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.”

The FDA hasn’t detected any potential safety signals, including for stroke, in people aged 65 years and older who have received Pfizer’s bivalent Covid booster, one senior official told members of the agency’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Thursday.

The update comes as the FDA and CDC investigate a “preliminary signal” that may indicate an increased risk of ischemic stroke in older Americans who received Pfizer’s updated shot.

The FDA has stopped use of another drug as a result of the new coronavirus variants. On Thursday, the agency announced that AstraZeneca’s antibody combo Evusheld, which was an important prevention option for many immunocompromised people and others, is no longer authorized.

The FDA said it made its decision based on the fact that Evusheld works on fewer than 10% of circulating variants.

Evusheld was initially given emergency authorization at the end of 2021. However, as Omicron emerged, so did studies that showed Evusheld might not work against the dominant Omicron strain. In October, the FDA warned healthcare providers that Evusheld was useless against the Omicron subvariant BA.4.6. It followed that up with another announcement earlier this month that it did not think Evusheld would work against the latest Omicron subvariant XBB.1.5.

Magenta Therapeutics is pausing an early-stage clinical trial after a patient died. The death was deemed to be possibly related to its drug, MGTA-117.

The biotech said the pause of the Phase I/II trial is voluntary and gives it time to review all available data before deciding what to do next. It’s also reported the known information to the FDA.

The dose-escalation trial was designed to test whether MGTA-117, an antibody-drug conjugate, could serve as a more targeted alternative to high-intensity chemotherapy as a conditioning agent for cancer patients who are set to receive a stem cell transplant. It recruited patients with relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome.