Rajesh Devraj, Rectify Pharmaceuticals CEO

At­las backs a long­time Ver­tex em­ploy­ee’s quest to bring CF suc­cess to nu­mer­ous oth­er dis­eases

One of Ver­tex’s longest-tenured em­ploy­ees be­lieves he can take the biotech’s biggest med­ical and sci­en­tif­ic ac­com­plish­ments and use it to de­vel­op treat­ments for more than just cys­tic fi­bro­sis.

Three years ago, Jonathan Moore, a sci­en­tist and then ex­ec­u­tive at Ver­tex from 1990 to 2018, found­ed a com­pa­ny to de­vel­op treat­ments for dis­eases that, like CF, are caused by mu­ta­tions in a “su­per fam­i­ly” of pro­teins known as ABC trans­porters.

Jonathan Moore

He’s since man­aged to con­vince a few in­vestors. On Thurs­day, Moore, At­las Ven­ture and a hand­ful of oth­er blue-chip funds de­buted Rec­ti­fy Phar­ma­ceu­ti­cals, backed with $100 mil­lion in Se­ries A funds to find small mol­e­cules that can cor­rect such mu­ta­tions in a broad suite of ge­net­ic dis­or­ders. They’ll start in the liv­er, but even­tu­al­ly plan to hit dis­eases in near­ly every or­gan in the body.

“These are ex­pressed in all dif­fer­ent or­gans, liv­er, lungs, brain, and they play an im­por­tant role in the body,” CEO Ra­jesh De­vraj told End­points News. “You can imag­ine the amount of tar­gets that are avail­able and de­liv­ered for us to go af­ter.”

The new com­pa­ny builds off Ver­tex’s suc­cess in de­vel­op­ing a type of drug that es­sen­tial­ly had nev­er been built be­fore, a small mol­e­cule that can take a mu­tant, dys­func­tion­al pro­tein and morph it back in­to a func­tion­al one.

That work, con­ceived of and fund­ed in part by the CF Foun­da­tion, ul­ti­mate­ly led to four it­er­a­tions of mol­e­cules and com­bi­na­tions of mol­e­cules that over a decade turned one of the most com­mon ge­net­ic dis­eases from a like­ly death sen­tence in­to a treat­able con­di­tion for most pa­tients.

The pro­tein be­hind CF, known as CFTR, is not unique, though. It’s a trans­port pro­tein, shep­herd­ing salts in­to and out of cells. And the con­stant­ly whirling, busy, huffy place that is the hu­man body has many such trans­port pro­teins — 48 to be ex­act. Ac­cord­ing­ly, hu­mankind has many dis­eases as­so­ci­at­ed with mu­ta­tions in those pro­teins.

There are nu­mer­ous ben­e­fits to go­ing af­ter this class of dis­ease, De­vraj points out: They are mono­genic and re­searchers can be cer­tain that, if they suc­cess­ful­ly cor­rect the pro­tein, they can slow or re­verse the dis­ease; and it’s been done be­fore, in­clud­ing by Rec­ti­fy’s founder.

Small mol­e­cules are al­so eas­i­er to de­vel­op, make and de­liv­er than gene ther­a­py or oth­er bi­o­log­ic drugs, es­pe­cial­ly for rare dis­eases that of­ten get over­looked by drug de­vel­op­ers.

“We are — I think we are the foun­da­tion,” De­vraj said. “We are the lead­ers in ABC trans­porter bi­ol­o­gy.”

But there are al­so key hur­dles. Al­though Ver­tex proved that one could de­vel­op a small mol­e­cule to trans­form a mu­tant pro­tein, no one has yet done it suc­cess­ful­ly for any oth­er dis­ease. The big biotech has had its own trou­bles ap­ply­ing the strat­e­gy to a new con­di­tion.

Among the biggest chal­lenges with these dis­eases, De­vraj ac­knowl­edged, is that of­ten dif­fer­ent pa­tients with a sin­gle dis­ease have dif­fer­ent ge­net­ic mu­ta­tions. Like words, genes can be mis­pelled in any num­ber of ways.

That means Rec­ti­fy will po­ten­tial­ly need dif­fer­ent drugs or dif­fer­ent com­bi­na­tions of drugs to fix all or most of the mu­ta­tions in a giv­en dis­ease. It took Ver­tex a decade to find the right tri­ad of mol­e­cules that work for 90% of pa­tients’ CF and it could be even hard­er, both sci­en­tif­i­cal­ly and eco­nom­i­cal­ly, to do the same for dis­or­ders that are even more rare — as the ones Rec­ti­fy pur­sues will al­most cer­tain­ly be.

To over­come those chal­lenges, Rec­ti­fy is ap­ply­ing some of the same tricks Ver­tex did, in­clud­ing de­vel­op­ing tests that, in the­o­ry, could al­low them to quick­ly tell in a lab whether a mol­e­cule is hav­ing its in­tend­ed ef­fect and restor­ing ship­ping lanes in­to and out of cells. De­vraj said they have al­ready de­vel­oped the first as­says but de­clined to de­scribe them.

The com­pa­ny is keep­ing its in­di­ca­tions undis­closed for now, too, ex­cept that they will start in the liv­er, be­fore hope­ful­ly ex­pand­ing to the lung and cen­tral ner­vous sys­tem. The cur­rent fi­nanc­ing, co-led by Omega, For­bion and Long­wood, should get them through proof-of-con­cept clin­i­cal tri­als for the first mol­e­cule.

“Noth­ing is go­ing to be easy,” De­vraj said. That’s “why we do this job.”

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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Ugur Sahin, AP Images

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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