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.”

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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.

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.

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.