L to R: Jeroen Bekaert, Ivan Dimov and Nate Fernhoff, Orca Bio (Orca)

Months af­ter achiev­ing uni­corn sta­tus, Or­ca steers in­to the fast lane with an RMAT des­ig­na­tion for cell ther­a­py can­di­date

A few months af­ter its last big catch — a $192 mil­lion Se­ries D and rare uni­corn sta­tus — Or­ca Bio has reeled in the FDA’s re­gen­er­a­tive med­i­cine ad­vanced ther­a­py (RMAT) des­ig­na­tion for its ex­per­i­men­tal cell ther­a­py to treat blood stem cell trans­plant pa­tients.

Or­ca, one of this year’s End­points 11 star­tups, was found­ed by three en­tre­pre­neurs who were in­trigued by Irv Weiss­man’s cell pu­rifi­ca­tion work while study­ing at Stan­ford. No­var­tis — then San­doz — had pur­chased Weiss­man’s spin­out SyS­temix in the ’90s, then scrapped it due to prac­ti­cal chal­lenges. But Jeroen Bekaert, Ivan Di­mov and Nate Fern­hoff saw po­ten­tial there.

Now, the biotech has RMAT and or­phan drug des­ig­na­tion to steer its cell ther­a­py can­di­date Or­ca-T through the clin­ic. RMAT des­ig­na­tion comes with the ben­e­fits of both fast track and break­through ther­a­py des­ig­na­tions. Or­ca-T en­tered a Phase I/II tri­al in pa­tients un­der­go­ing stem cell trans­plants for var­i­ous blood can­cers last No­vem­ber, ac­cord­ing to an NIH fil­ing. The biotech ex­pects to read out da­ta from that tri­al this De­cem­ber at the an­nu­al Amer­i­can So­ci­ety of Hema­tol­ogy meet­ing.

“The RMAT and ODD for Or­ca-T is up­lift­ing news for pa­tients with var­i­ous blood can­cers in­clud­ing acute myeloid leukemia, acute lym­phoid leukemia, myelodys­plas­tic syn­drome and myelofi­bro­sis,” Di­mov said in a state­ment.

While a hematopoi­et­ic stem cell trans­plant is the best op­tion for some pa­tients with blood can­cer, the pro­ce­dure can lead to dan­ger­ous com­pli­ca­tions, such as graft-ver­sus-host dis­ease, in which im­mune cells from the donor at­tack the pa­tient’s healthy cells. Or­ca-T is de­signed to re­pro­gram the dis­eased blood sys­tem, by match­ing “a spe­cif­ic com­po­si­tion of stem and im­mune cells to the im­muno­log­i­cal needs of a pa­tient.”

Or­ca’s re­cent Se­ries D brought its to­tal raise to about $300 mil­lion, and came in as the com­pa­ny’s val­u­a­tion sur­passed $1 bil­lion. Plus, the biotech touts Weiss­man, for­mer No­var­tis CEO Joe Jimenez, and Lyell head and for­mer NCI chief Rick Klaus­ner as its ad­vi­sors.

Di­mov spoke with End­points News last month about the in­spi­ra­tion be­hind the biotech’s 2016 launch:

It al­most seems un­eth­i­cal from a hu­man per­spec­tive be­cause some of the ini­tial re­sults sug­gest high­ly cu­ra­tive ef­fects and the whole thing was shut down for over a decade be­cause of some of these chal­lenges of prac­ti­cal­ly im­ple­ment­ing these things and de­ploy­ing them. So that was sort of the key dri­ver for us to get start­ed. And we al­ways knew we want­ed to ful­fill that dream all the way through and not re­al­ly stop at a cer­tain stage and leave it for some­one else.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Ex­elix­is pulls a sur­prise win in thy­roid can­cer just days ahead of fi­nal Cabome­tyx read­out

Exelixis added a thyroid cancer indication to its super-seller Cabometyx’s label on Friday — months before the FDA was expected to make a decision, and days before the company was set to unveil the final data at #ESMO21.

At a median follow-up of 10.1 months, differentiated thyroid cancer patients treated with Cabometyx (cabozantinib) lived a median of 11 months without their disease worsening, compared to just 1.9 months for patients given a placebo, Exelixis said on Monday.

Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

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Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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Rafaèle Tordjman (Jeito Capital)

Con­ti­nu­ity and di­ver­si­ty: Rafaèle Tord­j­man's women-led VC firm tops out first fund at $630M

For a first-time fund, Jeito Capital talks a lot about continuity.

Rafaèle Tordjman had spotlighted that concept ever since she started building the firm in 2018, promising to go the extra mile(s) with biotech entrepreneurs while pushing them to reach patients faster.

Coincidentally, the lack of continuity was one of the sore spots listed in a report about the European healthcare sector published that same year by the European Investment Bank — whose fund is one of the LPs, alongside the American pension fund Teacher Retirement System of Texas and Singapore’s Temasek, to help Jeito close its first fund at $630 million (€534 million). As previously reported, Sanofi had chimed in €50 million, marking its first investment in a French life sciences fund.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Mi­rati tri­umphs again in KRAS-mu­tat­ed lung can­cer with a close­ly watched FDA fil­ing now in the cards

After a busy weekend at #ESMO21, which included a big readout for its KRAS drug adagrasib in colon cancer, Mirati Therapeutics is ready to keep the pressure on competitor Amgen with lung cancer data that will undergird an upcoming filing.

In topline results from a Phase II cohort of its KRYSTAL-1 study, adagrasib posted a response rate of 43% in second-line-or-later patients with metastatic non-small cell lung cancer containing a KRAS-G12C mutation, Mirati said Monday.