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.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. The idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.

Arde­lyx bags its first FDA OK for IBS, set­ting up a show­down with Al­ler­gan, Iron­wood

In the first of what it hopes will be a couple of major regulatory milestones for its new drug, Ardelyx has bagged an FDA approval to market Ibsrela (tenapanor) for irritable bowel syndrome.

The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase III trials.

Ver­tex deal for Scot­land — no deal for Eng­land

Cystic fibrosis (CF) drug maker Vertex Pharmaceuticals — which is still locked in negotiation with NHS England to endorse the use of its medicines — has successfully negotiated a deal with Scottish authorities.

A month ago, the Scottish Medicines Consortium spurned two of the company’s medicines — Orkambi and Symkevi — citing uncertainty over their long-term efficacy in relation to their cost.

Tony Kulesa, Brian Baynes. Petri

Star founders, in­ves­ti­ga­tors hud­dle around new Boston ac­cel­er­a­tor spot­light­ing young en­tre­pre­neurs

As a widely recognized biotech hub, Boston is undoubtedly one of the best places to start a new company at the frontier of biology and engineering. With a dense network of incubators, venture capitalists and talent, seasoned company founders can have their pick of partners and models launching their latest startups.

But for young, aspiring entrepreneurs, it’s a very different scene.

Big VC firms might hire you to work on their ideas instead of yours, and accelerators may not offer the kind of deep technical expertise and guidance needed to make it in the field.

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From part­ner to knight in shin­ing ar­mor: Cas­tle Creek to buy Fi­bro­cell

In April, Castle Creek swooped in to partner with the embattled gene and cell therapy Fibrocell to shepherd its lead gene therapy for a type of “butterfly” disease into late-stage development. Now, the New Jersey-based dermatology company is acquiring its partner in a deal worth $63.3 million.

Pennsylvania-based Fibrocell last year initiated a review of strategic alternatives, including a sale.