Part pro­ce­dure, part drug: Robert Ang joins Sid­dhartha Mukher­jee in pi­o­neer­ing a new type of cell en­gi­neer­ing

In a re­cent chron­i­cle on the promise and price of cell ther­a­pies, Sid­dhartha Mukher­jee — au­thor, on­col­o­gist and Co­lum­bia pro­fes­sor among his oth­er ti­tles — mused about how en­gi­neered T cells had blurred the tra­di­tion­al bound­aries sep­a­rat­ing a pro­ce­dure and a drug. “Pro­ce­dures come alive in the tin­ker­ing, fuss­ing hands of their op­er­a­tors,” he ob­served, while a “drug, in con­trast, is a de­per­son­al­ized en­ti­ty.” For the new gen­er­a­tion of metic­u­lous­ly man­u­fac­tured CAR-T to reach the mass­es, in­no­va­tors must mar­ry the it­er­a­tive na­ture of a pro­ce­dure with the pro­duc­tion ef­fi­cien­cy of a drug — and add a ruth­less pur­suit of the lean­est, cheap­est process pos­si­ble.

Robert Ang

He’s putting that con­cept to work in a new ven­ture. Vor Bio­phar­ma, a biotech fledg­ling bred at PureTech and now set­tling in­to a new nest at Kendall Square, is en­gi­neer­ing hematopoi­et­ic stem cells to rid them of the anti­gen CD33 so that acute myeloid leukemia pa­tients may reap the ben­e­fits of CD33- tar­get­ed ther­a­pies with­out the tox­i­c­i­ties.

What Mukher­jee came up with is an “ex­treme­ly el­e­gant” ap­proach, says Robert Ang, who’s start­ing his first week as CEO of Vor. The prod­uct, VOR33, would in ef­fect shield healthy blood cells and the bone mar­row from a T cell at­tack.

Sid­dhartha Mukher­jee

An ex vi­vo pro­ce­dure in­volv­ing a sin­gle ge­net­ic ed­it of healthy donor cells rep­re­sent “ide­al en­try points for CRISPR-based ther­a­peu­tics, and that’s pre­cise­ly what VOR33 is,” he says. “And so we ex­pect the pro­duc­tion process to be rel­a­tive­ly straight­for­ward with on­ly sev­er­al-day process but of course you can nev­er take that for grant­ed.”

“I’ve just been itch­ing to get go­ing,” adds the biotech vet­er­an, who had keen­ly learned lessons about com­pa­ny build­ing as em­ploy­ee #5 and chief busi­ness of­fi­cer of neoanti­gen play­er Neon Ther­a­peu­tics.

Kush Par­mar

Soon af­ter he start­ed pon­der­ing about lead­ing a com­pa­ny of his own he got in touch with Kush Par­ma, man­ag­ing part­ner at 5AM Ven­tures and Vor chair­man who’s al­so work­ing with Mukher­jee on an­oth­er CAR-T project. 5AM was joined by RA Cap­i­tal Man­age­ment, Os­age Uni­ver­si­ty Part­ners and PureTech, as well as J&J’s ven­ture arm and No­var­tis In­sti­tutes for Bio­Med­ical Re­search, for a $42 mil­lion Se­ries A an­nounced ear­li­er this year.

It’s still ear­ly days — so ear­ly that he’s not ready to share how many are on the team — but Ang al­so has a clear time­line for en­ter­ing the clin­ic in 18 months. A more near-term goal would be to sit down with the FDA about an IND by the end of the year.

“We want to make sure that the prod­uct is safe, and that the prod­uct en­grafts just like oth­er typ­i­cal trans­plants,” he says of the first hu­man tri­al they are work­ing to­ward. “But at the same time we’re al­so very in­ter­est­ed to see what hap­pens when the pa­tients — if they do progress, how the trans­plants be­have when tar­get­ed ther­a­py is ap­plied.”

The com­par­i­son to stan­dard-of-care would be cru­cial as Vor proves that it’s just as good, if not bet­ter than the bone mar­row trans­plants that AML pa­tients would oth­er­wise have re­ceived.

As for the sub­se­quent tar­get­ed ther­a­py that VOR33 would dove­tail in­to, the sea­soned BD ex­ec main­tains there’s a good amount of op­tion­al­i­ty as to which modal­i­ty they go with. So far Pfiz­er claims the on­ly mar­ket­ed drug hit­ting CD33 in My­lotarg, and a slate of oth­ers are in de­vel­op­ment. Nonethe­less, he notes that J&J and No­var­tis both have strong pipelines in tar­get­ed ther­a­py and CAR-T.

