Al­ny­lam makes RNAi his­to­ry again as patisir­an gets ap­proved in Eu­rope; Ver­tex buys in­to UK-based ma­chine learn­ing group

→ Just days af­ter Al­ny­lam land­ed a his­toric FDA OK on patisir­an — and quick­ly launched it as On­pat­tro — the Eu­ro­pean Com­mis­sion has come through with its own mar­ket­ing au­tho­riza­tion. The drug, which treats hered­i­tary AT­TR amy­loi­do­sis by si­lenc­ing mes­sen­ger RNA and block­ing the pro­duc­tion of TTR pro­tein, is the first RNAi ther­a­py to be ap­proved in Eu­rope. It’s al­so a like­ly block­buster-to-be, with Clar­i­vate peg­ging 2022 pro­ject­ed sales at $1.21 bil­lion even though the dis­ease af­flicts on­ly about 30,000 peo­ple world­wide. In­ot­ersen, a ri­val prod­uct from Akcea and Io­n­is, was ap­proved in Eu­rope in Ju­ly.

Ver­tex is part­ner­ing with UK-based Ge­nomics plc on their plat­form for ge­net­ics and ma­chine learn­ing. Ver­tex $VRTX made a £10.5 mil­lion eq­ui­ty in­vest­ment in Ge­nomics plc as part of its £25 mil­lion Se­ries B fund­ing round — al­so an­nounced to­day. “Hu­man bi­ol­o­gy and ge­net­ics are a foun­da­tion of drug dis­cov­ery at Ver­tex and the team at Ge­nomics brings to­geth­er a unique com­bi­na­tion of so­phis­ti­cat­ed da­ta sci­ence and hu­man ge­net­ics,” said Ver­tex CSO David Alt­shuler. “This part­ner­ship will pi­o­neer new us­es of ge­nom­ic tools and tech­nolo­gies to ad­vance Ver­tex’s on­go­ing work and in­vest­ment at our Ox­ford Re­search Site and glob­al­ly to bring new med­i­cines to the pa­tients that need them.”

→ We fi­nal­ly know what to call Chi­na’s re­vamped FDA. The drug reg­u­la­tor will now go by the Na­tion­al Med­ical Prod­ucts Ad­min­is­tra­tion, or NM­PA, to re­flect its new man­date af­ter a gov­ern­ment re­or­ga­ni­za­tion sep­a­rat­ed med­i­cine reg­u­la­tion from oth­er con­sumer goods — in­clud­ing every­thing food-re­lat­ed. NM­PA re­ports to the new­ly cre­at­ed State Ad­min­is­tra­tion for Mar­ket Reg­u­la­tion.

Evotec and the CH­DI Foun­da­tion have ex­tend­ed their neu­ro­science col­lab­o­ra­tion for a few more years. In the pact, CH­DI will help fund the work of up to 75 sci­en­tists fo­cused on Hunt­ing­ton’s dis­ease.

Tem­pus, a pre­ci­sion med­i­cine com­pa­ny pro­vid­ing da­ta an­a­lyt­ic ser­vices to physi­cians and re­searchers, has raised $110 mil­lion in Se­ries E fi­nanc­ing. Er­ic Lefkof­sky — best known for co-found­ing Groupon — start­ed the com­pa­ny to as­sist re­al-time de­ci­sion-mak­ing for pa­tients, first in can­cer care. Har­ness­ing their pool of pa­tient da­ta, the com­pa­ny al­so op­er­ates an organoid de­vel­op­ment and test­ing fa­cil­i­ty to build bi­o­log­i­cal mod­els that they be­lieve can in­form clin­i­cal tri­als.

John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

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Dave Barrett, Brian Chee, Amir Nashat, Amy Schulman. Polaris

Bob Langer's first port of call — Po­laris Part­ners — maps $400M for ninth fund

Health and tech ven­ture group Po­laris Part­ners, which counts Alec­tor, Al­ny­lam and Ed­i­tas Med­i­cine as part of its port­fo­lio, is set­ting up its ninth fund, rough­ly two years af­ter it closed Po­laris VI­II with $435 mil­lion in the bank, sur­pass­ing its tar­get by $35 mil­lion.

The Boston-based firm, in an SEC fil­ing, said it in­tends to raise $400 mil­lion for the fund. Po­laris — which rou­tine­ly backs com­pa­nies mold­ed out of the work done in the lab of pro­lif­ic sci­en­tist Bob Langer of MIT  — typ­i­cal­ly in­vests ear­ly, and sticks around till com­pa­nies are in the green. Like its peers at Flag­ship and Third Rock, Po­laris is all about cham­pi­oning the lo­cal biotech scene with a steady flow of start­up cash.

