On May 7, 2025 Schrödinger, Inc. (Nasdaq: SDGR) reported financial results for the quarter ended March 31, 2025 (Press release, Schrodinger, MAY 7, 2025, View Source [SID1234652649]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are very pleased with Schrödinger’s performance in the first quarter of 2025, with strong software and drug discovery revenue growth. Our proprietary pipeline is progressing, and we are looking forward to reporting initial data from the Phase 1 clinical study of SGR-1505 next month," said Ramy Farid, Ph.D., chief executive officer of Schrödinger. "More broadly, the pharmaceutical industry and even regulatory agencies are seeking to increase usage of computational solutions in R&D, and we continue to fortify our position as a scientific powerhouse in this field. With our growing software business and advancing pipeline of collaborative and proprietary programs, we believe we have a solid foundation that positions us for long-term growth."

First Quarter 2025 Financial Results
•Total revenue for the first quarter increased 63% to $59.6 million, compared to $36.6 million in the first quarter of 2024.
•Software revenue for the first quarter increased 46% to $48.8 million, compared to $33.4 million in the first quarter of 2024. The increase was primarily due to early renewals by large customers as well as increases in hosted contracts and contribution revenue.
•Drug discovery revenue was $10.7 million for the first quarter, compared to $3.2 million in the first quarter of 2024. First quarter 2025 drug discovery revenue included the recognition of $5.7 million from the company’s collaboration with Novartis.
•Software gross margin was 72% for the first quarter, compared to 76% in the first quarter of 2024, primarily reflecting the costs associated with the company’s predictive toxicology initiative.
•Operating expenses were $82.0 million for the first quarter, compared to $86.3 million for the first quarter of 2024. The decrease was primarily due to lower R&D expenses.
•Other expense was $8.9 million for the first quarter, which included changes in fair value of equity investments and interest income/expense, compared to other income of $13.2 million for the first quarter of 2024.
•Net loss for the first quarter was $59.8 million, compared to $54.7 million in the first quarter of 2024.

Three Months Ended
March 31,
2025 2024 % Change
(in millions)
Total revenue $ 59.6 $ 36.6 63%
Software revenue 48.8 33.4 46%
Drug discovery revenue 10.7 3.2 237%
Software gross margin 72 % 76 %
Operating expenses $ 82.0 $ 86.3 (5.0)%
Other (expense) income $ (8.9) $ 13.2 —
Net loss $ (59.8) $ (54.7) —

For the three months ended March 31, 2025, Schrödinger reported a net loss of $59.8 million, compared to a net loss of $54.7 million for the three months ended March 31, 2024.
For the three months ended March 31, 2025, Schrödinger reported a non-GAAP net loss of $46.7 million, compared to a non-GAAP net loss of $62.4 million for the three months ended March 31, 2024. See "Non-GAAP Information" below and the table at the end of this press release for a reconciliation of non-GAAP net loss to GAAP net loss.

2025 Financial Outlook
As of May 7, 2025, Schrödinger maintained its previously issued financial guidance for the fiscal year ending December 31, 2025:
•Software revenue growth is expected to range from 10% to 15%.
•Drug discovery revenue is expected to range from $45 million to $50 million.
•Software gross margin is expected to range from 74% to 75%.
•Operating expense growth in 2025 is expected to be less than 5%.
•Cash used for operating activities in 2025 is expected to be significantly lower than cash used for operating activities in 2024.

For the second quarter of 2025, software revenue is expected to range from $38 million to $42 million.
Key Highlights
Proprietary and Collaborative Pipeline
•Schrödinger continues to progress the Phase 1 clinical study of SGR-1505, the company’s MALT1 inhibitor, in patients with relapsed/refractory B-cell malignancies and expects to report initial clinical data from the trial in June.

•Schrödinger is continuing to progress the Phase 1 clinical study of SGR-2921, its CDC7 inhibitor, in patients with AML and myelodysplastic syndrome (MDS). The company expects to report initial data from this trial at a medical meeting in the second half of 2025.

•Schrödinger is continuing to progress the Phase 1 clinical study of SGR-3515, the company’s Wee1/Myt1 co-inhibitor, in patients with advanced solid tumors. Initial clinical data from this study are expected in the second half of 2025. In April, Schrödinger presented preclinical data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting demonstrating that SGR-3515 has improved anti-tumor activity in preclinical models compared to known Wee1 and Myt1 monotherapy inhibitors.

