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.”

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

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Another day, another SPAC vying for a spot on Nasdaq.

On Wednesday, OncoSec Medical CEO Daniel O’Connor filed the S-1 paperwork for a new blank-check company he’s leading called Larkspur Health Acquisition. The former Advaxis chief penciled in a $75 million raise, with plans to offer 7.5 million shares at $10 apiece.

The Biomedical Advanced Research and Development Authority is cooling its jets in looking for new, potential Covid-19 treatments, at least in the near term.

An HHS spokesperson told Endpoints News via email, “to date, BARDA has obligated more than $9 billion for the development and/or purchase of 13 therapeutics, beginning in February 2020 with support to develop Regeneron’s monoclonal antibody therapeutic. Therapeutics are an important element of the COVID-19 response, and we are focused on the programs currently underway and/or in negotiation using the funds available to us.”

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.