On February 20, 2025 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS’’ or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported data from Phase 2a trial of SLS009 (tambiciclib), a highly selective CDK9 inhibitor, in relapsed/refractory Diffuse Large B-Cell Lymphoma (r/r DLBCL) (Press release, Sellas Life Sciences, FEB 20, 2025, View Source [SID1234650419]).

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The trial, conducted and funded by GenFleet Therapeutics (Shanghai), Inc. ("Genfleet"), was an open-label single-arm multicenter Phase 2a study in China evaluating SLS009 in combination with BTK inhibitor, Brukinsa (zanubrutinib) in r/r DLBCL. The results showed an overall response rate of 67%, more than double the expected overall response rate (ORR) of zanubrutinib alone. Among responders, one achieved complete response (CR), while three had partial response (PR) with target lesion shrinkages of 89%, 78%, and 56%, respectively. As of the last follow-up, after the median of 4.6 (range: 1.4 – 7.4) months follow-up, median overall survival (OS) was not reached, and six out of 9 patients were alive.

"These results represent a promising step forward in improving outcomes for DLBCL patients and underscores the potential of SLS009 in combination with zanubrutinib to deliver meaningful clinical benefits," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "Achieving an ORR that significantly exceeds expectations, along with a complete response and multiple partial responses is a testament to the power of collaboration and innovation in tackling this challenging disease. We believe that the combination of SLS009 and zanubrutinib demonstrates a synergy that could pave the way for more effective treatment options. Moving forward, GenFleet will determine the next steps regarding the trial’s continuation around lymphoma as SELLAS’ focus remains in AML and spliceosome – chromatin mutations, including ASXL1 mutations."

Summary of Phase 2a data of SLS009 in DLBCL

Patients Characteristics

9 r/r DLBCL patients were enrolled: 3 with germinal center B-cell like (GCB) and 6 with activated B-cell like (ABC) subtype of DLBCL
ABC DLBCL, also known as non-GCB DLBCL, carries a worse prognosis vs. GCB DLBCL
The median age was 55 years old and the median of previous lines of therapy was 2 (range 2-4)
Efficacy and Safety

Among 6 non-GCB DLBCL (ABC DLBCL) patients, 4 had an objective response and one patient achieved stable disease (SD) for the disease control rate (DCR) of 5/6 (83%)
Overall response rate (ORR) was 4/6 (67%), more than double the expected ORR with zanubrutinib alone
One patient achieved complete response (CR), and three patients had partial response (PR) with target lesion shrinkages of 89%, 78%, and 56%, respectively
As of the last follow-up, after the median of 4.6 (range: 1.4 – 7.4) months follow-up, median overall survival (OS) was not reached
Six patients were alive as of the last follow-up, including 5 non-GCB DLBCL and 1 GCB DLBCL. Adverse events (AEs) grade ≥ 3 AEs were reported in 55.6% of patients, comparable to safety outcomes expected with Zanubrutinib alone
Genetic data of 6 out of 9 enrolled patients showed that none of the patients carried MYD88 or CD79B mutations predictive of better response to BTK inhibitors. The patient who achieved complete response (CR) by CT had MYC amplification, which is expected, but interestingly also harbored TP53 mutations, indicating that CDK9 inhibition with SLS009 could circumvent TP53 mutated cancers drug resistance.
"These additional data from yet another indication help us further expand the scope of SLS009," said Dragan Cicic, MD, Chief Development Officer of Sellas. "In parallel with our very advanced clinical development in acute myeloid leukemia, we are continuously working on additional clinical and preclinical programs in other indications and uncovering genetic biomarkers that make all the difference in today’s drug development."

On February 20, 2025 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that its executives will participate in fireside chats at the following investor conferences (Press release, PTC Therapeutics, FEB 20, 2025, View Source [SID1234650418]):

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TD Cowen 45th Annual Health Care 2025 Conference
Tuesday, March 4 at 11:10 a.m. ET

Barclays 27th Annual Global Healthcare Conference
Tuesday, March 11 at 12:30 p.m. ET

The presentations will be webcast live on the Events and Presentations page under the Investor section of PTC Therapeutics’ website at View Source and will be archived for 30 days following the presentation. It is recommended that users connect to PTC’s website several minutes prior to the start of the webcast to ensure a timely connection.

