Vincent Sandanayaka (file photo)

Ex-MD An­der­son chief De­Pin­ho is help­ing launch an­oth­er biotech — and he's stick­ing with fa­mil­iar ground

Years af­ter co-found­ing SINE-fo­cused Karyopharm and stir­ring up con­tro­ver­sy at MD An­der­son, Ronald De­Pin­ho is help­ing un­cloak a new biotech tar­get­ing solute car­ri­er trans­porter pro­teins — and Karyopharm’s for­mer head of chem­istry is lead­ing the charge.

Nir­ogy Ther­a­peu­tics emerged from stealth mode on Tues­day with a $16.5 mil­lion Se­ries A round and plans to hit the clin­ic by 2022. The fi­nanc­ing should be enough to car­ry the start­up’s lead pro­gram, a small mol­e­cule lac­tate trans­port in­hibitor, through Phase I, CEO Vin­cent San­danaya­ka said.

San­danaya­ka said he first be­came in­trigued by tu­mor me­tab­o­lism while lead­ing the chem­istry unit at Karyopharm — an­oth­er on­col­o­gy com­pa­ny that De­Pin­ho helped start be­fore join­ing MD An­der­son. That biotech land­ed a Se­ries A in 2010 for its work on se­lec­tive in­hibitors of nu­clear ex­port (SINE), and raised eye­brows last year when its mul­ti­ple myelo­ma drug got ap­proval from the FDA de­spite ob­jec­tions from 8 of the 13 ex­perts on an out­side pan­el.

Af­ter co-found­ing Karyopharm, De­Pin­ho served a six-year tenure as pres­i­dent at MD An­der­son, dur­ing which time the cen­ter re­port­ed op­er­at­ing loss­es of more than $460 mil­lion over 16 months and hun­dreds of staffers were laid off. He stepped down in 2017.

But while many re­searchers in the space were go­ing af­ter in­tra­cel­lu­lar tar­gets, not many were work­ing on solute car­ri­er trans­porters — mem­brane-bound pro­teins that block or reg­u­late metabo­lites like glu­cose, San­danaya­ka said. Solute car­ri­er trans­porter pro­teins (SLCTs) act as gate­keep­ers of im­por­tant phys­i­o­log­i­cal func­tions, in­clud­ing nu­tri­ent up­take and metabo­lite dis­pos­al. The pro­teins are aber­rant­ly al­tered in many dis­eases — but they’re dif­fi­cult to iso­late, and thus dif­fi­cult to study.

“If you take these trans­porters out of the mem­brane, like if you’re try­ing to iso­late it, you may lose the func­tion­al state of the pro­tein,” San­danaya­ka said. Nir­ogy gets around the is­sue by us­ing com­pu­ta­tion­al mod­el­ing.

The CEO found­ed Nir­ogy in 2014 and reached out to De­Pin­ho in 2019, the same year the start­up got some seed fund­ing. Their lead pro­gram takes a two-pronged ap­proach to fight can­cer. Un­like nor­mal cells, can­cer cells con­sume large amounts of glu­cose, and ex­crete lac­tic acid in­to the tu­mor mi­croen­vi­ron­ment through lac­tate trans­porters. This cre­ates a hos­tile en­vi­ron­ment for im­mune cells to sur­vive.

But if that lac­tic acid isn’t re­leased, it ac­cu­mu­lates, caus­ing the cell to die, San­danaya­ka said. Nir­ogy’s can­di­date fights can­cer cells by in­hibit­ing them from ex­pelling the lac­tate, while al­so boost­ing im­mune re­sponse.

San­danaya­ka be­lieves this “one-two punch” is what dif­fer­en­ti­ates it from Jnana Ther­a­peu­tics, which struck an al­liance with Roche in Ju­ly to ex­plore SLCT tar­gets in im­mune-me­di­at­ed and neu­ro­log­i­cal dis­eases. That deal starts small at $40 mil­lion, but can add up to $1 bil­lion if the phar­ma part­ner com­mits to de­vel­op­ing the pro­grams.

“As you might imag­ine for a lot of drugs can­cer cells de­vel­op re­sis­tance. So that’s part of the prob­lem, it may be ac­tive ini­tial­ly, but then can­cer cells de­vel­op a re­sis­tance… So, that is why we thought we want to have a mul­ti-pronged ap­proach to ad­dress this drug re­sis­tance,” San­danaya­ka said.

The start­up plans to test the lac­tate in­hibitor both as a monother­a­py and com­bi­na­tion ther­a­py, and has done pre­clin­i­cal mod­els in triple neg­a­tive breast can­cer, melanoma, and col­orec­tal can­cer, in com­bi­na­tion with an­ti-PD1 and an­ti-CT­LA4 im­munother­a­pies.

In ad­di­tion to the lead pro­gram, Nir­ogy has a sec­ond trans­porter tar­get in can­cer and a third pro­gram in im­munol­o­gy. The tiny five-per­son com­pa­ny could grow to 15-20 peo­ple over the next few months, San­danaya­ka 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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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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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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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.”

Lorenz Mayr, Vector BioPharma CEO (PhoreMost)

Up­dat­ed: 'Close to the mid­dle': Ver­sant launch­es Vec­tor with 'gut­less ade­n­ovirus' de­liv­ery method

Most of the gene therapy world centers around viral vectors, but a few biotechs have sprung up in recent years to try and avoid the safety issues that can come with adeno-associated viruses.

A new biotech has emerged to straddle somewhere “close to the middle,” in the words of Alex Mayweg, Versant managing director and board member of the incubator’s latest startup, Vector BioPharma.