Eye­ing a po­ten­tial mar­ket launch, Astel­las or­ders up a $95M third course of Cy­to­ki­net­ics’ pipeline

What­ev­er Astel­las learned about Cy­to­ki­net­ics $CYTK in the three years since first sign­ing on as a col­lab­o­ra­tor, the ex­pe­ri­ence must have giv­en its re­search team plen­ty of con­fi­dence in the biotech’s fu­ture. Astel­las is an­nounc­ing to­day that it’s com­ing back for a big­ger share of the pipeline, its third big por­tion of Cy­to­ki­net­ics’ tech­nol­o­gy and mus­cle ac­ti­va­tion ther­a­peu­tics.

Astel­las is com­mit­ting $95 mil­lion in the deal. That cov­ers $65 mil­lion up front in ex­change for an op­tion on the de­vel­op­ment and com­mer­cial­iza­tion of tirasem­tiv, Cy­to­ki­net­ics’ lead mus­cle drug now in late-stage de­vel­op­ment, for amy­otroph­ic lat­er­al scle­ro­sis (ALS). And there’s an­oth­er $30 mil­lion to pay for a mid-stage ALS study for an­oth­er mus­cle ac­ti­va­tor in the pipeline, CK-2127107. If Astel­las picks up its op­tion, Cy­to­ki­net­ics can gain a to­tal of more than $100 mil­lion in the deal.

Shares of Cy­to­ki­net­ics surged 6% this morn­ing.

Once the Phase II is done, the part­ners plan to work to­geth­er on a piv­otal study for the ex­per­i­men­tal ther­a­py, with Cy­to­ki­net­ics in line for ad­di­tion­al mile­stones and roy­al­ties if these drugs are ap­proved. An­oth­er drug, ome­cam­tiv mecar­bil, is part­nered with Am­gen on heart fail­ure.

For the South San Fran­cis­co-based biotech’s ex­ec­u­tive team, the Astel­las deal po­si­tions the com­pa­ny for its next long hoped-for strate­gic ad­vance in­to com­mer­cial­iza­tion, spurring plans to prep for their first mar­ket launch.

“We are de­vel­op­ing com­pounds that are di­rect­ly act­ing on mus­cles,” CEO Robert Blum tells me, first am­pli­fy­ing mus­cles for pa­tients in a range of dis­eases, but al­so keep­ing in mind a fu­ture where new ther­a­pies that can amp up mus­cles could have a big im­pact on the emerg­ing field of healthy ag­ing.

It hasn’t all been up­beat news at the com­pa­ny. Back in 2014 tirasem­tiv failed a crit­i­cal Phase IIb study for ALS. But Cy­to­ki­net­ics kept the faith, en­cour­aged by its ef­fect on slow­ing the de­cline of res­pi­ra­to­ry fail­ure.

“In fact,” Blum says, “we be­lieve that our tri­al was the on­ly that ever demon­strat­ed an ef­fect on ALS pa­tients’ mus­cle strength.”

So they’re now test­ing the drug with res­pi­ra­to­ry ca­pac­i­ty as the pri­ma­ry end­point, mea­sur­ing against fac­tors like time to res­pi­ra­to­ry fail­ure or time be­fore pa­tients re­quire me­chan­i­cal ven­ti­la­tion to stay alive. That da­ta is com­ing up in the sec­ond half of next year, and Blum hopes that it will take the part­ners to the thresh­old of mar­ket­ing ap­proval.

Their fo­cus on 7107 re­flects some dose-lim­it­ing prob­lems with tirasem­tiv, which Blum at­trib­ut­es to the lead drug’s abil­i­ty to slip through the blood brain bar­ri­er. That’s not so much the case for 7107, which could make it more suc­cess­ful — if it gets through the clin­ic.

Astel­las’ first move with Cy­to­ki­net­ics oc­curred back in 2013, when they part­nered on non-neu­ro­mus­cu­lar con­di­tions. A year lat­er, the Japan­ese phar­ma came back for a deal in the neu­ro­mus­cu­lar area, tak­ing a po­si­tion on spinal mus­cu­lar at­ro­phy.

If it all leads to glob­al ap­provals, Cy­to­ki­net­ics will lead com­mer­cial­iza­tion on tirasem­tiv in North Amer­i­ca and Eu­rope and a few oth­er coun­tries, while Astel­las has its sights set on Asia and a few oth­er coun­tries out­side the biotech’s com­mer­cial turf. The co-de­vel­op­ment deal on CK-2127107 leaves the biotech co-pro­mo­tion rights on key mar­kets.

There are still plen­ty of po­ten­tial pit­falls along the way, but the lat­est Astel­las deal leaves the com­pa­ny in bet­ter shape for what­ev­er comes next.