On February 20, 2025 Oxford Vacmedix (OVM) reported the first patient being treated with new extended dosing of OVM-200, at the prestigious Sarah Cannon Research Institute in London (Press release, Oxford Vacmedix, FEB 20, 2025, View Source;utm_medium=rss&utm_campaign=first-patient-ovm-200-cancer-vaccine-extended-dosing [SID1234650417]). The extended dosing protocol was first suggested by the clinical investigators in the trial, following the excellent safety record seen in Phase 1a. The new regime will allow up to 11 vaccinations of OVM-200 over a six-month period and has been approved by the UK Medicines and Healthcare products Regulatory Agency (MHRA). OVM-200 is a new therapeutic cancer vaccine developed using OVM’s novel recombinant overlapping peptide (ROP) platform. It targets survivin, a protein overexpressed by cancer cells, which prevents them being attacked by the body’s immune system.

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The Phase I trial of OVM-200 is focused on safety and on establishing an immune response in patients with three tumour types – non small cell lung cancer (NSCLC), prostate cancer and ovarian cancer. It is being run at four sites in the UK including the Sarah Cannon Institute and University College Hospital (UCH) in London, the Churchill hospital of the Oxford University Hospitals Foundation Trust (OUHFT) and the Christie NHS Foundation Trust in Manchester. The first part of the trial, Phase 1a, has been completed and has shown both excellent safety and a strong immune response. The Chief Investigator for the trial is Professor Martin Forster, based at UCH. This trial is both the first time OVM-200 has been used in people and also the first time any ROP-based vaccine has been tested in the clinic.

William Finch, CEO of Oxford Vacmedix, said:

The ROP technology has been developed from an initial concept in the laboratory to now being tested as a treatment for critically ill patients. We see the potential benefits of a vaccination approach both in stimulating the body’s immune system to attack the cancer and also, in future trials, enhancing the efficacy of other immune oncology agents. This Phase I trial is a first step towards having effective cancer vaccines.

Dr Anja Williams Principal Investigator at the Sarah Cannon Research Institute UK, added:

It is a privilege to work with Oxford Vacmedix on this innovative vaccine programme for patients with lung, prostate, and ovarian cancer. We are very pleased with the results to date and strongly believe that vaccine treatments will play a major role in future cancer treatments. Extending the dosing will maximise the potential benefits of the vaccine.

On February 20, 2025 Oxcia reported that the FDA (the United States Food and Drug Administration ) has granted ODD (orphan drug designation) to OXC-101 as a potential treatment option for patients with AML (acute myeloid leukemia) (Press release, Oxcia, FEB 20, 2025, View Source;utm_medium=rss&utm_campaign=oxc-101-has-been-granted-odd-status-by-the-fda-in-aml [SID1234650416]).

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OXC-101 is Oxcia’s lead clinical candidate, a first-in class mitotic MTH1 inhibitor with a unique dual mechanism of action. In short, OXC-101, fights cancer by taking advantage of one of the Achilles heels of cancer cells – the high endogenous oxidative stress and DNA damage. In preclinical studies, OXC-101 significantly prolongs survival and reduces tumor growth in AML disease models.

Clinical Phase 1 data in advanced hematological cancer patients is reassuring. Oxcia is now performing an expansion trial, a phase 1/2 study in a subpopulation (relapsed/recurrent AML), in combination with one of the standard treatments, idarubicin. The aim is to confirm the preliminary efficacy previously shown in AML to support further clinical development in a pivotal phase 2 trial that can be the base for regulatory fast-track approval.

Oxcia AB was in the beginning of 2023 awarded a grant of 3 million SEK by Swelife and Medtech4Health for this study. To ensure the recruitment of patients with this rare AML disease in a cost- and time-efficient manner, we are now adding additional clinical sites. Rigshospitalet in Denmark will be enrolled shortly. In addition, Bulgaria and Serbia will be added during 2025.

"We are delighted with the FDA’s decision to grant orphan drug designation to OXC-101 for AML. This is a significant milestone and underscores the significant unmet need for novel medicines and the unique approach by OXC-101. We believe OXC-101 holds potential to greatly improve treatment for patients who suffer from AML", says Ulrika Warpman Berglund, Oxcia’s CEO. We are presently preparing an application for ODD also with EMA (European Medicines Agency). Early engagement with the FDA and EMA is pivotal.

