Sy­ros push­es for a turn­around with new PhII AML re­sults, but in­vestors are unim­pressed

CEO Nan­cy Si­mon­ian

Two years af­ter weak Phase II re­sults cut their stock price in half, Sy­ros Phar­ma­ceu­ti­cals (SYRS) is back with re­sults from an on­go­ing Phase II tri­al try­ing their SY-1425 “su­per-en­hancer” ther­a­py in com­bi­na­tion with chemother­a­py on acute myeloid leukemia (AML) pa­tients. 

It’s ear­ly-stage and the num­bers are small, but of the 13 evalu­able pa­tients who test­ed pos­i­tive for the RARA path­way bio­mark­er Sy­ros’ ther­a­py tar­gets, re­searchers say that 8 had a com­plete re­sponse. That com­pared with 8 com­plete re­spons­es out of 22 pa­tients who didn’t have the gene. The longest CR was 344 days, com­pared with 168 for RARA neg­a­tive pa­tients, al­though Sy­ros not­ed the study is still on­go­ing.

“We show how when we se­lect the pa­tients who have po­ten­tial for a re­sponse, we have a high re­sponse rate,” Sy­ros CMO David Roth told End­points News. “And we show how these re­spons­es oc­cur rel­a­tive­ly quick­ly.”

Daniel J. DeAn­ge­lo, an on­col­o­gy ex­pert at the Dana-Far­ber In­sti­tute who is not af­fil­i­at­ed with Sy­ros, said the pa­tient pop­u­la­tion was small and the da­ta pre­lim­i­nary but that the re­sults were nev­er­the­less promis­ing for a sub­group of pa­tients. He not­ed the da­ta showed the drug on­ly worked on one of two bio­mark­ers Sy­ros had screened for, but that in pa­tients with RARA the re­sponse rate was around 70%.

“That’s about twice the re­sponse you would nor­mal­ly ex­pect” from chemother­a­py alone, DeAn­ge­lo told End­points.  “We don’t have any pro­gres­sion-free, sur­vival da­ta, and it’s a small num­ber [of pa­tients] – we’d want to con­firm it and all that – but looks re­al­ly in­ter­est­ing for that group of pa­tients.”

In­vestors, though, don’t ap­pear en­thused. Sy­ros’ stock dropped 4% to­day, con­tin­u­ing a fall of near­ly $5 per share since Oc­to­ber 17, when the biotech an­nounced it was shelv­ing a lead CDK7 in­hibitor in fa­vor of an oral ver­sion.

The 2017 tri­al test­ed SY-1425 as a sin­gle agent and found one com­plete or par­tial re­sponse out of 48 evalu­able pa­tients. In­vestors re­spond­ed with a sell­off that cut Sy­ros’s stock by over 50%. The com­pa­ny said at the time and has main­tained since that the “clin­i­cal ac­tiv­i­ty” in pa­tients val­i­dat­ed their ap­proach and point­ed to­ward com­bi­na­tion ther­a­pies as a log­i­cal next step.

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.

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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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.

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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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.