Neu­rotrope bat­tered in lat­est Alzheimer's flop; Platelet Bio­Gen­e­sis rais­es $26M-plus

Neu­rotrope be­came the lat­est ca­su­al­ty in the high-risk Alzheimer’s R&D field. The biotech re­port­ed that their Phase II study of Bryo­statin-1 failed to show any im­prove­ment over place­bo us­ing the Se­vere Im­pair­ment Bat­tery (SIB) to­tal score at week 13. In fact, the drug arm did a lit­tle worse than a sug­ar pill, with an av­er­age in­crease of 1.3 points for the drug and 2.1 points for place­bo. Their shares $NTRP cratered in the rout that fol­lowed, plung­ing 78%. It’s cur­rent­ly a pen­ny stock.

The Alzheimer’s R&D field hasn’t record­ed a suc­cess in chang­ing the tra­jec­to­ry of the dis­ease, with re­cent piv­otal fail­ures forc­ing all the play­ers to re­con­sid­er their strate­gies.

Platelet Bio­Gen­e­sis (PBG) — a com­pa­ny fo­cused on stem cell-de­rived, on-de­mand hu­man platelets and platelet-based ther­a­peu­tics — has raised more than $26 mil­lion in Se­ries A-1 fi­nanc­ing. In com­bi­na­tion to this round, as well as Se­ries A fi­nanc­ing and grants, the com­pa­ny’s to­tal fund­ing is brought to about $45 mil­lion. The fi­nanc­ing was co-led by Ziff Cap­i­tal Part­ners and Qim­ing Ven­ture Part­ners USA in ad­di­tion to ex­ist­ing in­ves­tures Nest.Bio Ven­tures and eCoast An­gels.

The com­pa­ny said that it “will use the funds from the Se­ries A-1 round to ac­cel­er­ate the de­vel­op­ment of its donor-in­de­pen­dent platelet man­u­fac­tur­ing ca­pa­bil­i­ties and ad­vance its proof of con­cept work around platelet-based ther­a­peu­tics.” PBG plans to dou­ble its staff to ap­prox­i­mate­ly 50 em­ploy­ees in 2020.

Stu­art Levy, co-founder of Paratek Phar­ma­ceu­ti­cals, passed away last week.

“Stu­art was a dis­tin­guished physi­cian who was the fore­front of an­tibi­ot­ic de­vel­op­ment and a ded­i­cat­ed cham­pi­on for the pru­dent use of an­tibi­otics,” said Evan Loh, Paratek CEO. “More im­por­tant­ly, he was a dear friend and men­tor to so many and his pas­sion lives on in each of us as we work to con­tin­ue his mis­sion of com­bat­ing the dai­ly threat of life-threat­en­ing in­fec­tions. We will great­ly miss him.”

Levy co-found­ed Paratek more than 20 years ago along with Wal­ter Gilbert. He was in­stru­men­tal in the de­vel­op­ment of the com­pa­ny’s lead an­tibi­otics, Nuzyra and Seysara. Levy dis­cov­ered the ef­flux mech­a­nism for drug (tetra­cy­cline) re­sis­tance and was among the first sci­en­tists to doc­u­ment the trans­fer of re­sis­tant bac­te­ria from an­i­mals to farm­work­ers. His 1992 book, The An­tibi­ot­ic Para­dox: How Mir­a­cle Drugs Are De­stroy­ing the Mir­a­cle, has been cit­ed wide­ly and trans­lat­ed in­to four lan­guages.

The news comes a few weeks af­ter the com­pa­ny pub­lished pos­i­tive Phase III da­ta for Nuzyra for pa­tients with AB­SS­SI.

→ In the midst of a sur­prise in­ter partes re­view by the US Patent Tri­als Ap­peal Board (PT­AB) for Alex­ion’s Soliris, the com­pa­ny and Bridge­Bio’s sub­sidiary Ei­dos Ther­a­peu­tics have inked an agree­ment grant­i­ng Alex­ion an ex­clu­sive li­cense to de­vel­op and com­mer­cial­ize their oral­ly-ad­min­is­tered small mol­e­cule AG10 — de­signed to treat transthyretin amy­loi­do­sis (AT­TR) by bind­ing and sta­bi­liz­ing transthyretin (TTR) in the blood— in Japan.

“Ei­dos is cur­rent­ly eval­u­at­ing AG10 in a Phase III study in the US and Eu­rope for AT­TR car­diomy­opa­thy (AT­TR-CM) – a pro­gres­sive, fa­tal dis­ease caused by the ac­cu­mu­la­tion of mis­fold­ed TTR amy­loid in the heart – and plans to be­gin a Phase III study in AT­TR polyneu­ropa­thy (AT­TR-PN) – a pro­gres­sive, fa­tal dis­ease caused by the ac­cu­mu­la­tion of mis­fold­ed TTR amy­loid in the pe­riph­er­al ner­vous sys­tem.”

→ A few months af­ter Ac­celeron and its part­ner at Cel­gene se­cured pri­or­i­ty re­view for their red blood cell boost­ing drug, lus­pa­ter­cept — to man­age ane­mia re­sult­ing from myelodys­plas­tic syn­drome (MDS) as well as be­ta tha­lassemia — the com­pa­ny has an­nounced an­oth­er win. This time they’ve gained an or­phan drug des­ig­na­tion by the FDA for its in­ves­ti­ga­tion­al ther­a­py so­tater­cept for the treat­ment of pa­tients with pul­monary ar­te­r­i­al hy­per­ten­sion (PAH).