•Also at the AACR (Free AACR Whitepaper) Annual Meeting, Schrödinger presented initial preclinical data for SGR-4174, the company’s investigational SOS1 inhibitor. The preclinical data demonstrated that SGR-4174 exhibited potent and selective SOS1 inhibition and has strong tumor growth inhibition as a monotherapy and in combination with MEK or KRAS inhibitors.

Platform
•In April, the company issued a statement following the FDA’s announcement outlining plans to reduce existing animal testing requirements for monoclonal antibodies and other drugs with new approaches, including computation. Schrödinger is advancing its predictive toxicology initiative focused on small molecules, which is expected to be available to customers in the second half of 2025. The company currently offers computational solutions for small molecules and biologics that can be used to evaluate selectivity and the potential for off-target interactions.

•In March, Schrödinger scientists published research in Nature Communications describing a novel, computational crystal structure prediction (CSP) method that predicts crystal polymorphs with a high degree of accuracy and reliability. The ability to quickly and accurately predict crystal polymorphs has important applications for drug formulation.

Corporate
•In March, Schrödinger appointed Bridget van Kralingen to its Board of Directors. Ms. van Kralingen brings more than 35 years of experience leading and growing global technology solution and software businesses and is currently a senior partner at Motive Partners, an investment firm focused on technology-enabled companies. Prior to Motive Partners, Ms. van Kralingen spent nearly 18 years in a variety of executive roles at IBM, including as chief executive/senior vice president of IBM Global Markets and as chief executive of IBM’s Industry Platforms software division.

Webcast and Conference Call Information
Schrödinger will host a conference call to discuss its first quarter 2025 financial results on Wednesday, May 7, 2025, at 4:30 p.m. ET. The live webcast can be accessed under "News & Events" in the investors section of Schrödinger’s website, View Source To participate in the live call, please register for the call here. It is recommended that participants register at least 15 minutes in advance of the call. Once registered, participants will receive the dial-in information. The archived webcast will be available on Schrödinger’s website for approximately 90 days following the event.

On May 7, 2025 Revolution Medicines, Inc. (Nasdaq: RVMD), a late-stage clinical oncology company developing targeted therapies for patients with RAS-addicted cancers, reported its financial results for the quarter ended March 31, 2025, and provided an update on corporate progress (Press release, Revolution Medicines, MAY 7, 2025, View Source [SID1234652648]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The company continues to make meaningful progress on its 2025 strategic priorities:

Execute pivotal trials with daraxonrasib monotherapy in patients with previously treated metastatic pancreatic ductal adenocarcinoma (PDAC) and non-small cell lung cancer (NSCLC)

In RASolute 302, a global Phase 3 trial in patients with previously treated PDAC, the company continues its strong pace of enrollment in the U.S. and has begun enrolling patients in the EU and Japan. The company expects to substantially complete enrollment this year to enable an expected data readout in 2026.

For RASolve 301, a global Phase 3 trial in patients with previously treated NSCLC, the company is currently activating study sites.

Advance daraxonrasib into first-line metastatic and earlier-line randomized pivotal trials in patients with PDAC

Planning continues for registrational trials for daraxonrasib as first-line (1L) treatment for patients with metastatic PDAC and as adjuvant treatment for patients with resectable disease, and the company expects to initiate both studies in the second half of this year.

Generate sufficient data to inform development priorities for the mutant-selective inhibitors elironrasib and zoldonrasib and prepare to initiate one or more pivotal trials either as monotherapy or in a drug combination

The company expects to initiate one or more pivotal combination trials in 2026 that incorporate either zoldonrasib or elironrasib. The company believes the updated data shared today support this approach.

Last month, zoldonrasib monotherapy data in patients with previously treated KRAS G12D mutant NSCLC were featured in a late-breaking oral presentation and included in the press program at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.

The company reported updated elironrasib monotherapy data in patients with previously treated KRAS G12D mutant NSCLC.

The company today also announced several clinical combination updates in NSCLC, including daraxonrasib with pembrolizumab, elironrasib with pembrolizumab, and first data for the RAS(ON) inhibitor doublet combination of daraxonrasib with elironrasib in NSCLC.

Progress earlier-stage pipeline, including advancing next-generation innovations from the company’s highly productive discovery organization

Last month at AACR (Free AACR Whitepaper), RMC-5127, a RAS(ON) G12V-selective inhibitor, was highlighted in a New Drugs on the Horizon presentation. This program is on track to reach a clinic-ready stage later this year to enable expected Phase 1 initiation in 2026.

Grow commercial and operational capabilities and increase pre-commercial activities in support of a potential launch

The company continues building its commercialization capabilities in the U.S. and welcomed Anthony Mancini as chief global commercialization officer and member of its senior management team. Mr. Mancini, who brings substantial experience from a decades-long career in the biotech and biopharmaceutical industry, will oversee commercialization strategy and operations and will contribute additional strength both to the company’s commercialization approach in the U.S. and its evaluation of options for reaching patients outside of the U.S.

"We are executing well in our ongoing registrational studies of daraxonrasib in patients with previously treated PDAC and NSCLC, and continuing preparations to start the earlier-line PDAC trials this year. Today we’ve shared important data from all three of our clinical-stage investigational RAS(ON) inhibitors that reinforce exciting opportunities in NSCLC," said Mark A. Goldsmith, M.D., Ph.D., chief executive officer and chairman of Revolution Medicines. "As we continue growing our commercial and operational capabilities and advancing launch readiness activities, I’m pleased to welcome Anthony Mancini to our executive team."

Clinical Data Highlights

Data presented today, including items first shared at last month’s AACR (Free AACR Whitepaper) meeting, create exciting opportunities for the company in both previously treated and 1L metastatic NSCLC, including development approaches with mutant-selective inhibitors and drug combinations.

Zoldonrasib monotherapy in previously treated G12D NSCLC patients

Zoldonrasib, the company’s RAS(ON) G12D-selective inhibitor, demonstrated acceptable tolerability and encouraging initial antitumor activity in patients with previously treated KRAS G12D mutant NSCLC. The company believes these results support further evaluation of zoldonrasib as monotherapy and in combinations.

On April 27, 2025, the company presented initial clinical results from the zoldonrasib monotherapy trial. In 90 solid tumor patients treated at the candidate recommended Phase 2 dose of 1200 mg once daily (QD), as of a December 2, 2024 data cutoff, zoldonrasib was well tolerated with predominantly low-grade treatment-related adverse events (TRAEs). Grade 3 TRAEs were reported in 2% of patients and there were no Grade 4 or 5 TRAEs. The mean dose intensity (MDI) was favorable at 98%.

Preliminary antitumor activity was assessed in 18 efficacy-evaluable patients with previously treated NSCLC. The objective response rate (ORR) (confirmed or pending confirmation) was 61% and the disease control rate (DCR) was 89%.

Elironrasib monotherapy in previously treated G12C NSCLC patients

Elironrasib, the company’s RAS(ON) G12C-selective inhibitor, demonstrated acceptable tolerability and encouraging initial antitumor activity in patients with KRAS G12C NCSLC who were previously treated with immunotherapy and chemotherapy.

Today, the company shared data for 36 patients who were treated with elironrasib monotherapy at the candidate monotherapy recommended dose of 200 mg twice daily (BID) as of an April 7, 2025 data cutoff. Elironrasib was generally well tolerated with predominantly low-grade TRAEs. Grade 3 TRAEs were reported in 19% of patients and there were no Grade 4 or 5 TRAEs. The MDI was favorable at 94%.

At the 200 mg BID dose, the ORR in NSCLC patients was 56%, and the DCR was 94%. The estimated median progression-free survival (PFS) in these patients was 9.9 months.

Combinations in NSCLC

The company presented several datasets that support combination strategies to enable its goal of improving treatment outcomes for patients with RAS mutant NSCLC in the 1L metastatic setting.

Daraxonrasib and pembrolizumab with or without chemotherapy

Daraxonrasib, a RAS(ON) multi-selective inhibitor, demonstrated acceptable tolerability and encouraging preliminary antitumor activity in combination with pembrolizumab with or without chemotherapy.

Today, the company shared data for ten patients in the 1L NSCLC setting treated with daraxonrasib and pembrolizumab, as of a February 10, 2025 data cutoff. No new safety signals were seen, and the overall safety profile was consistent with those of daraxonrasib alone and the standard of care agents.

Hepatotoxicity did not appear as a safety signal (no Grade 3 or higher events) in the combination of daraxonrasib and pembrolizumab with or without chemotherapy. No Grade 3 or higher AST or ALT elevations were reported and there was also no evidence of increased immune-related adverse events, such as colitis or pneumonitis, with the addition of daraxonrasib to pembrolizumab.

Daraxonrasib achieved a favorable MDI of 93% in combination with pembrolizumab and 90% with the addition of chemotherapy.

Of the seven 1L patients who had sufficient follow up to have had a tumor assessment, preliminary antitumor activity was encouraging.

Patients selected for treatment with daraxonrasib and pembrolizumab had tumors with a tumor proportion score (TPS) greater than or equal to 50%, consistent with a population where pembrolizumab is the preferred standard of care. Among seven efficacy-evaluable patients treated with the combination, the ORR was 86% and the DCR was 100%.

Patients selected for treatment with daraxonrasib with pembrolizumab and chemotherapy had tumors with TPS scores of less than 50%, where pembrolizumab and chemotherapy is the preferred standard of care. Among ten efficacy-evaluable patients treated with the combination, the ORR was 60% and the DCR was 90%. The company believes these data collectively support continued development of daraxonrasib with standard of care in RAS mutant NSCLC in the 1L metastatic setting.

Elironrasib with pembrolizumab in 1L RAS G12C mutant NSCLC

Today, the company shared data for eight patients with 1L KRAS G12C NSCLC from the combination of elironrasib with pembrolizumab, which showed acceptable tolerability and encouraging preliminary antitumor activity. As of a February 10, 2025 data cutoff, Grade 3 or higher TRAEs were reported in 25% of patients, and no new safety signals were observed. The MDI was favorable at 85%.

Among five efficacy-evaluable patients with 1L NSCLC with a TPS score of greater than or equal to 50% (where pembrolizumab monotherapy is the preferred standard of care), the ORR and DCR were both 100%.

RAS(ON) inhibitor doublet of elironrasib with daraxonrasib in NSCLC

Today, the company presented data that demonstrate acceptable tolerability and encouraging antitumor activity of the RAS(ON) inhibitor doublet of elironrasib with daraxonrasib in NSCLC patients previously treated with a KRAS G12C(OFF) inhibitor.

As of a February 10, 2025 data cutoff, in 33 patients treated with elironrasib at 200 mg BID and daraxonrasib at doses ranging from 100 mg to 200 mg daily, Grade 3 TRAEs were reported in 46% of patients. There were no Grade 4 or 5 TRAEs. Hepatotoxicity was not observed as a safety signal (no Grade 3 or higher events) and QT prolongation was limited with one asymptomatic Grade 3 event (3%).

The MDIs were favorable at 95% for elironrasib and 85% for daraxonrasib.

The combination of elironrasib with daraxonrasib showed encouraging preliminary antitumor activity in patients with NSCLC who have been previously treated with a KRAS G12C(OFF) inhibitor. The ORR was 62% and the DCR was 92%.

The company believes the preliminary results described for the above pairwise combinations support its goal of developing a chemotherapy-sparing triplet combination including elironrasib, daraxonrasib and pembrolizumab in patients with first-line metastatic RAS G12C NSCLC.

Financial Highlights

First Quarter Results

Cash Position: Cash, cash equivalents and marketable securities were $2.1 billion as of March 31, 2025.

R&D Expenses: Research and development expenses were $205.7 million for the quarter ended March 31, 2025, compared to $118.0 million for the quarter ended March 31, 2024. The increase in expenses was primarily due to increases in clinical trial expenses and manufacturing expenses for daraxonrasib, zoldonrasib and elironrasib, and personnel-related expenses and stock-based compensation expense related to additional headcount.

G&A Expenses: General and administrative expenses were $35.0 million for the quarter ended March 31, 2025, compared to $22.8 million for the quarter ended March 31, 2024. The increase was primarily due to increases in personnel-related expenses and stock-based compensation expense associated with additional headcount, and an increase in commercial preparation activities.

Net Loss: Net loss was $213.4 million for the quarter ended March 31, 2025, compared to net loss of $116.0 million for the quarter ended March 31, 2024.

Financial Guidance

Revolution Medicines is reiterating projected full year 2025 GAAP net loss guidance of between $840 million and $900 million, which includes estimated non-cash stock-based compensation expense of between $115 million and $130 million. Based on the company’s current operating plan, the company projects current cash, cash equivalents and marketable securities can fund planned operations into the second half of 2027.

