J&J antes $50M to get Zymework­s' 6th big bis­pecifics al­liance un­der­way, with $6B in to­tal mile­stones on the ta­ble

New­ly pub­lic Zymeworks $ZYME has just lined up its 6th ma­jor bio­phar­ma dis­cov­ery al­liance, this time bring­ing J&J $JNJ in­to a fold that al­ready in­cludes such big play­ers as Mer­ck, Eli Lil­ly, Cel­gene, GSK and Dai­ichi Sankyo. And this deal starts with a sub­stan­tial — for a dis­cov­ery deal — up­front along with $1.4 bil­lion in to­tal mile­stones on the ta­ble.

Zymeworks’ some­what bat­tered shares took off on the news, spik­ing 38% Mon­day morn­ing.

As in some of its oth­er deals, Zymeworks is bring­ing its Azy­met­ric and EFECT plat­forms to the ta­ble — this time look­ing to iden­ti­fy 6 bis­pecifics for a slate of un­de­clared tar­gets picked by Janssen. And J&J is pay­ing $50 mil­lion to get things start­ed with an­oth­er $282 mil­lion in de­vel­op­ment mile­stones and $1.12 bil­lion in com­mer­cial cash on the line for suc­cess­ful work.

Ali Tehrani

Zymeworks CEO Ali Tehrani tells me that this brings the grand to­tal of biobucks now up for grabs to about $6 bil­lion, with around $100 mil­lion claimed in up­fronts and sup­port pay­ments from part­ners.

That’s about all the de­tail you’re go­ing to get on this par­tic­u­lar pact. Tehrani can’t go in­to specifics, a com­mon as­pect of any re­search pact this ear­ly in the game. But he does al­low that J&J isn’t look­ing at this in a nar­row frame­work, in­stead tak­ing a “broad mind­set” on what Zymeworks can do.

This has been some­thing of a tough year for Zymeworks. The Van­cou­ver-based biotech — which has been ab­sorb­ing new plat­form tech in a fast-paced dri­ve to ex­pand al­liances as it went af­ter its own in-house pro­grams — took in $59 mil­lion on its IPO af­ter pen­cil­ing in a high­er fig­ure, then watched its share price grad­u­al­ly slide to about half of the open.

The lock­up on the stock end­ed a cou­ple of weeks ago, says the CEO, and he’s hap­py enough with where the com­pa­ny is on the street. Franklin Tem­ple­ton and Cor­morant came in on the stock. He ex­pects more such longterm in­vestors to fol­low, with a new road show in the off­ing. About $40 mil­lion of the stock was spo­ken for go­ing in, a com­mon fea­ture in the biotech IPO world, and now every time a gen­er­al­ist sells a thou­sand shares on a thin­ly trad­ed stock like this, it can have a big im­pact on the price.

“Our ob­jec­tive was to in­tro­duce the street (to Zymeworks) piece by piece,” he says. And that means deals like this, more da­ta on the in-house drug ZW25, with an up­date com­ing at the San An­to­nio Breast Can­cer Sym­po­sium and a splash planned for AS­CO next sum­mer.

“If you look at every con­ver­sa­tion we’ve had,” Tehrani tells me, “you know we promise and we de­liv­er. We do what we say.”

And as the biotech per­forms for Wall Street, he be­lieves that mes­sage will res­onate and the price will head back up. As it did this morn­ing.

He’s promised. Now he gets to de­liv­er.

Con­quer­ing a silent killer: HDV and Eiger Bio­Phar­ma­ceu­ti­cals

Hepatitis delta, also known as hepatitis D, is a liver infection caused by the hepatitis delta virus (HDV) that results in the most severe form of human viral hepatitis for which there is no approved therapy.

HDV is a single-stranded, circular RNA virus that requires the envelope protein (HBsAg) of the hepatitis B virus (HBV) for its own assembly. As a result, hepatitis delta virus (HDV) infection occurs only as a co-infection in individuals infected with HBV. However, HDV/HBV co-infections lead to more serious liver disease than HBV infection alone. HDV is associated with faster progression to liver fibrosis (progressing to cirrhosis in about 80% of individuals in 5-10 years), increased risk of liver cancer, and early decompensated cirrhosis and liver failure.
HDV is the most severe form of viral hepatitis with no approved treatment.
Approved nucleos(t)ide treatments for HBV only suppress HBV DNA, do not appreciably impact HBsAg and have no impact on HDV. Investigational agents in development for HBV target multiple new mechanisms. Aspirations are high, but a functional cure for HBV has not been achieved nor is one anticipated in the forseeable future. Without clearance of HBsAg, anti-HBV investigational treatments are not expected to impact the deadly course of HDV infection anytime soon.

