J&J-led coali­tion launch­es a land­mark ef­fi­ca­cy study for an HIV mo­sa­ic vac­cine

For decades now, in­ves­ti­ga­tors have been drawn to HIV vac­cine re­search — a Holy Grail in an­ti-vi­ral re­search that would have an enor­mous im­pact in a world that still sees two mil­lion new in­fec­tions every year.

Jo­han Van Hoof

Now J&J is step­ping for­ward and spear­head­ing the first ef­fi­ca­cy study — HVTN 705/HPX2008, al­so known as “Im­boko­do” — of a new, so-called mo­sa­ic vac­cine that is en­rolling 2,600 African women to check on its abil­i­ty to quell the spread of the lethal virus.

Glax­o­SmithK­line, mean­while, si­mul­ta­ne­ous­ly an­nounced its plans to start their own Phase III study of long-act­ing cabote­gravir to see if it can ef­fec­tive­ly shut down the spread of HIV. Their re­searchers at Vi­iV will be test­ing the drug in 3,200 women in sub-Sa­ha­ran Africa.

HIV has de­feat­ed re­peat­ed vac­cine pro­grams, large­ly be­cause of the way it can mu­tate rapid­ly, of­fer­ing a puz­zling and con­stant­ly shift­ing ge­net­ic di­ver­si­ty that cre­ates a pletho­ra of sub­types around the world.

To counter that, re­searchers with J&J pieced to­geth­er vac­cines that re­ly on mul­ti­ple genes from a wide va­ri­ety of sub­types, look­ing for a com­plex pro­file that would trig­ger an im­mune re­sponse re­gard­less of where it was used around the world. An­oth­er study — HVTN 702 — has a dif­fer­ent vac­cine un­der the mi­cro­scope.

In an­i­mal stud­ies us­ing non-hu­man pri­mates, the vac­cine wound up with an ef­fi­ca­cy rate of 66%, said Jo­han Van Hoof, Janssen R&D chief for In­fec­tious Dis­eases & Vac­cines. And re­searchers were able to come up with the bio­mark­ers need­ed to check ef­fec­tive im­mune re­spons­es in healthy vol­un­teers, which al­so checked out.

So what con­cen­tra­tion would need to be pro­tect­ed in or­der to stem the epi­dem­ic?

Go­ing as low as 50% would still de­liv­er a marked im­pact on the ki­net­ics of the dis­ease, says Van Hoof. And stud­ies sug­gest that even as low as 30% could have an ef­fect.

Re­searchers will find out a lot more as they track their sub­jects over an ini­tial 2-year pe­ri­od, with plans to con­tin­ue to fol­low sub­jects to check on dura­bil­i­ty. But un­der the best of cir­cum­stances, says the sci­en­tist, it would prob­a­bly take 5 years to get a vac­cine in use around the world.

I asked Van Hoof how many peo­ple would need to be vac­ci­nat­ed glob­al­ly to stop HIV, and he paused for a mo­ment be­fore re­ply­ing: “That’s the one bil­lion dol­lar ques­tion.”

In the mean­time, re­searchers are still seek­ing a cure, look­ing to erad­i­cate the reser­voirs of virus that can re­main hid­den in cells while drug cock­tails keep the lid on AIDS.

J&J is backed by a large coali­tion that in­cludes sup­port from the Gates Foun­da­tion as well as NI­AID and a wide range of groups rang­ing from Har­vard and MIT to the US Mil­i­tary HIV Re­search Pro­gram at the Wal­ter Reed Army In­sti­tute of Re­search.

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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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.

Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three major buyouts announced: Takeda/Shire; Bristol-Myers/Celgene and now AbbVie/Allergan. And with this latest deal it’s increasingly clear that the sharp fall from grace suffered by high-profile players which have seen their share prices blasted has created an opening for the growth players in big pharma to up their game — in sharp contrast to the popular bolt-on deals that have been driving the growth strategy at Novartis, Merck, Roche and others.

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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.

Suf­fer­ing No­var­tis part­ner Cona­tus grabs the ax and packs it in on NASH af­ter a se­ries of set­backs

The NASH par­ty is over at No­var­tis-backed Cona­tus. And this time they’re turn­ing off the lights.

More than 2 years af­ter No­var­tis sur­prised the biotech in­vest­ment com­mu­ni­ty with its $50 mil­lion up­front and promise of R&D sup­port to part­ner with the lit­tle biotech on NASH — ig­nit­ing a light­ning strike for the share price — Cona­tus $CNAT is back with the lat­est bit­ter tale to tell about em­ri­c­as­an, which once in­spired con­fi­dence at the phar­ma gi­ant.

Bet­ter than Am­bi­en? Min­er­va soars on PhI­Ib up­date on sel­torex­ant for in­som­nia

A month af­ter roil­ing in­vestors with what skep­tics dis­missed as cher­ry pick­ing of its de­pres­sion da­ta, Min­er­va is back with a clean slate of da­ta from its Phase IIb in­som­nia tri­al.

In a de­tailed up­date, the Waltham, MA-based biotech said sel­torex­ant (MIN-202) hit both the pri­ma­ry and sev­er­al sec­ondary end­points, ef­fec­tive­ly im­prov­ing sleep in­duc­tion and pro­long­ing sleep du­ra­tion. In­ves­ti­ga­tors made a point to note that the ef­fects were con­sis­tent across the adult and el­der­ly pop­u­la­tions, with the lat­ter more prone to the sleep dis­or­der.

Gene ther­a­py biotech sees its stock rock­et high­er on promis­ing re­sults for rare cas­es of but­ter­fly dis­ease

Shares of Krys­tal Biotech took off this morn­ing $KRYS af­ter the lit­tle biotech re­port­ed promis­ing re­sults from its gene ther­a­py to treat a rare skin dis­ease called epi­der­mol­y­sis bul­losa.

Fo­cus­ing on an up­date with 4 new pa­tients, re­searchers spot­light­ed the suc­cess of KB103 in clos­ing some stub­born wounds. Krys­tal says that of 4 re­cur­ring and 2 chron­ic skin wounds treat­ed with the gene ther­a­py, the KB103 group saw the clo­sure of 5. The 6th — a chron­ic wound, de­fined as a wound that had re­mained open for more than 12 weeks — was par­tial­ly closed. That brings the to­tal so far to 8 treat­ed wounds, with 7 clo­sures.

Ab­b­Vie gets a green light to re­sume re­cruit­ing pa­tients for one myelo­ma study — but Ven­clex­ta re­mains un­der a cloud

Three months af­ter reg­u­la­tors at the FDA forced Ab­b­Vie to halt en­rolling pa­tients in its tri­als of a com­bi­na­tion us­ing Ven­clex­ta (vene­to­clax) to treat drug-re­sis­tant cas­es of mul­ti­ple myelo­ma, the agency has green-light­ed the re­sump­tion of one of those stud­ies, while keep­ing the rest on the side­lines.

The CANO­VA (M13-494) study can now get back in busi­ness re­cruit­ing pa­tients to test the drug for a pop­u­la­tion that shares a par­tic­u­lar ge­net­ic bio­mark­er. To get that per­mis­sion, Ab­b­Vie — which is part­nered with Roche on this pro­gram — was forced to re­vise the pro­to­col, mak­ing un­spec­i­fied changes in­volv­ing risk mit­i­ga­tion mea­sures, pro­to­col-spec­i­fied guide­lines and an up­dat­ed fu­til­i­ty cri­te­ria.