In the study we are also integrating precision medicine screening to help identify responders/non-responders. Precision medicine screening is a novel way of personalizing treatment for patients and thus identifying the best treatment strategy for individuals reducing unsuccessful treatments with unnecessary suffering and lack of effect.

Briefly about AML

Acute myeloid leukemia (AML) is an aggressive blood cancer associated with infection, anemia and bleeding. It is the most common acute leukemia in adults, accounting for more than 80% of the cases. In 2021, there were 162,200 incident cases of AML worldwide, and the number is forecasted to increase to 169,000 incident cases by 2027 (Datamonitor). Even though the overall survival rate among AML patients has been increasing in the past decades, it remains poor with an overall 5-year survival rate of 20% in selected subpopulations.

AML is a very heterogenous disease, which explains why classical intensive chemotherapy, constituting of a cytarabine /anthracycline backbone, remains the standard of care (SoC). The medical need is high. Although we have seen recent approvals for biomarker led AML subsets, the majority of patients are still in need of well-tolerated and effective novel therapies.

Briefly about Orphan Drug Designation

The FDA and EMA grant orphan status to products intended to treat, diagnose, or prevent a life-threatening rare disease or condition that affects fewer than five in 10,000 people in Europe, or under 200,000 people in the US, and with either no currently approved method of diagnosis, prevention, or treatment, or with significant benefit to those affected by the disease. Orphan drug designation provides certain benefits, including the potential for extensive marketing exclusivity following regulatory approval, reduction in regulatory fees and, in the case of EU, a centralized approval process.

On February 20, 2025 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, reported financial results for the quarter and full year ended December 31, 2024 (Press release, Guardant Health, FEB 20, 2025, View Source [SID1234650415]).

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Fourth Quarter 2024 Financial Highlights

For the three-month period ended December 31, 2024, as compared to the same period of 2023:
•Total revenue of $201.8 million, an increase of 30%
•Reported approximately 57,300 oncology clinical tests (excluding Shield) and approximately 11,050 biopharma tests, an increase of 24% and 16%, respectively
•Reported approximately 6,400 Shield screening tests, with revenue of $4.1 million

Full Year 2024 Financial Highlights

For the twelve-month period ended December 31, 2024, as compared to the same period of 2023:
•Total revenue of $739.0 million, an increase of 31%
•Reported approximately 206,700 oncology clinical tests (excluding Shield) and approximately 40,500 biopharma tests, an increase of 20% and 35%, respectively
•Improved full year 2024 free cash flow burn to $275 million, compared to $345 million for the full year 2023

Recent Operating Highlights

•Received Medicare coverage for Guardant Reveal on Smart Liquid Biopsy platform for colorectal cancer surveillance
•Shield selected by NIH for inclusion in the Vanguard multi cancer detection study based on strong feasibility data in predicting presence of ten cancer types and cancer site of origin
•Shield selected for a government funded population scale screening program in Abu Dhabi and surrounding regions
•Entered into a collaboration with ConcertAI to create a differentiated data-as-a-service platform that integrates comprehensive EMR records with both genomic and epigenomic tumor profiling data to accelerate cancer therapy research and development
•Entered into a strategic collaboration with Boehringer Ingelheim to develop a companion diagnostic for zongertinib to detect specific mutations in non-small cell lung cancer
•Completed a convertible debt exchange to extend the maturity on $600 million of outstanding debt
"We fired on all cylinders throughout 2024, delivering remarkable revenue growth and upgrading our products to our smart liquid biopsy platform," said Helmy Eltoukhy, co-founder and co-CEO. "Last month, we were pleased to receive Medicare reimbursement for Guardant Reveal in the CRC surveillance setting, which was an important milestone for our MRD business. We are entering 2025 with a strong oncology product portfolio and anticipate volume acceleration across all our products this year."

"We made strong progress with Shield during Q4, our first full quarter since commercial launch," said AmirAli Talasaz, co-founder and co-CEO. "We screened thousands of patients with our simple blood test and are looking forward to scaling our impact quickly. Beyond CRC, the selection of Shield for the NIH Vanguard study underscores the strength of our platform and establishes our leadership in the field of multi cancer detection. With a robust pipeline of catalysts in front of us, we are excited to execute on the opportunities ahead in 2025."

