In­smed shares soar as PhI­II lung dis­ease study proves pos­i­tive, point­ing to quick FDA pitch

Shares of In­smed $IN­SM rock­et­ed up 133% in pre­mar­ket trad­ing on Tues­day af­ter the biotech re­port­ed that its Phase III study had hit the pri­ma­ry end­point it be­lieves will per­suade the FDA to hand over a break­through ap­proval. The da­ta on ef­fec­tive­ness in im­prov­ing pa­tients’ abil­i­ty to walk, though, is lack­ing and could cloud the up­com­ing shot at an OK.

Will Lewis

Re­searchers in the study say that the in­haled drug Arikayce — now re­ferred to as ALIS — added to stan­dard of care elim­i­nat­ed ev­i­dence of a lung in­fec­tion known as Non­tu­ber­cu­lous My­cobac­te­r­i­al (NTM) dis­ease in 29% of pa­tients, com­pared to on­ly 9% in the stan­dard of care group.

That was a clear hit with in­vestors this morn­ing.

There was no dif­fer­ence be­tween the drug and con­trol groups in 6-minute walk test re­sults, though, a key mea­sure of ef­fi­ca­cy. But if you mixed in all the pa­tients from both arms that “achieved cul­ture con­ver­sion,” then that group did achieve a faster 6-minute test re­sult. That, how­ev­er, is not the kind of clear drug ef­fi­ca­cy the biotech was hop­ing for.

Still, in­ves­ti­ga­tors note that it’s the elim­i­na­tion of NTM that’s the key goal here, and they be­lieve the da­ta point to an ac­cel­er­at­ed ap­proval. The FDA, though, will have to de­cide if the agency agrees with that hope­ful as­sess­ment.

The drug arm al­so ex­pe­ri­enced a slight­ly high­er rate of ad­verse events — 20.2% ver­sus 17.9%. That didn’t es­cape the at­ten­tion of Leerink’s Joseph Schwartz, who still saw more to cel­e­brate than fear in the da­ta. His note:

ALIS led to low­er in­ci­dence of pt. death (2.7% ALIS vs. 4.5% GBT alone) in ad­di­tion to car­diac dis­or­ders (0.4% ALIS vs. 4.5% GBT alone). Al­though p-val­ues were not pro­vid­ed for these in­ci­dents, the ab­solute im­prove­ments are like­ly to sup­port ALIS ef­fi­ca­cy in con­junc­tion with the pri­ma­ry end­point of cul­ture con­ver­sion. That said, we do see a gen­er­al­ly high SAE (20.2% ALIS vs. 17.9% GBT alone), and in­fec­tions and in­fes­ta­tions (9% ALIS vs. 5.4% GBT alone) marked by high­er pneu­mo­nia in­fec­tions (3.6% ALIS vs. 1.8%). Since mgmt. had in­clud­ed geno­typ­ing in their pro­to­col, we should have more in­sight on the fre­quen­cy of re-in­fec­tions among pts. in the CON­VERT study.

This pro­gram ran in­to some se­ri­ous is­sues with in­vestors back in 2014, when In­smed dis­ap­point­ed its most en­thu­si­as­tic back­ers who thought they would shoot for a quick OK at the time. Plans for the Phase III set that time­line back by sev­er­al years. But the com­pa­ny be­lieves it was worth it.

“We con­sid­er these com­pelling top-line da­ta to be a re­mark­able ac­com­plish­ment in a rare dis­ease state with no cur­rent­ly ap­proved ther­a­pies,” said Will Lewis, In­smed’s chief ex­ec­u­tive. “We are par­tic­u­lar­ly en­cour­aged by the con­sis­ten­cy of these da­ta when com­pared with our Phase 2 study re­sults, and look for­ward to ad­di­tion­al da­ta as the CON­VERT study con­tin­ues over the next two years.”

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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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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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.