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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Turned back at the FDA, Im­muno­Gen is ax­ing 220 staffers, sell­ing pro­grams and hun­ker­ing down for a new PhI­II gam­ble

After being stymied by FDA regulators who were unconvinced by ImmunoGen’s $IMGN desperation shot at an accelerated OK based on a secondary endpoint, the struggling biotech is slashing its workforce, shuttering R&D projects and looking for buyers to pick up some of its experimental cancer assets as it goes back into a new Phase III with the lead drug.

We found out last month that the FDA had batted back their case for an accelerated approval of their antibody-drug conjugate mirvetuximab soravtansine, which had earlier failed a Phase III study for ovarian cancer. Now the other shoe is dropping.

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As­traZeneca chal­lenges Roche on front­line SCLC af­ter seiz­ing an in­ter­im win — and Mer­ck may not be far be­hind

The crowded playing field in the PD-1/L1 marketing game is about to get a little more complex.

This morning AstraZeneca reported that its CASPIAN study delivered a hit in an interim readout for their PD-L1 Imfinzi combined with etoposide and platinum-based chemotherapy options for frontline cases of small cell lung cancer, a tough target which has already knocked back Bristol-Myers’ shot in second-line cases. The positive data  — which we won’t see before they roll it out at an upcoming scientific conference — give AstraZeneca excellent odds of a quick vault to challenging Roche’s Tecentriq-chemo combo, approved 3 months ago for frontline SCLC in a landmark advance.

“This is the first trial offering the flexibility of combining immunotherapy with different platinum-based regimens in small cell lung cancer, expanding treatment options,” noted AstraZeneca cancer R&D chief José Baselga in a statement.

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Bridge­Bio takes crown for biggest biotech IPO of 2019, as fel­low uni­corn Adap­tive rais­es of­fer­ing size and price

Bridge­Bio Phar­ma and Adap­tive Biotech­nolo­gies have not just up­sized IPO of­fer­ings — the pair of uni­corns have al­so raised their of­fer­ing prices above the range, haul­ing in a com­bined $648.5 mil­lion.

Neil Ku­mar’s Bridge­Bio Phar­ma, found­ed in 2015, has a sta­ble of com­pa­nies fo­cused on dis­eases that are dri­ven by de­fects in a sin­gle gene — en­com­pass­ing der­ma­tol­ogy, car­di­ol­o­gy, neu­rol­o­gy, en­docrinol­o­gy, re­nal dis­ease, and oph­thal­mol­o­gy — and can­cers with clear ge­net­ic dri­vers. The start­up mill birthed a pletho­ra of firms such as Ei­dos, Navire, QED Ther­a­peu­tics and Pelle­Pharm, which func­tion as its sub­sidiaries.

Two biotech uni­corns swell pro­posed IPOs, eye­ing a $600M-plus wind­fall

We’ve been wait­ing for the ar­rival of Bridge­Bio’s IPO to top off the wave of new biotech of­fer­ings sweep­ing through Nas­daq at the end of H1. And now we learn that it’s been sub­stan­tial­ly up­sized.

Ini­tial­ly pen­ciled in at a uni­corn-sized $225 mil­lion, the KKR-backed biotech has spiked that to the neigh­bor­hood of $300 mil­lion, look­ing to sell 20 mil­lion shares at $14 to $16 each. That’s an added 5 mil­lion shares, re­ports Re­nais­sance Cap­i­tal, which fig­ures the pro­posed mar­ket val­u­a­tion for Neil Ku­mar’s com­pa­ny at $1.8 bil­lion.

No­var­tis holds back the copy­cat brigade's at­tack on its top drug fran­chise — for now

A fed­er­al judge has put a gener­ic chal­lenge to No­var­tis’ block­buster mul­ti­ple scle­ro­sis drug Gilenya on hold while a patent fight plays out in court.

Judge Leonard P. Stark is­sued a tem­po­rary in­junc­tion ear­li­er this week, forc­ing My­lan, Dr. Red­dy’s Lab­o­ra­to­ries and Au­robindo Phar­ma to shelve their launch plans to al­low the patent fight to pro­ceed. He ruled that al­low­ing the gener­ics in­to the mar­ket now would per­ma­nent­ly slash the price for No­var­tis, even if it pre­vails. 

Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

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FDA re­jects Ac­er's rare dis­ease drug, asks for new tri­al — shares crater

Ac­er Ther­a­peu­tics’ bid to re­pur­pose celipro­lol — a be­ta-block­er on the mar­ket for hy­per­ten­sion — as a treat­ment for a rare, in­her­it­ed con­nec­tive tis­sue dis­or­der has hit a se­vere set­back. The New­ton, Mass­a­chu­setts-based com­pa­ny on Tues­day said the FDA re­ject­ed the drug and has asked for an­oth­er clin­i­cal tri­al.

The com­pa­ny’s shares $AC­ER cratered near­ly 77% to $4.47 in Tues­day morn­ing trad­ing.