CEO Carmine Stengone (Pipeline Therapeutics)

Ver­sant spin­out lands $80M to go 'much broad­er' than the old Roche part­ner­ship that birthed it

About three years af­ter split­ting from Ver­sant Ven­tures’ dis­cov­ery group, Pipeline Ther­a­peu­tics has land­ed an $80 mil­lion Se­ries C round to go “much broad­er” than the Roche-part­nered pro­gram that birthed it.

Daniel Lor­rain

Ver­sant an­nounced Pipeline’s launch in 2018, four years af­ter Roche agreed to pay the re­search tab at Ver­sant’s In­cep­tion Sci­ences for re­myeli­na­tion work in mul­ti­ple scle­ro­sis. Re­myeli­na­tion in­volves a re­pair to the nerve dam­age that caus­es MS. Roche took over one of the pro­grams, called In­cep­tion 5, and the team that helped de­vel­op it was spun out in­to Pipeline, in­clud­ing cur­rent CSO Daniel Lor­rain.

“When that was done, the as­pi­ra­tions for Pipeline were broad­er than that,” CEO Carmine Sten­gone said of Roche’s In­cep­tion 5 ac­qui­si­tion. “The frame­work was around re­myeli­na­tion to start, but the vi­sion for the com­pa­ny was much broad­er.”

Pipeline is con­tin­u­ing the re­myeli­na­tion work in MS, but al­so ex­pand­ing in­to ax­on­al re­pair and synap­to­ge­n­e­sis. Their lead can­di­date, PIPE-505, fo­cus­es on the lat­ter.

PIPE-505 is cur­rent­ly en­rolling pa­tients in a Phase I/IIa study for sen­sorineur­al hear­ing loss, which af­fects up­wards of 40 to 45 mil­lion peo­ple in the US, ac­cord­ing to Sten­gone. The neu­rode­gen­er­a­tive con­di­tion is char­ac­ter­ized by two main hear­ing prob­lems, the first of which is out­er hair cell de­gen­er­a­tion.

As you lose out­er hair cells, you lose vol­ume con­trol, mean­ing every­thing’s qui­eter, Sten­gone said. There’s less speech clar­i­ty and less am­pli­fi­ca­tion. But in Sten­gone’s opin­ion, that’s not where the most un­met need is. The biggest com­plaint pa­tients have is that they can’t hear when there’s back­ground noise. And to ad­dress that, one must look to the cochlear synapse.

The cochlear synapse con­nects the au­di­to­ry nerve fibers to the in­ner hair cells, and over time — ei­ther due to age or pro­longed noise ex­po­sure — there’s a re­trac­tion of that synapse, Sten­gone ex­plained.

“What we’re do­ing with PIPE-505 is re-en­gag­ing guid­ance cues that al­low for that synapse to reach out that 500 mi­crons to ful­ly form that cir­cuit. And once you re­store the cochlear synapse, you should have an im­prove­ment in speech and noise pro­cess­ing,” the CEO said.

There are cur­rent­ly no drugs ap­proved for SNHL, mean­ing pa­tients are lim­it­ed to hear­ing aids and cochlear im­plants, which don’t quite ad­dress the un­der­pin­nings of hear­ing loss. The bur­geon­ing field in­cludes Fre­quen­cy Ther­a­peu­tics, which read out longer-term dura­bil­i­ty da­ta to back its SNHL can­di­date, which us­es prog­en­i­tor cells, in Sep­tem­ber. The new da­ta build on Phase I/II re­sults re­leased in 2019. Then there’s Ak­ou­os, which scored a $105 mil­lion Se­ries B a year ago to take its lead SNHL can­di­date to the clin­ic. And Deci­bel Ther­a­peu­tics, an­oth­er Boston-based com­pa­ny, has an SNHL pro­gram in the dis­cov­ery phase.

Pipeline’s sec­ond can­di­date, a re­myeli­na­tion pro­gram for MS dubbed PIPE-307, re­cent­ly en­tered Phase I. They’ve al­so got an LPA1 re­cep­tor an­tag­o­nist pro­gram for re­myeli­na­tion and neu­roin­flam­ma­tion, for which they’re look­ing to nom­i­nate a de­vel­op­ment can­di­date in mid-2021.

In ad­di­tion to ad­vanc­ing the pipeline, Sten­gone plans on us­ing Se­ries C funds to bulk up Pipeline’s staff. The 22-per­son com­pa­ny is look­ing at four new hires in the near term, he said.

As for the pos­si­bil­i­ty of go­ing pub­lic in the near fu­ture, Sten­gone said, “We’ll con­sid­er all fi­nanc­ing op­tions go­ing for­ward, but noth­ing that we would lock our­selves down to at this point.”

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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In the lat­est big in­vest­ment in gene ther­a­py man­u­fac­tur­ing, Bio­gen com­mits $200M to a ma­jor new fa­cil­i­ty in NC

You’d be forgiven for thinking that the only R&D effort of any consequence at Biogen belongs to aducanumab, its controversial Alzheimer’s drug. But behind the uproar around that drug, the big biotech has a full scale pipeline in play that includes a growing focus on developing gene therapies.

Now Biogen plans to build up the kind of manufacturing muscle that will give it an advantage in gaining FDA approvals — where CMC is always key — and then marketing them around the world.

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Angie You (file photo)

With $117M in fresh cash, Amu­nix paves path to the clin­ic for 'u­ni­ver­sal mask' pro­drug on the hunt for HER2 tu­mors

Despite all the excitement over the possibility of T cell therapies to crack the code against solid tumors, early safety data have limited the burgeoning field’s promise. A clutch of players hope to solve that problem by “masking” their drugs, and now one of those outfits has snared fresh investor cash to take its shot in the clinic.

Amunix closed a $117 million Series B to guide AMX-818, a masked protease-activated T cell engager for HER2 expressing tumors, to the clinic as well as shepherding its bustling pipeline of tumor hunters through the preclinical stage, the company said Thursday.

Andrew Hopkins, Exscientia

Black­Rock push­es Ex­sci­en­tia Se­ries C to $100M as AI biotech boom con­tin­ues

The jury’s still out on whether the first wave of AI companies can significantly change drug development, but investors are increasingly buying into the hype.

Exscientia, the decade-old UK machine learning outfit, announced Thursday that they’ve expanded their Series C, first announced in May, from $60 million to $100 million. The expansion most notably includes BlackRock, the private equity firm that has been wading deeper and deeper into biotech. They now join Novo Holdings, Bristol Myers Squibb and others among the company’s most recent backers.

Eli Lil­ly claims a TKO in its long-run­ning ti­tle fight with No­vo Nordisk for the block­buster di­a­betes mar­ket — but there’s a hitch

Eli Lilly isn’t just gunning for a better diabetes drug in tirzepatide. They want to cut ahead of Novo Nordisk’s blockbuster rival Ozempic (semaglutide) on the obesity front as well. But a newly-claimed win in a head-to-head Phase III showdown over reducing A1C while shedding pounds — complete with clear evidence of superiority over the approved rival — could prove a tough sell right now.

Let’s start with the latest data from Lilly.

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In­tro­duc­ing End­points FDA+, our new pre­mi­um week­ly reg­u­la­to­ry news re­port led by Zachary Bren­nan

CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.

Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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