Francisco, when she got a call that shook her. The pharma company whose drug she had been prepping for a trial had decided, despite all preclinical evidence, to not run any trials on kids, only adults. Haas-Kogan’s patients would not get the therapy.

“Ultimately the company had to make a decision for what trials they would support,” Haas-Kogan said. “I can still recall my blood pressure rising as I found out.”

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Re­searchers de­fine ex­act­ly what they saw in the first pos­i­tive remde­sivir study for Covid-19. But what's that worth to Gilead?

Remdesivir can work in fighting Covid-19, particularly for patients with less severe cases, but this is just a first step in the journey to finding combos that can do the job much better,

That’s the bottom line from Gilead’s randomized study published in the New England Journal of Medicine. Analysts were quick to draw conclusions about how the big biotech could turn this into a profitable advantage — with widespread expectation of considerable pricing restraint on Gilead’s part. Anyone looking for a new mountain of cash to count as the world grapples with the pandemic is likely to come away disappointed.

FDA ap­proves the first gener­ic for Amar­in's Vas­cepa — but is a fish oil price war im­mi­nent?

Late last year, enthusiasm for Amarin’s fish-oil pill Vascepa burgeoned when the FDA signed off on expanding the cholesterol fighter’s label to include the drug’s beneficial impact on cardiovascular risk, but months later the exuberance for the blockbuster-to-be took a big hit when a judge invalidated key patents protecting Vascepa.

Despite Amarin’s $AMRN pledge to appeal — a process that could take months — the ruling opened the door for generic competition. Hikma Pharmaceuticals, one of three challengers in the Nevada suit, on Friday said that its generic copy of pure EPA, the omega-3 fatty acid that constitutes Vascepa, has been approved by the FDA.