Siddhartha Mukherjee, AP Images

Sid Mukher­jee's Vor taps a CAR-T de­vel­op­ment part­ner to strap to its at­tempt at bet­ter stem cell trans­plants

In the buzzy world of on­col­o­gy, few names shine brighter than Co­lum­bia on­col­o­gist and best­selling au­thor Sid­dhartha Mukher­jee, whose Vor Bio­phar­ma is look­ing to rewrite the rules of stem cell trans­plants. If his team’s tech works as planned, it would al­so rewrite the rules on how CAR-Ts can be de­ployed in blood can­cer — and now it’s part­ner­ing up with a lit­tle-known biotech to test that hy­poth­e­sis.

Robert Ang

Vor has inked a mul­ti-year dis­cov­ery deal with Abound Bio to ac­cess its plat­form of sin­gle- and mul­ti-tar­get CAR-Ts to com­bine with Vor’s pipeline of en­gi­neered blood stem cells, the part­ners said Thurs­day.

The biotechs will ini­tial­ly tar­get acute myeloid leukemia and think that a mul­ti-tar­get­ed CAR-T paired with an HSC trans­plant could be a path to bet­ter treat­ing hard-to-hit blood can­cer. The Pitts­burgh-based on­col­o­gy play­er has shown pre­clin­i­cal da­ta for a CD19/20-tar­get­ed CAR that caught Vor’s in­ter­est, CEO Robert Ang told End­points News.

Fi­nan­cial terms of the deal were not dis­closed.

The prob­lem with us­ing mul­ti-tar­get­ed CARs for years has been the off-tar­get side ef­fects on healthy blood cells. Vor is work­ing on trans­plant­i­ng stem cells in­to pa­tients’ bone mar­row that re­move or mod­i­fy the tar­get­ed anti­gens, the­o­ret­i­cal­ly al­low­ing a CAR con­struct to bet­ter tar­get tu­mors with­out the dele­te­ri­ous safe­ty risks.

Ang was un­equiv­o­cal when talk­ing about Vor’s aims: The biotech is look­ing to com­plete­ly re­place stan­dard-of-care stem cell trans­plants with its plat­form. While he couldn’t dis­cuss what fu­ture mile­stones look like for the Abound deal, Ang said the de­vel­op­ment phase of that pact would run “in par­al­lel” with Vor’s work on its stem cell plat­form.

“(Abound) is re­al­ly a team that’s al­ready demon­strat­ed sci­en­tif­ic prowess in this are­na, and we think it’s a mar­velous op­por­tu­ni­ty to work with them to­wards tar­gets that we can uti­lize in our own ap­proach,” Ang said.

John Mel­lors

Abound Bio was co-found­ed back in 2019 by Dimiter “Mitko” Dim­itrov, the com­pa­ny’s chief sci­en­tif­ic of­fi­cer and a se­nior in­ves­ti­ga­tor at the Na­tion­al Can­cer In­sti­tute, and CEO John Mel­lors. CAR-T ther­a­py re­searcher Ri­mas Orentas serves as the biotech’s VP for cel­lu­lar ther­a­py. On top of its nov­el CD19/20 bis­pe­cif­ic CAR-T, the team re­cent­ly post­ed pre­clin­i­cal da­ta on a trispe­cif­ic CAR con­struct that could show promise far down the road.

Vor, mean­while, is the brain­child of Mukher­jee, who found­ed the com­pa­ny back in 2016 and even­tu­al­ly took it pub­lic in Feb­ru­ary. The com­pa­ny’s lead pro­gram is VOR33, an HSC the biotech is study­ing so­lo and as a pair­ing with CD33 tar­get­ing CAR-T can­di­date VCAR33, which it in-li­censed from the NIH, in IND en­abling stud­ies in AML.

Ang was quick to point out the Abound deal wasn’t to be viewed as an in­ter­rup­tion or re­place­ment for the VOR33/VCAR33 pro­gram, where ini­tial da­ta is ex­pect­ed lat­er in the year to ear­ly next year.

“We’ve worked re­al­ly hard on one tar­get, and that’s CD33 … but to take this to an­oth­er lev­el to look at tar­gets out­side of CD33 and to look at tar­get­ing mul­ti­ple epi­topes … we think that is a re­al­ly at­trac­tive ap­proach,” Ang said.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Who are the women blaz­ing trails in bio­phar­ma R&D? Nom­i­nate them for End­points' 2022 spe­cial re­port

Over the past three years, Endpoints News has spotlighted 60 women who have blazed trails and supercharged R&D across the biopharma world. And judging from the response we’ve received, to both our special reports and live events, telling their stories — including any obstacles they may have had to overcome — has inspired our readers in many different ways.

But change takes time, and the fact remains that women are still underrepresented at the upper ranks of the drug-making world.