It’s not just CD33, ei­ther. Vor plans to ap­ply the same gene-edit­ing ap­proach to a num­ber of tar­gets, cre­at­ing com­pan­ion pro­ce­dures for mul­ti­ple tar­get­ed ther­a­pies.

Ang, who trained as a doc­tor in Aus­tralia and had stints at both Boston Con­sult­ing Group and Fra­zier Health­care Ven­tures be­fore tak­ing on front­line biotech roles, sees build­ing up the cul­ture at Vor as one of his key tasks.

“Neon’s cul­ture is ab­solute­ly in­cred­i­ble, I’d love to em­u­late that, and al­so build on the great cul­ture that Vor it­self has cre­at­ed over the past few months,” he says. “Vor peo­ple are high­ly col­lab­o­ra­tive, love sci­ence, but are al­so dri­ven by a high­er pur­pose. So build­ing on that and for­mal­iz­ing that will be re­al­ly im­por­tant in the months ahead.”

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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David Loew (Ipsen)

Ipsen snags an ap­proved can­cer drug in $247M M&A deal as an­oth­er bat­tered biotech sells cheap

You can add Paris-based Ipsen to the list of discount buyers patrolling the penny stock pack for a cheap M&A deal.

The French biotech, which has had plenty of its own problems to grapple with, has swooped in to buy Epizyme $EPZM for $247 million in cash and a CVR with milestones attached to it. Epizyme shareholders, who had to suffer through a painfully soft launch of their EZH2a inhibitor cancer drug Tazverik, will get $1.45 per share along with a $1 CVR tied to achieving $250 million in sales from the drug over four consecutive quarters as well as an OK for second-line follicular lymphoma by 1 Jan. 2028.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Yong Dai, Frontera Therapeutics CEO

Scoop: Lit­tle-known Or­biMed-backed biotech clos­es $160M round to start gene ther­a­py tri­al

Frontera Therapeutics, a China and US biotech, has closed a $160 million Series B and received regulatory clearance to test its first gene therapy stateside, Endpoints News has learned.

Led by the largest shareholder, OrbiMed, the biotech has secured $195 million total since its September 2019 founding, according to an email reviewed by Endpoints. The lead AAV gene therapy program is for an undisclosed rare eye disease, according to the source.

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Joe Papa (Ryan Remiorz/The Canadian Press via AP, File)

Joe Pa­pa re­signs as chair of Bausch Health as bil­lion­aire John Paul­son takes over

Joe Papa, chair of Bausch Health, officially resigned on Thursday and the board appointed billionaire hedge fund manager John Paulson as the new chair, effective immediately.

The specialty pharma company sought to make clear that Papa’s abrupt departure “was not due to any dispute or disagreement with the Company, its management or the Board on any matter relating to the Company’s operations, policies or practices.”

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Matt Kapusta, uniQure CEO

In trou­bled Hunt­ing­ton’s space, uniQure’s gene ther­a­py shows ear­ly promise

In randomized clinical trial data from a small number of patients, Dutch biotech uniQure shared that its gene therapy for Huntington’s disease seems to reduce the amount of the mutant protein responsible for the disease over the course of a year.

In seven patients with early-stage Huntington’s — four who got the treatment and three who got a placebo — mutant huntingtin protein levels in the cerebrospinal fluid decreased by an average of just over 50% in patients who got the gene therapy compared to around a 17% drop in patients who got the placebo after a year.

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DEM BioPharma CEO David Donabedian (L) and executive chair Jan Skvarka

Long­wood sets an­oth­er 'don't eat me' biotech in­to gear with help of for­mer Tril­li­um CEO Jan Skvar­ka

Jonathan Weissman and team are out with a cancer-fighting biotech riding the appetite for those so-called “don’t eat me” and “eat me” signals.

The scientific co-founder — alongside fellow Whitehead Institute colleague Kipp Weiskopf and Stanford biologist Michael Bassik — has launched DEM BioPharma with incubator Longwood Fund and a crop of other investors.

In all, the nascent, 10-employee biotech has $70 million to bankroll hematology- and solid tumor-based programs, including a lead asset that could enter human trials in two to three years, CEO David Donabedian told Endpoints News.

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GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.