UP­DAT­ED: In a land­mark first glimpse of hu­man da­ta from Ver­tex, CRISPR/Cas9 gene ther­a­py sig­nals ear­ly ben­e­fit

Preliminary data on two patients with blood disorders that have been administered with Vertex and partner CRISPR Therapeutics’ gene-editing therapy suggest the technology is safe and effective, marking the first instance of the benefit of the use of CRISPR/Cas9 technology in humans suffering from disease.

Patients in these phase I/II studies give up peripheral blood from which hematopoietic stem and progenitor cells are isolated. The cells are tinkered with using CRISPR/Cas9 technology, and the edited cells — CTX001 — are infused back into the patient via a stem cell transplant. The objective of CTX001 is to fix the errant hemoglobin gene in patents with two blood disorders: beta-thalassemia and sickle cell disease, by unleashing the production of fetal hemoglobin.

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UP­DAT­ED: Make that 2 ap­proved RNAi drugs at Al­ny­lam af­ter the FDA of­fers a speedy OK on ul­tra-rare dis­ease drug

Seventeen years into the game, Alnylam’s pivot into commercial operations is picking up speed.
The bellwether biotech $ALNY has nabbed their second FDA OK for an RNAi drug, this time for givosiran, the only therapy now approved for acute hepatic porphyria. This second approval came months ahead of the February deadline — even after winning priority review following their ‘breakthrough’ title earlier.
AHP is an extremely rare disease, with some 3,000 patients in Europe and the US, not all diagnosed, and analysts have projected peak revenue of $600 million to $700 million a year. The drug will be sold as Givlaari.

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David Ricks. Eli Lilly

Eli Lil­ly touts $400M man­u­fac­tur­ing ex­pan­sion, 100 new jobs to much fan­fare in In­di­anapo­lis — even though it's been chop­ping staff

Eli Lilly is pouring in $400 million to beef up manufacturing facilities at its home base of Indianapolis. The investment, which was lauded by the city’s mayor, is expected to create 100 new jobs.

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Am­gen chops 172 more staffers in R&D, op­er­a­tions and sales amid neu­ro­science ex­it, rev­enue down­turn

Neuroscience wasn’t the only unit that’s being hit by a reorganization underway at Amgen. As well as axing 149 employees in its Cambridge office, the company has disclosed that 172 others nationwide, including some from its Thousand Oaks, CA headquarters, are being let go.

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Stephen Hahn (via Senate HELP Committee)

Stephen Hahn gets through Sen­ate’s soft­ball job in­ter­view — but most­ly plays dodge­ball on the is­sues fac­ing the FDA

Anyone looking for fresh insights on what kind of FDA commissioner Stephen Hahn will be got precious few clues during Wednesday’s Senate hearing on the nomination.

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Op­di­vo/Yer­voy com­bo for melanoma fails in key pa­tient pop­u­la­tion

Bristol-Myers Squibb’s efforts to expand their checkpoint inhibitor combination have run into another recalcitrant cancer.

The NJ-based pharma announced that a combination of Yervoy and Opdivo didn’t beat out Opdivo alone in patients with resected high-risk melanoma who had very low levels of PD-L1. The drug combo couldn’t improve recurrence-free survival in these post-surgery patients.

Ver­tex's stel­lar quar­ter car­ries on with French re­im­burse­ment deal

Vertex’s golden quarter just got brighter. About a month after the US drugmaker finally clinched a deal with UK authorities to cover its slate of cystic fibrosis (CF) drugs following years of protracted negotiations, the company on Wednesday secured a deal with France for its CF therapy, Orkambi.

After the UK, France has one of the largest CF populations outside the United States. Achieving French reimbursement unlocks an ~7000-patient CF population, around ~2500-3000 of which will likely be eligible to receive (and be reimbursed for) Orkambi, Stifel’s Paul Matteis wrote in a note.

Nello Mainolfi, Kymera via Youtube

Kymera hands the helm to No­var­tis vet — and found­ing CSO — Nel­lo Main­olfi

Kymera Therapeutics is turning to a co-founder to run the company.
The protein degradation specialist with a deep-pocket syndicate behind them has opted to give the helm officially to Nello Mainolfi. The new CEO is a veteran of the Novartis Institutes for Biomedical Research. He joined Atlas Venture in their entrepreneur-in-residence program and helped launch Kymera as the CSO three years ago with Atlas’ Bruce Booth.
The boast at Kymera is that they’re angling to create a new class of protein degraders, a popular field where there’s been a variety of startups. One of its chief advocates is NIBR head Jay Bradner, who launched C4 just ahead of joining Novartis, where he’s also been doing new work in the field.