Webcast

Revolution Medicines will host a webcast this afternoon, May 7, 2025, at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time). To listen to the live webcast, or access the archived webcast, please visit: View Source Following the live webcast, a replay will be available on the company’s website for at least 14 days.

On May 7, 2025 QIAGEN N.V. (NYSE: QGEN; Frankfurt Prime Standard: QIA) reported strong Q1 2025 results in line with the preliminary announcement and reaffirmed its FY 2025 outlook based on the solid start to the year and evolving macroeconomic trends (Press release, Qiagen, MAY 7, 2025, View Source [SID1234652647]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

As announced on April 6, net sales in Q1 2025 rose 5% to $483 million from Q1 2024, with 7% growth at constant exchange rates (CER) well above the outlook. Core sales (excludes discontinued products such as NeuMoDx and Dialunox) also rose 7% CER. The adjusted operating income margin improved by 4.1 percentage points to 29.8%, supported by broad efficiency gains and the 2024 decision to discontinue the NeuMoDx clinical PCR testing system. Adjusted diluted earnings per share (EPS) were $0.55, and results of $0.56 CER exceeded the outlook for at least $0.50 CER.

In view of the Q1 2025 results, as well as ongoing macro trends (including U.S. and China import tariffs), QIAGEN reaffirms its FY 2025 net sales outlook for about 4% CER (core sales growth of about 5% CER). The outlook for adjusted EPS was upgraded in April to about $2.35 CER. QIAGEN also reaffirmed its 2025 adjusted operating income margin target for above 30%, and expects to reach its mid-term margin goal of at least 31% well ahead of the original 2028 target.

"QIAGEN delivered a solid start to 2025, exceeding our outlook on both sales and adjusted earnings. Our growth pillars QIAstat-Dx and QuantiFERON both posted double-digit sales gains, while QIAcuity and QIAGEN Digital Insights both continued to build momentum and in Sample technologies we are focusing on automation and preparing for new instrument launches. These advancements highlight the execution of our focused strategy and our ability to perform amid global uncertainty. We have reaffirmed our full-year 2025 targets and remain committed to delivering solid, profitable growth as a foundation for achieving our 2028 ambitions," said Thierry Bernard, CEO of QIAGEN.

"We maintained strong financial discipline in the first quarter, generating solid cash flow levels while funding key investments and advancing efficiency initiatives. This strength has enabled QIAGEN to expand our capital allocation strategy with the proposal to initiate an annual dividend and also to seek shareholder approval for a new synthetic share repurchase authorization. These actions reflect our commitment to creating greater value by generating operational leverage and strong cash flows while increasing returns," said Roland Sackers, CFO of QIAGEN.

Please find the full press release incl. tables as a PDF for download at the top of this page.

Investor presentation and conference call

A conference call is scheduled for Thursday, May 8, 2025, at 15:00 Frankfurt Time / 14:00 London Time / 09:00 New York Time. A live audio webcast will be accessible in the investor relations section of the QIAGEN website (www.qiagen.com), with a recording available after the event. The presentation will be published ahead of the call in this section: QIAGEN Investor Relations – Events and Presentations.

Use of adjusted results

QIAGEN reports adjusted results, as well as results on a constant exchange rate (CER) basis, along with other non-U.S. GAAP (generally accepted accounting principles) measures, to provide deeper insights into its performance. These include metrics such as core sales (excluding discontinued products), adjusted gross margin, adjusted gross profit, adjusted operating income, adjusted operating expenses, adjusted operating income margin, adjusted net income, adjusted net income before taxes, adjusted diluted EPS, adjusted EBITDA, adjusted EPS, adjusted income taxes, adjusted tax rate, and free cash flow. Free cash flow is calculated by subtracting capital expenditures for property, plant and equipment from cash flow from operating activities. Adjusted results are non-GAAP financial measures that QIAGEN considers complementary to GAAP-reported results but not as substitutes. These measures exclude items that QIAGEN believes are outside of ongoing core operations, fluctuate significantly between periods, or hinder the comparability of results with competitors and prior periods. QIAGEN also uses non-GAAP and constant currency financial measures internally in planning, forecasting and reporting, and also for employee compensation. Additionally, adjusted results are used to compare current performance with historical results, which have consistently been presented on an adjusted basis.