Up­dat­ed: Amid mas­sive re­struc­tur­ing, Bio­gen looks to re­duce phys­i­cal pres­ence in Boston

Biogen is putting a sizable chunk of office and research space in Kendall Square and Weston, MA up for sublease, marking another big change as the biotech grapples with the aftershock of a disastrous and controversial rollout for its Alzheimer’s drug.

The subbleases are “part of Biogen’s overall implementation of the ‘Future of Work,’ which is allowing us to optimize our footprint and reduce the amount of space we occupy, taking into consideration new elements such as the hybrid work model,” Biogen spokesperson Ashleigh Koss wrote in a statement to Endpoints News, adding that the company has had subleases across several buildings for years.

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Alessandro Maselli, Catalent CEO

Catal­ent ac­quires North Car­oli­na CD­MO for $475M, boost­ing oral solids work

As Catalent has been expanding its reach in the US this year, as well as recently completing a C-suite shuffle, the company announced last night that it has acquired the CDMO Metrics Contract Services for $475 million from Mayne Pharma Group.

The acquisition will increase Catalent’s capabilities in oral solid formulation development, manufacturing and packaging as well as expand its capacity to handle more highly potent compounds.

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Craig Thompson, Cerevance CEO

UP­DAT­ED: Mer­ck makes first big splash for Alzheimer’s drug R&D since 2017 fail, ink­ing re­search pact with Cere­vance

For the first time since discontinuing its late-stage Alzheimer’s program, Merck has found promise on the path forward in the memory-robbing disease.

After a Phase III flop of its drug verubecestat, the New Jersey Big Pharma axed the study in early 2018. More than four years later, the company is ready to sign up for another pact to test the waters of the befuddling disease.

This time, there’s $1.1 billion in biobucks on the line and a target that its partner says no other biopharma is looking at en route to finding the next treatment for Alzheimer’s, a neuroscience field that has hit hurdle after hurdle for decades.

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Pfiz­er launch­es re­bate pro­gram for rare dis­ease pa­tients who have to stop tak­ing Panzy­ga

Pfizer is launching its second-ever rebate program, this time for Panzyga, its treatment for a rare neurological disease of the peripheral nerves.

The program began last month, according to STAT which first reported the news, and offers a refund of out-of-pocket costs for patients who must discontinue their course before the fifth treatment for “clinical reasons.”

Panzyga was approved back in 2018 to treat primary immunodeficiency (PI) in patients two years and older and chronic immune thrombocytopenia (cITP) in adults. It has since picked up an indication in chronic inflammatory demyelinating polyneuropathy (CIDP), a condition that’s characterized by weakness of the arms or legs, tingling or numbness, and a loss of deep tendon reflexes, according to the NIH.

Horizon's back-to-school campaign for children with cystinosis includes an all about me poster as part of a care package box.

Hori­zon read­ies kids and fam­i­lies for back to school with week­long ac­tiv­i­ties around rare dis­ease cysti­nosis

Going back to school is usually a bumpy readjustment from summer freedom for all kids, but especially for kids with chronic health conditions. Horizon Therapeutics is hoping to help smooth the way for some who have the rare disease cystinosis. Cystinosis is a genetic disease that causes the amino acid cystine to build up in different tissues and organs.

The “Gear Up” for school campaign is running all week with different online and at-home events and activities for families and children with cystinosis. Each family who signed up receives a care package mailed to their home including an activity coloring book “Michael’s Show-and-Tell.” The book tells Michael’s story about living with cystinosis while offering kids matching, coloring and finding object games along with information.

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Phar­mas spend mil­lions on di­a­betes ad­ver­tis­ing, but few pa­tients can re­call brand names — sur­vey

While many Big Pharma diabetes brands spend millions of dollars on TV ads every year, most people with type 2 diabetes don’t recognize specific drug brand names, according to a new study.

No brand garnered more than 30% recognition in Phreesia Life Science’s latest in-office patient survey of more than 4,000 adults with type 2 diabetes. Eli Lilly’s Trulicity topped the list as the most recognized brand with 29% of those surveyed recalling it, followed by Boehringer Ingelheim and Lilly’s Jardiance at 27% and Merck’s Januvia and Novo Nordisk tying for the third spot with 24%. Meanwhile, 76% of the patients surveyed were familiar with the generic treatment metformin.

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Faced with thou­sands of opi­oid law­suits, En­do says it will like­ly file for bank­rupt­cy 'im­mi­nent­ly'

Endo International will likely be the next pharma company to file for bankruptcy under a mountain of opioid lawsuits.

The Dublin, Ireland-based company revealed in its Q2 results on Tuesday that it’s in talks with first lien creditors, and that “these negotiations will likely result in a pre-arranged filing under Chapter 11 of the U.S. Bankruptcy Code by Endo International plc and substantially all of its subsidiaries, which could occur imminently.”