News brief­ing: Tiny Vac­cinex's drug flops in PhII Hunt­ing­ton's tri­al, stock craters; Siol­ta nabs $30M Se­ries B to de­vel­op mi­cro­bio­me drug

Siol­ta Ther­a­peu­tics, a mi­cro­bio­me com­pa­ny tar­get­ing al­ler­gic dis­eases, raked in a $30 mil­lion Se­ries B to de­vel­op its lead can­di­date, STMC-103H. The drug, which has been FDA fast-tracked, is head­ed for proof-of-con­cept tri­als, ac­cord­ing to the com­pa­ny. Its var­i­ous in­di­ca­tions in­clude al­ler­gic asth­ma, food al­ler­gies, atopic der­mati­tis, al­ler­gic rhini­tis, and al­ler­gy pre­ven­tion.

The news comes just af­ter the Cal­i­for­nia-based biotech added a promi­nent bio­phar­ma vet­er­an as an ad­vi­sor: 20-year Gilead CEO John Mar­tin. The biotech al­so gained Richard Shames as CMO, who came by way of Pro­tag­o­nist Ther­a­peu­tics.

Marc Be­nioff, CEO of the soft­ware gi­ant Sales­force, in­vest­ed in the com­pa­ny’s Se­ries A and re­turned to chip in to the B round. Khosla Ven­tures, Sev­en­ture (Health for Life Cap­i­tal Fund), Sym­Bio­sis, and Glob­al Brain (Kirin Health In­no­va­tion Fund/GB-VII) al­so pitched in.

Vac­cinex drug flops in PhII Hunt­ing­ton’s dis­ease tri­al

Vac­cinex $VC­NX an­nounced its drug pepinemab missed both pri­ma­ry end­points in a Phase II tri­al for ear­ly man­i­fest and pro­dro­mal Hunt­ing­ton’s dis­ease, caus­ing its stock to plunge more than 57% on Tues­day morn­ing.

Pepinemab didn’t show sta­tis­ti­cal sig­nif­i­cance com­pared to a place­bo in two as­sess­ments from the Hunt­ing­ton’s Dis­ease Cog­ni­tive As­sess­ment Bat­tery and Clin­i­cal Glob­al Im­pres­sion of Change (CG­IC), ac­cord­ing to topline da­ta. The hu­man­ized mon­o­clon­al an­ti­body is de­signed to bind and block the ac­tiv­i­ty of sem­a­phorin 4D. De­spite the fail­ure, CEO Mau­rice Za­ud­er­er said the re­sults sug­gest the an­ti­body po­ten­tial­ly tar­gets cor­ti­cal cen­ters, which might ben­e­fit pa­tients at a more ad­vanced stage of HD. This study co­hort in­clud­ed 179 par­tic­i­pants.

“The in­sights gained from this study al­so sug­gest that pepinemab might be an im­por­tant treat­ment op­tion for Alzheimer’s and oth­er neu­rode­gen­er­a­tive dis­eases known to pri­mar­i­ly af­fect frontal cor­tex and to im­pact cog­ni­tion,” Za­ud­er­er hoped in a state­ment.

Vac­cinex says it’s look­ing to en­roll pa­tients this month in a new Alzheimer’s dis­ease study of pepinemab at 15 sites in the US.

The biotech’s shares dipped to $2.36 apiece on Tues­day, down from $5.47 at close on Mon­day.

Ex-CRO boss scores vet­er­an VCs and $59M for his Eu­ro-fo­cused fund

Hav­ing worked with hun­dreds of biotechs as a clin­i­cal tri­al ser­vice provider, Novotech founder Alek Sa­far­i­an is piv­ot­ing to a new ca­reer in ven­ture cap­i­tal.

His firm, AL­SA Ven­tures, has raised $59 mil­lion for the first close of a Eu­rope-fo­cused fund. The fi­nal tar­get? $150 mil­lion.

With the ex­cep­tion of chief in­vest­ment of­fi­cer Graeme Mar­tin, the AL­SA team is based in Lon­don, scout­ing dis­cov­ery stage to late pre­clin­i­cal star­tups that could use their help speed­ing up a move in­to the clin­ic. Mar­tin, the for­mer CEO of Take­da Ven­tures, is based in Pa­lo Al­to.

So far they have al­ready backed three com­pa­nies: UK based Ep­silo­gen and Ox­ford Bio­ther­a­peu­tics, as well as the Bel­gian start­up Mon­tis Bio­sciences. All are tack­ling can­cer from dif­fer­ent an­gles.

That said, “we’re ag­nos­tic to modal­i­ty or ther­a­peu­tic area,” Sa­far­i­an not­ed in a state­ment.

ALX and Mer­ck col­lab­o­rate on car­ci­no­ma com­bo

ALX On­col­o­gy is team­ing up with Mer­ck to study its CD47 block­er ALX148 in com­bi­na­tion with Keytru­da to treat head and neck squa­mous cell car­ci­no­ma (HN­SCC).

As part of the agree­ment, ALX will con­duct two Phase II stud­ies: one to test the two drugs as a first-line treat­ment in pa­tients with PD-L1 ex­press­ing metasta­t­ic or un­re­sectable re­cur­rent HN­SCC, and an­oth­er to test a cock­tail with the two drugs plus stan­dard chemother­a­py as a first-line treat­ment for metasta­t­ic or un­re­sectable re­cur­rent HN­SCC.

The stud­ies are based on Phase Ib da­ta re­leased at AS­CO 2020, which showed that ALX148 plus Keytru­da achieved a 40% ob­jec­tive re­sponse rate and a me­di­an pro­gres­sion-free sur­vival of 4.6 months in HN­SCC pa­tients who re­ceived pri­or treat­ment.

Charles Baum, Mirati CEO

Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Biond­Vax stock im­plodes af­ter a big PhI­II gam­ble for its uni­ver­sal flu vac­cine fails

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.

Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

News brief­ing: Gilead com­pletes $21B buy­out of Im­munomedics; In­nate re­ceives $50M mile­stone pay­ment from As­traZeneca

Gilead’s $21 billion mega-acquisition of Immunomedics is now officially complete, the companies announced Friday morning.

The full merger process took a little over a month, with Gilead and Immunomedics signing an agreement on Sept. 13. Gilead acquired all outstanding stock of Immunomedics for $88 per share, a 108% premium on the previous day’s closing price.

Gilead’s big prize was Trodelvy, approved in July for the treatment of metastatic triple-negative breast cancer. The drug also impressed at last month’s ESMO conference, reducing the risk of death by 52% in a Phase III study.

A top drug pro­gram at Bay­er clears a high bar for CKD — open­ing the door to an FDA pitch

Over the past 4 years, Bayer has been steering a major trial through a pivotal program to see if their drug finerenone could slow down the pace of chronic kidney disease in patients suffering from both CKD as well as Type 2 diabetes.

Today, their team jumped on a virtual meeting hosted by the American Society of Nephrology to offer a solid set of pivotal data to demonstrate that the drug can delay dialysis or a kidney replacement as well as cardio disease, while also adding some worrying signs of hyperkalemia among the patients taking the drug. And they’re hustling it straight to regulators in search of an approval for kidney disease and cardio patients — one of the toughest challenges in the book, as demonstrated by repeated past failures.

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