The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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Partners Innovation Fund

David de Graaf now has his $28.5M launch round in place, build­ing a coen­zyme A plat­form in his lat­est start­up

Long­time biotech ex­ec David de Graaf has the cash he needs to set up the pre­clin­i­cal foun­da­tion for his coen­zyme A me­tab­o­lism com­pa­ny Comet. A few high-pro­file in­vestors joined the ven­ture syn­di­cate to sup­ply Comet with $28.5 mil­lion in launch mon­ey — enough to get it two years in­to the plat­form-build­ing game, with­in knock­ing dis­tance of the clin­ic.

Canaan jumped in along­side ex­ist­ing in­vestor Sofinno­va Part­ners to co-lead the round, with par­tic­i­pa­tion by ex­ist­ing in­vestor INKEF Cap­i­tal and new in­vestor BioIn­no­va­tion Cap­i­tal.

A uni­corn stalks Wall Street in search of IPO cash; CASI Phar­ma in-li­cens­es CD19 ther­a­py from Chi­na’s Ju­ven­tas

→ A herd of up­start biotechs will look to Wall Street for some ma­jor wind­falls this week as a burst of new of­fer­ings con­tin­ues to feed cash in­to the R&D sys­tem. To­day we learned that Bridge­Bio will look to raise in the neigh­bor­hood of $225 mil­lion by of­fer­ing 15 mil­lion shares for $14 to $16 each. And they have a string of joint bookrun­ners: J.P. Mor­gan, Gold­man Sachs, Jef­feries, SVB Leerink, KKR, Piper Jaf­fray, Mizuho Se­cu­ri­ties, BMO Cap­i­tal Mar­kets and Ray­mond James. If suc­cess­ful, Bridge­Bio will emerge with a mar­ket cap of around $1.7 bil­lion. There are 5 biotechs look­ing to IPO this week, in­clud­ing Akero and Pre­vail.

Right back at you, Pfiz­er: BeiGene and a Pfiz­er spin­out launch a new­co to de­vel­op a MEK/BRAF in­hibitor that could ri­val $11.4B com­bo

A day af­ter Pfiz­er bought Ar­ray and its ap­proved can­cer com­bo, BeiGene and Pfiz­er spin­out Spring­Works have part­nered in launch­ing a new biotech that has an eye on the very same mar­ket the phar­ma gi­ant just paid bil­lions for. And they’re plan­ning on us­ing an ex-Pfiz­er drug to do it.

In a nut­shell, Chi­na’s BeiGene is toss­ing in a pre­clin­i­cal BRAF in­hibitor — BGB-3245, which cov­ers both V600 and non-V600 BRAF mu­ta­tions — for a big stake in a new, joint­ly con­trolled biotech called Map­Kure with Bain-backed Spring­Works.

Step­ping on Roche's toes, Mer­ck cuts in­to SCLC niche with third-line Keytru­da OK

In the in­creas­ing­ly crowd­ed check­point race, small cell lung can­cer has been a rare area where Roche, a sec­ond run­ner-up, has a lead over the en­trenched lead­ers Mer­ck and Bris­tol-My­ers Squibb. But Mer­ck is fi­nal­ly mak­ing some head­way in that di­rec­tion with the lat­est ap­proval for its PD-1 star.

The lat­est green light en­dors­es Keytru­da in the third-line treat­ment of metasta­t­ic SCLC, where it would be giv­en to pa­tients whose dis­ease ei­ther don’t re­spond to or re­lapse af­ter chemother­a­py, which would have fol­lowed at least one pri­or line of ther­a­py.

Sanofi aligns it­self with Google to stream­line drug de­vel­op­ment

Tech­nol­o­gy is bleed­ing in­to health­care, and big phar­ma is rid­ing the wave. Sanofi $SNY ap­point­ed its first chief dig­i­tal of­fi­cer this Feb­ru­ary, fol­low­ing the foot­steps of its peers. By May, the French drug­mak­er and some of its big phar­ma com­pa­tri­ots joined forces with Google par­ent Al­pha­bet’s Ver­i­ly unit to aug­ment clin­i­cal tri­al re­search. On Tues­day, the Parisian com­pa­ny tied up with Google to ac­cess its cloud com­put­ing and ar­ti­fi­cial in­tel­li­gence tech to spur the de­vel­op­ment of new ther­a­pies.

Af­ter watch­ing its share price soar on a Bloomberg re­port and heat­ed ru­mors, Bio­haven stock takes a bil­lion-dol­lar bath

Back in April, Biohaven Pharmaceutical became one hot biotech stock $BHVN based on a report in Bloomberg that some “potential bidders” had been kicking the tires at the biotech, which has a lead drug for migraines. Then the rumor mill really started to smoke when execs canceled a presentation at an investor conference a little more than a week ago.

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