The top 100 bio­phar­ma VCs, Bob Brad­way places $2B bet in can­cer, gene edit­ing pi­o­neer's new big idea, and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Before diving in, we had some news to share: Endpoints is launching a premium weekly report focusing on all things regulatory. Coverage will be led by our new senior editor, Zachary Brennan, who joins us from POLITICO. Arsalan Arif has more details in his Publisher’s Note.

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Robert Bradway (Photographer: Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Am­gen snaps up can­cer drug play­er Five Prime, adding PhI­II-ready FGFR2b drug in $2B M&A play

Amgen is making a long-awaited move on the M&A side, buying South San Francisco-based Five Prime $FPRX for close to $2 billion and adding a slate of new cancer drugs to the pipeline.

Amgen is paying $38 a share, putting the deal value at $1.9 billion. The stock closed at $21.26 last night, giving investors a 78% premium.

The jewel in the crown of this deal is bemarituzumab, which Amgen describes as a first-in-class, Phase III-ready anti-FGFR2b antibody. Amgen was drawn to the bargaining table by Five Prime’s mid-stage data on gastric cancer, satisfied by PFS and OS data helping to validate FGFR2b as a target. Amgen researchers will now expand on the R&D program in other epithelial cancers, including lung, breast, ovarian and other cancers.

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David Liu (Casey Atkins Photography courtesy Broad Institute)

David Liu has a new big idea: pro­teome edit­ing. It could one day shred tau, RAS and some of the worst dis­ease-caus­ing pro­teins

Before David Liu became famous for inventing new forms of gene editing, he was known around academia in part for a more obscure innovation: a Rube Goldberg-esque system that uses bacteria-infecting viruses to take one protein and turn it into another.

Since 2011, Liu’s lab has used the system, called PACE, to dream up fantastical new proteins: DNA base editors far more powerful than the original; more versatile forms of the gene editor Cas9; insecticides that kill insecticide-resistant bugs; enzymes that slide synthetic amino acids into living organisms. But they struggled throughout to master one of the most common and powerful proteins in the biological world: proteases, a set of Swiss army knife enzymes that cut, cleave or shred other proteins in everything from viruses to humans.

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The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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Bruce Cozadd, Jazz CEO (Jazz Pharmaceuticals)

Jazz CEO Bruce Cozadd cam­paigned for 6 months to buy GW Phar­ma. A 90% pre­mi­um sealed the deal — along with $17.6M in ‘re­ten­tion’ in­cen­tives

Jazz CEO Bruce Cozadd didn’t beat around the bush.

In his first video meeting with GW Pharma chief Justin Gover last July 8, he offered to pay $172 a share to get the company, which had beaten the odds in getting its remarkable cannabinoid drug Epidiolex across the regulatory finish line for epilepsy. GW’s stock closed at $129 that day.

Cozadd had already done his homework on the financing to make sure he could swing it the way he wanted. He just needed to do some due diligence before making the non-binding bid firm.

Seagen warns in­vestors against TRC Cap­i­tal’s lat­est 'mi­ni-ten­der of­fer'; BeiGene goes af­ter a new in­di­ca­tion for top PD-1 play­er

TRC Capital, which has selected various biotechs like Vertex and Biogen for the “mini-tender” treatment, jumped back into the game last month with an offer to buy shares in Seagen for $151. The problem, says Seagen, is that price was 4.28% lower than what the stock was selling for at the time they made the offer on Feb. 20, giving TRC a shot at an instant windfall.

So why sell for less than what it’s worth? Seagen notes warnings from regulatory authorities that these offers essentially try to trick investors into believing that they’re being offered a premium for the stock.

UP­DAT­ED: Not 3 weeks af­ter tak­ing Hu­ma­cyte pub­lic, Ra­jiv Shuk­la launch­es an­oth­er blank check com­pa­ny

One of biotech’s earliest SPAC investors is back with another blank-check company, less than a month after his last effort announced its intent to merge.

Rajiv Shukla is intending to take a third lucky winner public with Alpha Healthcare Acquisition III, filing to go public Thursday with a $150 million raise penciled in. The move comes just a couple of weeks after Shukla’s second SPAC said it would jump to Nasdaq in tandem with Laura Niklason’s Humacyte in a $255 million new investment.

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How does Paul Hud­son's $13.5M comp pack­age stack up against oth­er CEOs? He's in the 'first quar­tile'

Paul Hudson arrived at Sanofi like a hurricane, chopping off duds in the pipeline, shaking up the C-suite, striking big M&A deals and jumping into the Covid-19 vaccine race — all in an attempt to reboot a pharma giant notorious for its setbacks.

Now, we’re getting a look at what the CEO brought home in his first year on the job.

When all is said and done, Hudson will have made about $6.7 million in 2020, about $2.5 million of which has already been paid. The bigger figure includes a $2.3 million bonus that’s subject to approval at an April meeting, and another $1.8 million in variable compensation that has yet to be paid.

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Af­ter three years of courtship (and turn­downs), Mer­ck pounced on the first glance of clin­i­cal da­ta in $1.85B Pan­dion takeover

It’s almost become cliché for biotech executives to talk about the importance of keeping your options open and being prepared to go all the way. But when it comes to negotiating with a giant like Merck, a little patience can indeed go a long way.

Just ask Pandion Therapeutics.

Days ago we already learned that Merck is shelling out $1.85 billion to pick up the biotech and its slate of autoimmune hopefuls. What we didn’t know until the SEC disclosure dropped Thursday is that the deal comes after Pandion turned down two other proposals from Merck over the past three years and held out until the last minute for a sweetened deal.

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