Pos­i­tive piv­otal cell graft da­ta bring No­var­tis-backed Boston biotech step clos­er to fin­ish line

Gami­da Cell — the No­var­tis-backed Is­raeli biotech de­vel­op­ing a cell graft to help pa­tients with blood can­cer ad­dress­ing lim­i­ta­tions as­so­ci­at­ed with bone mar­row trans­plants — has its first taste of late-stage da­ta, paving the way for US ap­proval.

The ther­a­py, called omidu­bi­cel, is com­prised of stem cells, prog­en­i­tor cells as well as den­drit­ic cells ex­tract­ed from the um­bil­i­cal cord. It was test­ed in 125 pa­tients aged 12-65 years suf­fer­ing from high-risk lethal blood can­cers. One group of pa­tients un­der­go­ing a bone mar­row trans­plant re­ceived omidu­bi­cel, while the com­para­tor group was giv­en a stan­dard um­bil­i­cal cord blood trans­plant.

The study was de­signed this way be­cause many pa­tients in need of bone mar­row trans­plants do not get matched donors — and while un­re­lat­ed donor um­bil­i­cal cord blood can pro­vide a suf­fi­cient­ly matched donor to prac­ti­cal­ly all pa­tients, such trans­plants are as­so­ci­at­ed with a high risk of graft fail­ure.

The main goal of the study was time to neu­trophil en­graft­ment: Neu­trophils are the most com­mon type of white blood cell, and en­graft­ment refers to a process by which cells be­gin to grow and re­pro­duce new blood cells. Typ­i­cal­ly, en­graft­ment be­gins about 20 days post a bone mar­row trans­plant, ac­cord­ing to the NIH.

The me­di­an time to neu­trophil en­graft­ment was sta­tis­ti­cal­ly sig­nif­i­cant­ly short­er (p<0.001) for pa­tients who re­ceived omidu­bi­cel at 12 days, ver­sus 22 days in the com­para­tor group, the com­pa­ny said, adding that among pa­tients who were trans­plant­ed per pro­to­col, 96% of pa­tients who re­ceived omidu­bi­cel achieved suc­cess­ful neu­trophil en­graft­ment, com­pared to 88% in the com­para­tor group.

Ju­lian Adams

The graft was well-tol­er­at­ed, and de­tailed da­ta will be dis­closed at a lat­er date.

An ap­pli­ca­tion to mar­ket omidu­bi­cel, which has been grant­ed the FDA’s break­through ther­a­py sta­tus, is ex­pect­ed to be sub­mit­ted on a rolling ba­sis be­gin­ning the fourth quar­ter of this year, said CEO Ju­lian Adams, a well-known sci­en­tist whose biotech ex­pe­ri­ence spans stints at Mil­len­ni­um and In­fin­i­ty.

Shares of the com­pa­ny, which went pub­lic in a $50 mil­lion IPO in 2018, shot up more than 46% to $6.64 in morn­ing trad­ing.

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.

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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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.

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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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.

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.

As­traZeneca, Dai­ichi Sanky­o's ADC En­her­tu blows away Roche's Kad­cy­la in sec­ond-line ad­vanced breast can­cer

AstraZeneca and Japanese drugmaker Daiichi Sankyo think they’ve struck gold with their next-gen ADC drug Enhertu, which has shown some striking data in late-stage breast cancer trials and early solid tumor tests. Getting into earlier patients is now the goal, starting with Enhertu’s complete walkover of a Roche drug in second-line breast cancer revealed Saturday.

Enhertu cut the risk of disease progression or death by a whopping 72% (p=<0.0001) compared with Roche’s ADC Kadcyla in second-line unresectable and/or metastatic HER2-positive breast cancer patients who had previously undergone treatment with a Herceptin-chemo combo, according to interim data from the Phase III DESTINY-Breast03 head-to-head study presented at this weekend’s #ESMO21.

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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.

Covid-19 roundup: In­dia to re­sume vac­cine ex­ports as its own cam­paign is un­der con­trol; Pfiz­er to file for vac­cine OK in chil­dren ages 5-11

After vaccine exports were halted to focus on providing its own people with enough doses, India will begin shipping its Covid-19 vaccines to the Covax program again, the health minister said Monday.

In April, the country halted shipments as it faced a brutal wave of the pandemic, which saw daily case numbers skyrocket from around 25,000 in mid-March to more than 400,000 a day by May. Since then, however, the vaccine output has more than doubled, and minister Mansukh Mandaviya said that it is set to quadruple to more than 300 million doses in October. Only the excess supply will be sent to Covax, he said.