Gili Hart

CF Foun­da­tion backs a new round for a biotech up­start out to ri­val the mighty Ver­tex

As a post­doc fel­low in 1989, Bat­she­va Kerem was part of the dogged team that worked with cur­rent NIH di­rec­tor Fran­cis Collins to iden­ti­fy the faulty gene be­hind cys­tic fi­bro­sis. Now, more than three decades lat­er, she’s lead­ing one com­pa­ny’s ef­forts to al­ter a key mu­ta­tion in that gene — and she just got $28.5 mil­lion to do it.

On Thurs­day morn­ing, Jerusalem-based SpliSense took the wraps off a Se­ries B round to bring its an­ti­sense oligonu­cleotide (ASO) ther­a­py in­to the clin­ic. The round in­cludes up to $8.4 mil­lion from the Cys­tic Fi­bro­sis Foun­da­tion, bring­ing SpliSense’s to­tal raise to just over $30 mil­lion.

“It’s an in­trigu­ing tech­nol­o­gy that we re­al­ly haven’t in­vest­ed in with oth­er com­pa­nies and groups, so we thought this would be a very unique prospect,” CF Foun­da­tion ex­ec­u­tive VP and CSO William Skach told End­points News. The CF Foun­da­tion award­ed $400,000 to get SpliSense go­ing back in 2017, and Skach said it con­tributed again be­cause it was “very pleased” with ear­ly re­sults.

CF is caused by mu­ta­tions in the CFTR gene, which is re­spon­si­ble for shut­tling chlo­ride in­to and out of cells. It’s a de­bil­i­tat­ing dis­ease that leads to fre­quent lung in­fec­tions, breath­ing dif­fi­cul­ties, and short­er lifes­pan. SpliSense’s ap­proach us­es ASOs (short se­quences of RNA ad­min­is­tered by in­hala­tion) to cor­rect mu­ta­tions in CFTR mR­NA, thus dri­ving the cell to pro­duce func­tion­al CFTR pro­teins.

“Our tech­nol­o­gy ad­dress­es the un­der­ly­ing ge­net­ic cause, there­by of­fer­ing, for the first time, hope of restor­ing ad­e­quate lung func­tion to CF pa­tients,” CEO Gili Hart said in a state­ment. She was brought in about a year and a half ago to help Kerem, found­ing CSO, build the com­pa­ny.

The com­pa­ny’s pre­clin­i­cal can­di­date, SPL84-23, is de­signed to cor­rect the 3849+10kb C->T CFTR mu­ta­tion, and is head­ed for a Phase I/IIa tri­al in 2022. The goal is to treat the small por­tion of pa­tients with rare mu­ta­tions who don’t ben­e­fit enough or at all from Ver­tex’s al­ready ap­proved CFTR mod­u­la­tors.

“The pro­gram we’re fund­ing right now is look­ing at a mu­ta­tion which is par­tial­ly re­spon­sive to Ver­tex drugs, but we still think there could be more re­sponse, and a bet­ter re­sponse for peo­ple that could car­ry those mu­ta­tions,” Skach said.

There are a cou­ple oth­er RNA-fo­cused biotechs in the CF space, in­clud­ing Ar­row­head, which is us­ing RNA in­ter­fer­ence as op­posed to an­ti­sense. Io­n­is scrapped its own ear­ly-stage ef­fort this week, af­ter a long-term tox­i­col­o­gy in an­i­mals turned up con­cern­ing re­sults. Trans­late Bio has its own in­hal­able mR­NA can­di­date, but faced a set­back in March when it re­port­ed that the drug had vir­tu­al­ly no ef­fect on pa­tients’ lung func­tion.

“ASO is very sta­ble … with­in the body, with­in the cells, so you can give it less fre­quent­ly. It’s small, it can eas­i­ly pen­e­trate,” Hart told End­points. “We be­lieve our tech­nol­o­gy is very clean, sim­ple and unique.”

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

James Peyer, Cambrian CEO

Brent Saun­ders joins $100M Se­ries C for a com­pa­ny out to be the Bridge­Bio of ag­ing

About a year ago, James Peyer, a CEO and co-founder of the little known longevity biotech Cambrian Biopharma, was trying to find some R&D talent last year when he met with more than a bit of experience in that department: David Nicholson, the former R&D chief of the erstwhile pharma giant Allergan.

It turned out Nicholson already had an interest in Peyer’s field. In their Allergan days, he and COO Brent Saunders held weekly meetups where they tried to figure out how to take the company’s dominance in aesthetics — which, until recently, was often what people meant by anti-aging science — and expertise with more traditional drug development, and use it to make drugs that extend people’s lifespan.

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As pres­sure to share tech­nol­o­gy mounts, BioN­Tech se­lects Rwan­da for lat­est vac­cine site

BioNTech’s first mRNA-based vaccine site in Africa will call Rwanda home, and construction is set to start in mid-2022, the company announced Tuesday at a public health forum.

The German company signed a memorandum of understanding, after a meeting between Rwanda’s Minister of Health, Daniel Ngamije, Senegal’s Minister of Foreign Affairs Aïssata Tall Sall, and senior BioNTech officials. Construction plans have been finalized, and assets have been ordered. The agreement will help bring end-to-end manufacturing to Africa, and as many as several hundred million doses of vaccines per year, though initial production will be more modest.

No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

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Katie Fanning, Mozart Therapeutics CEO

Mozart Ther­a­peu­tics makes its of­fi­cial de­but, jump­ing in­to the hot Treg R&D field with some big-name in­vestors back­ing it

Treg cells have been getting more and more attention recently among autoimmune specialists. There’s been Jeff Bluestone’s Sonoma, the $157 million launch of GentiBio this summer and Egle Therapeutics — which launched just last week — to name a few.

Now, there’s a new Treg player jumping in that wants to distinguish itself in the market: Mozart Therapeutics. Today, the biotech is emerging from stealth in its official debut with a $55 million Series A — with a bunch of A-list Big Pharma names on board a syndicate led by ARCH.

Vas Narasimhan, Novartis CEO (Simon Dawson/Bloomberg via Getty Images)

With San­doz con­tin­u­ing to drag on No­var­tis, Vas Narasimhan says he may fi­nal­ly be ready for a sale or spin­off

After years of rehab work aimed at getting Sandoz in fighting trim to compete in a market overshadowed by declining prices, CEO Vas Narasimhan took a big step toward possibly selling or spinning off the giant generic drug player.

The pharma giant flagged plans to launch a strategic review of the business in its Q3 update, noting that “options range from retaining the business to separation.”

Analysts have been poking and prodding Novartis execs for years now as Narasimhan attempted to remodel a business that has been a drag on its performance during most of his reign in the CEO suite. The former R&D chief has made it well known that he’s devoted to the innovative meds side of the business, where they see the greatest potential for growth.

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FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

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