Asco 2023 preview – a microcap winner

One of the most momentous Asco abstracts drops of recent years sends Verastem up 126%.

It is often said that Asco is a big pharma conference, and the 2023 edition looks to be no exception. That said, there are always plenty of gainers among smaller biotechs, and Thursday night’s abstract unveiling put the markets into overdrive. The charge was led by the microcap player Verastem, which shot up an incredible 126% the next day.

Other notable gainers included Elevation Oncology, PDS Biotechnology and Effector Therapeutics, while Theseus and Leap Therapeutics sold off, and the Tigit space enjoyed its own resurgence. This market reaction is extraordinary, especially given that much of what has been revealed is merely abstract texts, some with rather old data cutoffs.

The most up-to-date results will only be revealed once Asco gets under way this weekend. However, this is not quite the case for Verastem’s dataset, covering the Raf/Mek inhibitor avutometinib, which the company itself updated in a press release accompanying the less mature abstract.

The data are important because they come from part A of the registrational Ramp-201 trial in low-grade serous ovarian cancer, for which there are currently no FDA-cleared drugs. The release cited a 45% ORR (13/29) with a combination of avutometinib and defactinib, a focal adhesion kinase inhibitor – better than the 28% in the Asco abstract.

Monotherapy with avutometinib, which Verastem acquired after it was cast off by Roche’s Chugai division, did not impress.


After Astellas’s anti-Claudin18.2 MAb zolbetuximab scored a double phase 3 success this novel antigen continues to generate interest, notwithstanding the field becoming highly competitive.

Elevation Oncology surged 67% on Friday after an Asco abstract (November 2022 cutoff) on its anti-Claudin18.2 ADC SYSA1801 showed a 38% ORR in Claudin18.2-positive solid tumours, including gastric cancer. The number is well below that in Astellas’s Spotlight study, however.

Investors reckon PDS Biotechnology might have found a way to improve on PD-(L)1 inhibitors in head and neck cancer with PDS0101, an HPV-targeted immunotherapy. Results to be presented at Asco come from the checkpoint inhibitor-naive arm of the Versatile-002 study in 34 HPV16-positive patients, testing PDS0101 plus Keytruda.

This yielded median PFS of 10.4 months, comparing well on a cross-trial basis versus Keytruda monotherapy, which showed median PFS of three months in Keynote-048. A press release cites ORR of 41%, which adds five unconfirmed to the nine confirmed remissions cited in the Asco abstract, and is in line with previous results.

However, a major caveat is that Versatile-002 comprised only HPV16-positive patients, a fact that might flatter the result. Next up for PDS is the pivotal Versatile-003 study, slated to start this year.

Effector was up 25% on hopes that it could have a breast cancer contender in zotatifin, an eIF4A inhibitor, with a press release updating its Asco abstract and showing a 26% ORR using a zotatifin/Verzenio/fulvestrant triplet. All five responders had failed CDK4/6 blockade and fulvestrant, and Stifel estimated that zotatifin could be doubling the efficacy of fulvestrant plus Verzenio alone.

Notable share price movers* after disclosure of Asco 2023 abstracts
Company Share price chg** Abstract Project Mechanism Study Setting
Verastem (ex Roche) +126% 5515 Avutometinib (VS-6766)  Raf/Mek inhibitor Ramp-201 Low-grade serous ovarian cancer
Elevation Oncology +67% 3016 SYSA1801  Anti-Claudin18.2 ADC NCT05009966 Solid tumours
PDS Biotechnology +27% 6012 PDS0101 + Keytruda HPV16-directed immunotherapy Versatile-002 HPV16+ head & neck cancer
Effector +25% 1080 Zotatifin  eIF4A inhibitor NCT04092673 ER+/Her2- breast cancer
Zentalis +11% 5513 Azenosertib (ZN-c3) Wee1 inhibitor NCT04516447 Ovarian and others
Gracell +6% 8005 GC012F  Anti-BCMA/CD19 Car-T NCT04236011 & NCT04182581 r/r multiple myeloma
Blueprint +6% 9011 BLU-945 EFGR inhibitor Symphony (Tagrisso combo) Post-Tagrisso NSCLC
9064 BLU-451 (ex Lengo) EGFR exon 20 inhibitor Concerto EGFR exon20ins tumours
3095 BLU-222 CDK2 inhibitor Vela Solid tumours
Cullinan +5% 2532 CLN-619 Anti-MicA/B MAb NCT05117476 Solid tumours
Prelude +5% 3008 PRT811  PRMT5 inhibitor NCT04089449 CNS lymphomas, gliomas & solid tumours
Geron +4% 7004 Imetelstat Telomerase inhibitor Imerge Low or intermediate-1 MDS
Affimed -5% 2533 AFM24 Anti-wtEGFR NK cell engager NCT04259450 Solid tumours
Erasca -5% 3558 ERAS-007 + Ibrance Erk inhibitor Herkules-3 Kras/Nras+ colorectal & others
Autolus -7% 7000 Obe-cel Anti-CD19 Car-T Felix (pivotal) r/r ALL
Springworks -8% 11564 Nirogacestat Gamma secretase inhibitor DeFi Desmoid tumours
Leap -10% 4027 DKN-01 + tislelizumab +/- chemo Anti-DKK1 MAb Distinguish 1L gastric/GEJ adenocarcinoma
Theseus -11% e23508 THE-630 Pan-variant Kit inhibitor NCT05160168 GIST
Note: *for Tigit-relevant stocks see separate story; **at close on Friday 26 May. 

Though Blueprint closed up only 6% it is notable for having three Asco abstracts that should be of interest to its investors, including the first data cut from a study of the EGFR exon 20 insertion inhibitor BLU-451, acquired through the $250m takeout of Lengo; the abstract only contains safety results.

Novel mechanisms include Blueprint’s CDK2 inhibitor BLU-222 – just one response among 27 patients, according to the abstract – and Cullinan’s anti-MicA/MicB MAb CLN-619. The latter too has yet to convince, with one response in the abstract being updated to three among 22 evaluable subjects in a press release, but nevertheless Cullinan closed up 5%.


Among fallers, updated results from the Distinguish trial of Leap Therapeutics’ DKN-01 in first-line gastric cancers were described by the sellside as “encouraging” and “intriguing”, but the group closed down 10%. Leap’s stock has been sliding since Esmo, despite promising response rates from the same trial, amid concerns about financing and dilution.

Distinguish tests the anti-DKK1 MAb on top of Beigene’s anti-PD-1 MAb tislelizumab and chemo. The Asco abstract details median PFS and OS of 11.3 and 19.5 months respectively; on a cross-trial basis this looks better than tislelizumab and chemo in Rationale-305, but the uncontrolled part A of Distinguish involved just 25 patients; data from the randomised, controlled part C could come by year end.

Autolus slid 7%, but a more relevant measure of the company might be the 55% climb its stock has enjoyed since disclosure on 12 May of an abstract at the EHA conference covering obe-cel’s pivotal Felix study. The Asco abstract is identical to the EHA’s.

As such, perhaps the biggest Asco disappointment so far has been the young biotech Theseus Pharmaceuticals, hit by disclosure of a patient death in a phase 1/2 trial of its pan-Kit inhibitor THE-630 in gastrointestinal stromal tumours. Theseus said the patient had received eight prior therapies and had comorbidities, but efficacy has hardly been stellar, with stable disease being the best response so far.

While that overshadowed a highly positive abstract drop, seasoned Asco watchers will note that much of biopharma sells off on the Monday of the conference. How Friday’s gainers perform once the coming weekend’s euphoria dies down will provide a better test of the health of the markets.

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