Fourth Quarter 2024 Financial Results
Revenue was $201.8 million for the fourth quarter of 2024, a 30% increase from $155.1 million for the corresponding prior year period. Precision oncology revenue grew 30%, to $184.6 million for the fourth quarter of 2024, from $142.2 million for the corresponding prior year period, driven by an increase in the volume of clinical tests (i.e., Guardant360, TissueNext, Response, and Reveal tests) and biopharma tests, which grew 24% and 16%, respectively, over the prior year period. The increase in precision oncology revenue was also attributable to an increase in reimbursement for our tests, due to an increase in Medicare reimbursement for our Guardant360 LDT test to $5,000, effective January 1, 2024; and an increase in both Medicare Advantage and commercial payer reimbursement. Development services and other revenue was $17.2 million for the fourth quarter of 2024, compared to $12.9 million for the corresponding prior year period. Other revenue includes $4.1 million derived from approximately 6,400 Shield screening tests reported in the fourth quarter of 2024.

Gross profit, or total revenue less cost of precision oncology testing and cost of development services and other, was $124.2 million for the fourth quarter of 2024, an increase of $31.7 million from $92.5 million for the corresponding prior year period. Gross margin, or gross profit divided by total revenue, was 62%, as compared to 60% for the corresponding prior year period. Precision oncology gross margin was 62% in the fourth quarter of 2024, as compared to 60% in the prior year period. Development services and other gross margin was 53% in the fourth quarter of 2024, as compared to 60% in the prior year period.

Non-GAAP gross profit was $126.4 million for the fourth quarter of 2024, an increase of $31.8 million, from $94.5 million for the corresponding prior year period. Non-GAAP gross margin was 63% for the fourth quarter of 2024, as compared to 61% for the corresponding prior year period.

Non-GAAP gross profit excluding screening was $126.3 million for the fourth quarter of 2024, an increase of $29.0 million, from $97.3 million for the corresponding prior year period. Non-GAAP gross margin excluding screening was 64% for the fourth quarter of 2024, as compared to 63% for the corresponding prior year period.
Operating expenses (research and development expense, sales and marketing expense, and general and administrative expense) were $250.2 million for the fourth quarter of 2024, as compared to $206.6 million for the corresponding prior year period. Other operating expense was $83.4 million for the fourth quarter of 2023, related to a non-recurring legal accrual. No such other operating expense was recorded for the same period of 2024. Non-GAAP operating expenses were $214.7 million for the fourth quarter of 2024, as compared to $183.1 million for the corresponding prior year period.
Net loss was $111.0 million for the fourth quarter of 2024, as compared to $187.0 million for the corresponding prior year period. Net loss per share was $0.90 for the fourth quarter of 2024, as compared to $1.58 for the corresponding prior year period. The year-over-year improvement in net loss was primarily due to the non-recurring $83.4 million legal accrual recorded in 2023.
Non-GAAP net loss was $77.3 million for the fourth quarter of 2024, as compared to $75.9 million for the corresponding prior year period. Non-GAAP net loss per share was $0.62 for the fourth quarter of 2024, as compared to $0.64 for the corresponding prior year period.
Adjusted EBITDA loss was $78.4 million for the fourth quarter of 2024, as compared to a $78.4 million loss for the corresponding prior year period.
Free cash flow for the fourth quarter of 2024 was $(83.4) million, as compared to $(82.8) million for the corresponding prior year period.

Full Year 2024 Financial Results

Revenue was $739.0 million for 2024, a 31% increase from $563.9 million for the corresponding prior year period. Precision oncology revenue grew 34%, to $687.9 million for 2024, from $514.2 million for the corresponding prior year period, driven by an increase in the volume of clinical tests (i.e., Guardant360, TissueNext, Response, and Reveal tests) and biopharma tests, which grew 20% and 35%, respectively, over the prior year period. The increase in precision oncology revenue was also attributable to an increase in reimbursement for our tests, due to an increase in Medicare reimbursement for our Guardant360 LDT test to $5,000, effective January 1, 2024; an increase in both Medicare Advantage and commercial payer reimbursement; and an approximately $22 million non-recurring, out of period revenue upside for 2024 resulting from better than expected cash collections from prior years. Development services and other revenue was $51.1 million for 2024, compared to $49.7 million for the corresponding prior year period. Other revenue includes $5.1 million derived from Shield screening tests reported in 2024 following the FDA approval.

