Backed by part­ners at Pfiz­er, eF­FEC­TOR brings its VC to­tal to $150M as PhII can­cer tri­al looms

With a big as­sist from its Big Phar­ma col­lab­o­ra­tor Pfiz­er, San Diego-based eF­FEC­TOR Ther­a­peu­tics has now added a $38.5 mil­lion round to pay for a Phase II pro­gram to test the com­bi­na­tion of its oral im­muno-ther­a­py drug with avelum­ab (Baven­cio) — Pfiz­er and Mer­ck KGaA’s PD-L1 check­point in­hibitor.

Steve Wor­land

The round com­pletes a se­ries of moves by eF­FEC­TOR, which has moved from pre­clin­i­cal tests to check on the po­ten­tial of a com­bo to ear­ly-stage monother­a­py work to test the drug in a small group of can­cer pa­tients. Pfiz­er had agreed to share the costs of the com­ing Phase II tri­als with eF­FEC­TOR, and Pfiz­er Ven­ture In­vest­ments took the lead on the new round, which brings its to­tal raised to $150 mil­lion.

Alexan­dria Ven­ture In­vest­ments al­so stepped in for the first time, along­side a big syn­di­cate of found­ing in­vestors that in­cludes a num­ber of cor­po­rate ven­ture arms: U.S. Ven­ture Part­ners, Abing­worth, No­var­tis Ven­ture Fund, SR One, The Col­umn Group, Al­ti­tude Life Sci­ence Ven­tures, Sec­toral As­set Man­age­ment, Ab­b­vie Biotech Ven­tures, Bio­Med Ven­tures, and Astel­las Ven­tures.

The biotech’s lead drug is eFT508, an oral in­hibitor of MNK 1 and 2 ki­nas­es that play a role in evad­ing an im­mune sys­tem at­tack. About a month ago Pfiz­er, Mer­ck KGaA and eF­FEC­TOR signed off on a col­lab­o­ra­tion to fund a split Phase II study that will test a com­bi­na­tion of their two ther­a­pies against colon can­cer with a monother­a­py arm for eFT508.

“The pri­ma­ry role is to trans­la­tion­al­ly reg­u­late gene ex­pres­sion,” CEO Steve Wor­land tells me, with a di­rect ef­fect on the tu­mor “as well as the ef­fect on the im­mune sys­tem’s ca­pa­bil­i­ty to at­tack the tu­mor.”

In Wor­land’s view, the lead drug has po­ten­tial as a monother­a­py, but its best use could well be in com­bi­na­tion with check­point in­hibitors like the PD-L1 drug avelum­ab or LAG3 or oth­er check­points. He ex­pects to have Phase II da­ta avail­able from the pro­gram in mid-2018, but Wor­land isn’t cer­tain that in­ves­ti­ga­tors will be able to pull all the num­bers to­geth­er in time for AS­CO.

Wor­land, the for­mer CEO of Anadys, which was ac­quired by Roche, snagged a $45 mil­lion A round to start the com­pa­ny in 2013, then fol­lowed that with $56 mil­lion more for the B round.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Can a mag­net­ic cell ther­a­py re­place corneal trans­plan­ta­tion? As eight-year jour­ney leads to the clin­ic, two broth­ers un­veil bold vi­sion

Jeff Goldberg was getting acquainted with a brand new way to do corneal transplants when an even newer, even bolder idea hit him.

It was almost 10 years ago, and Goldberg was in his first faculty position at Bascom Palmer Eye Institute at the University of Miami. Scientists had developed a new way to do cornea transplants where instead of sewing a whole donor cornea — a decades-old practice — they were just engrafting the inner layer of cells.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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Francesco De Rubertis

Medicx­i's David Grainger and Francesco De Ru­ber­tis pump €200M in­to six com­pa­nies and what they say is a first-of-its kind fund

In what they’re billing as a first for biotech, David Grainger, Francesco De Rubertis and their team at Medicxi have put down €200 million to sweep up stakes in six companies from their predecessor VC and pump new money into them.

Medicxi didn’t disclose which companies it was investing in but the portfolio draws from Index Ventures Life VI, one of the last funds the Medicxi team launched while they were still part of the multinational, tech-focused VC firm Index Ventures. That team kept advising Index on their life sciences portfolio even after they spun out to form their own firm in the middle of 2016.

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Blueprint CEO Jeff Albers (file photo)

Blue­print plots re­turn to FDA with new Ay­vak­it da­ta in rare con­di­tion — and the an­a­lysts cheer

Over a decade after launch, Blueprint Medicines nabbed the first approval for their first drug earlier this year. Now, as they move forward with a Roche-partnered global launch, they’re touting data that could push them into more patients.

The Jeff Albers-led Cambridge biotech released their full pivotal data for Ayvakit in patients with advanced systemic mastocytosis. In one 53-person study, they showed that 76% of patients responded to the drug, 36% had complete responses and that on average their responses lasted for just over 3 years. A smaller, 32-patient study had a 75% response rate and most were still responding after 10.4 months, the last follow-up.

President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists. HHS con­tin­ues to claim Azar “will de­fer com­plete­ly to the FDA"

President Donald Trump, who seems intent on announcing a Covid-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.