Francisco Salva, Azitra CEO

A small Con­necti­cut biotech throws hat in­to IPO ring

A biotech look­ing to treat skin dis­eases sent in word to the SEC that it wants to go pub­lic.

Az­i­tra joins a rel­a­tive­ly small group of biotechs seek­ing an IPO in the past few quar­ters, with on­ly a hand­ful go­ing pub­lic in re­cent mem­o­ry, in­clud­ing two on back-to-back Fri­days this month.

The biotech’s lead pro­gram is ATR-12, with a Phase Ib to start in the first half of this year for Nether­ton syn­drome, a rare au­to­so­mal dis­ease that is marked by in­flam­ma­tion, scal­ing, and red and de­hy­drat­ed skin that’s caused by mu­ta­tions in the SPINK5 gene. The com­pa­ny says there are about 20,000 pa­tients world­wide with the dis­ease and sees a $250 mil­lion peak sales op­por­tu­ni­ty. The tri­al is ex­pect­ed to read out in the first half of next year. The FDA has as­signed it pe­di­atric rare dis­ease des­ig­na­tion.

Fur­ther down the pipeline are ATR-04 for EGFR in­hibitor as­so­ci­at­ed rash (Phase Ib first half of next year) and ATR-01 for ichthyosis vul­garis (IND fil­ing in late 2024). An ear­ly-stage study test­ing ATR-04 in can­cer ther­a­py-as­so­ci­at­ed rash­es was sus­pend­ed in 2021, ac­cord­ing to a clin­i­cal tri­als data­base up­date. A tri­al in 2019 test­ed it as a cos­met­ic.

The Con­necti­cut biotech, found­ed in 2014, has pulled to­geth­er a li­brary of 1,500 bac­te­r­i­al strains to screen for ther­a­peu­tic char­ac­ter­is­tics.

Lead­ing the com­pa­ny is Fran­cis­co Sal­va, who co-found­ed Ac­er­ta Phar­ma, which sold to As­traZeneca for about $6 bil­lion in 2016. He al­so worked on com­pet­ing BTK in­hibitor mak­er Phar­ma­cyclics for a brief two-year pe­ri­od as cor­po­rate de­vel­op­ment se­nior di­rec­tor from 2008 to 2010. Pri­or to join­ing Az­i­tra in 2021, he was chief ex­ec­u­tive of fi­brot­ic and in­flam­ma­to­ry dis­eases biotech Com­plexa, which “went out of busi­ness” in 2020, per a for­mer em­ploy­ee’s de­scrip­tion on LinkedIn.

Part­ners in­clude Carnegie Mel­lon Uni­ver­si­ty, Yale, Jack­son Lab­o­ra­to­ry for Ge­nom­ic Med­i­cine and Fred Hutch. Az­i­tra al­so has a con­sumer health pro­gram in the works with Bay­er for over-the-counter cos­met­ic prod­ucts. The Big Phar­ma’s VC arm has al­so in­vest­ed $8 mil­lion in the com­pa­ny’s Se­ries B pre­ferred stock.

At the end of last year, Az­i­tra had work­ing cap­i­tal of $1.9 mil­lion and to­tal as­sets of $7.2 mil­lion, per the S1 fil­ing. KdT Ven­tures, Con­necti­cut In­no­va­tions, Bios Part­ners, God­frey Cap­i­tal and Pe­ter Thiel’s Break­out Labs have fi­nanced the biotech. With pro­ceeds from the pro­posed of­fer­ing, Az­i­tra thinks it could op­er­ate for an­oth­er 12 months. The biotech has 10 em­ploy­ees and full-time con­sul­tants, per the SEC pa­per­work.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Ivana Magovčević-Liebisch, Vigil Neuroscience CEO

FDA lifts par­tial clin­i­cal hold on Vig­il Neu­ro­science's TREM2 an­ti­body, re­mov­ing dos­ing cap

When Vigil Neuroscience filed its IPO papers in late 2021, the biotech revealed that the FDA had just cleared its Phase I trial — but with a partial clinical hold that limited dosing to under a certain level.

More than a year later, the FDA has lifted the hold.

Vigil is now free to dose VGL101, an antibody targeting TREM2, at levels higher than 20 mg/kg in its ongoing and future clinical trials in patients with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), an inherited condition that affects the brain and spinal cord.

Cedric Ververken, Confo Therapeutics CEO

Dai­ichi Sankyo inks $183M dis­cov­ery deal with GPCR biotech for CNS tar­get

Belgian biotech Confo Therapeutics has landed $183 million, plus potential royalties, in a drug-discovery deal with Daiichi Sankyo.

Early Thursday, Confo Therapeutics put out word of the deal that will be focused on small molecule antagonists to go after an undisclosed target that the company says is associated with CNS diseases.

Confo CEO Cedric Ververken told Endpoints News that Daiichi originally reached out to learn about the biotech’s technology. He added that Confo, founded in 2015, will use its platform to drug a GPCR target that Daiichi has struggled with internally.

Jeff Bluestone (R), Sonoma Biotherapeutics CEO

Jef­frey Blue­stone brings his start­up haul to $400M+, join­ing forces with Re­gen­eron on cell ther­a­pies

These days, when Jeffrey Bluestone gets together with his contemporaries in science, the conversation often turns to retirement plans.

But a little more than three years ago, Bluestone reached a momentous turning point in his career, exiting a prestigious post at UCSF, where he had spent decades in the scientific pursuit of new therapies. And it had nothing to do with retirement anytime in the near future.

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Dif­fu­sion to hand Nas­daq spot to EIP Phar­ma for PhI­Ib de­men­tia study of ex-Ver­tex drug

One of the more than a dozen bidders for Diffusion Pharmaceuticals’ spot on Nasdaq has prevailed.

Boston biotech EIP Pharma will merge with Diffusion in an all-stock deal, with plans to start a Phase IIb clinical trial in the coming months in a common form of dementia with no approved treatments. The combined company will be renamed CervoMed.

The nine-year-old privately-held EIP is working on a former Vertex drug that it will test in a 160-person Phase IIb in patients with dementia with Lewy bodies, or DLB. The National Institute on Aging is expected to fund that trial with a $21 million grant. With the reverse merger, slated for closing in the middle of this year, EIP will be funded through that readout in the second half of 2024. EIP’s equity and debt holders will own about 77.25% of the combined company.

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Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.