On May 7, 2025 Priothera Ltd., a late-stage biopharma company pioneering the development of mocravimod, a novel oral sphingosine 1 phosphate (S1P) receptor modulator, to treat hematologic malignancies, reported the appointment of Jens Hasskarl, MD, PhD, as Chief Medical Officer (CMO) (Press release, Priothera, MAY 7, 2025, View Source [SID1234652646]). Dr. Hasskarl will oversee the global Phase 3 clinical study MO-TRANS of mocravimod, which is being developed as an adjunctive treatment in acute myeloid leukemia (AML) to enhance the curative potential of allogeneic hematopoietic cell transplantation (allo-HCT).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Dr. Hasskarl brings over two decades of international leadership experience in clinical development, translational science and medical affairs across top-tier pharma, biotech and academic institutions. Most recently, he served as CMO at Advesya AG, where he led the strategic development of novel immunotherapies in haemato-oncology and autoimmunity. He previously held senior executive roles at Tigen Pharma, Celgene and Novartis, where he was instrumental in the development and global approval of multiple cellular therapies including Breyanzi, Abecma and Kymriah.

"Jens’ deep expertise in hematology, cellular therapy and translational drug development, combined with his entrepreneurial mindset and proven track record in leading successful global clinical programs, make him the ideal fit," said Florent Gros, Co-Founder and CEO of Priothera. "His insight and leadership will be critical as we prepare for the final phase of clinical execution and regulatory engagement for mocravimod. We would like to thank Dr. Elisabeth Kueenburg, Priothera’s former CMO, for her contributions and wish her every success in her future endeavors."

"I am thrilled to join Priothera during such an exciting phase," said Dr. Hasskarl, CMO of Priothera. "The company’s science-driven approach and commitment to improving outcomes for patients with AML aligns perfectly with my focus on advancing innovation in hematology. I look forward to working closely with the team to bring this promising therapy to patients worldwide."

Dr. Hasskarl holds an MD and PhD from Heidelberg University and the German Cancer Research Center. He completed a postdoctoral fellowship at Harvard Medical School and holds a diploma in Health Economics. He is a board-certified Internist with a specialty in hematology and oncology and continues to lecture at Freiburg Medical School in Germany.

On May 7, 2025 Phio Pharmaceuticals Corp. (Nasdaq: PHIO) reported it is a clinical-stage siRNA biopharmaceutical company developing therapeutics using its proprietary INTASYL gene silencing technology to eliminate cancer (Press release, Phio Pharmaceuticals, MAY 7, 2025, View Source [SID1234652645]). Phio reported that a complete pathologic response (100% tumor clearance) has been reported for 2 of 3 patients with cutaneous squamous cell carcinoma (cSCC) treated in the third dose cohort. The third patient was reported as having a pathologic non-response (<50% tumor clearance).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"These positive outcomes continue to indicate that PH-762 may present a viable non-surgical alternative in this large and continually expanding skin cancer market," said Robert Bitterman, President and CEO of Phio Pharmaceuticals.

Phio’s ongoing Phase 1b dose escalation clinical trial (NCT 06014086) is designed to evaluate the safety and tolerability of neoadjuvant use of intratumoral PH-762 in Stages 1, 2 and 4 cSCC, Stage 4 melanoma, and Stage 4 Merkel cell carcinoma.

To date, a total of 10 patients with cutaneous carcinomas have been treated in Cohorts 1, 2 and 3. These cohorts included 9 patients with cSCC and 1 patient with metastatic melanoma. At day 36 (planned tumor excision), of the 9 patients with cSCC, 4 patients had a pathologic complete response (100% tumor clearance). One patient had a near complete response (>90% clearance) and 1 patient had a partial response (>50% clearance). The other 3 cSCC and one metastatic melanoma patient had a pathologic non-response (< 50% clearance). Patients with pathologic complete response (100% tumor clearance) may have visual signs of residual scar or subdermal inflammation prior to resection. No patients, however, exhibited clinical progression of disease.

To date, there were no dose-limiting toxicities or clinically relevant treatment-emergent adverse effects in the patients receiving intratumoral PH-762 in this trial. Moreover, PH-762 has been well tolerated in all enrolled patients in each escalating dose cohort. The fourth cohort is currently enrolling patients; Phio expects to complete enrollment in the trial in the third quarter of 2025.

"The continued positive pathologic responses in this clinical trial of intratumoral PH-762 for cutaneous carcinoma increases our understanding of the potential therapeutic benefit for this immunotherapy. The safety profile supports ongoing dose escalation," said Mary Spellman, MD, Phio’s acting Chief Medical Officer.