Gross profit, or total revenue less cost of precision oncology testing and cost of development services and other, was $449.2 million for 2024, an increase of $112.3 million from $336.9 million for the corresponding prior year period. Gross margin, or gross profit divided by total revenue, was 61%, as compared to 60% for the corresponding prior year period. Precision oncology gross margin was 62% in the year of 2024, as compared to 60% in the prior year period. Development services and other gross margin was 43% in the year of 2024, as compared to 57% in the prior year period.

Non-GAAP gross profit was $459.6 million for 2024, an increase of $114.7 million, from $344.9 million for the corresponding prior year period. Non-GAAP gross margin was 62% for 2024, as compared to 61% for the corresponding prior year period.
Non-GAAP gross profit excluding screening was $466.9 million for 2024, an increase of $111.2 million, from $355.7 million for the corresponding prior year period. Non-GAAP gross margin excluding screening was 64% for 2024, as compared to 63% for the corresponding prior year period.
Operating expenses (research and development expense, sales and marketing expense, and general and administrative expense) were $892.8 million for 2024, as compared to $818.2 million for the corresponding prior year period. Other operating expense was $83.4 million for 2023, related to the non-recurring legal accrual discussed above. No such other operating expense was recorded for 2024. Non-GAAP operating expenses were $757.3 million for 2024, as compared to $729.2 million for the corresponding prior year period.
Net loss was $436.4 million for 2024, as compared to $479.4 million for the corresponding prior year period. Net loss per share was $3.56 for 2024, as compared to $4.28 for the corresponding prior year period. The year-over-year improvement in net loss was primarily due to the non-recurring $83.4 million legal accrual recorded in 2023, $37.7 million year-over-year improvement in loss from operations, excluding the legal accrual impact, $29.1 million of impairment recorded for our non-marketable equity security investments and other related assets in 2023 and $18.3 million increase in interest income, partially offset by $124.1 million negative change in fair value adjustments recorded for our marketable equity security investment in Lunit, Inc.
Non-GAAP net loss was $247.2 million for 2024, as compared to $352.3 million for the corresponding prior year period. Non-GAAP net loss per share was $2.01 for 2024, as compared to $3.15 for the corresponding prior year period.
Adjusted EBITDA loss was $257.5 million for 2024, as compared to a $344.2 million loss for the corresponding prior year period.
Free cash flow for 2024 was $(274.9) million, as compared to $(345.5) million for the corresponding prior year period.
Cash, cash equivalents, restricted cash and marketable debt securities were $944.2 million as of December 31, 2024. In addition, the aggregate principal amount of the 2027 Convertible Senior Notes (the "2027 Notes") was $1.15 billion as of December 31, 2024.
In February 2025, Guardant Health entered into privately negotiated exchange agreements with certain holders of the outstanding 0% 2027 Notes, pursuant to which Guardant Health issued $600.0 million aggregate principal amount of 1.25% Convertible Senior Notes due 2031 (the "New Notes") in exchange for the retirement of $659.3 million aggregate principal amount of the 2027 Notes (the "Transaction"). Total transaction costs related to the issuance of the New Notes were approximately $12.4 million. In connection with the Transaction, Guardant Health repurchased $45.0 million of shares of its common stock from certain participants in the Transaction through a financial intermediary at a price of $46.09 per share, which was the last reported sale price of its common stock on February 6, 2025 (the "Stock Repurchase"). As of December 31, 2024, pro forma for the Transaction and the Stock Repurchase, cash, cash equivalents, restricted cash and marketable debt securities would have been $886.8 million, and the aggregate principal amount of the 2027 Notes would have been $490.7 million, and the aggregate principal amount of the New Notes would have been $600.0 million, respectively.
2025 Guidance
Guardant Health expects full year 2025 revenue to be in the range of $850 to $860 million, representing growth of 15% to 16% compared to full year 2024. Excluding non-recurring, out of period revenue recorded in 2024, this range implies total revenue growth of 19% to 20% in 2025.
Within this revenue range,
•Oncology revenue is expected to grow approximately 15% year over year in 2025. Excluding non-recurring, out of period revenue recorded in 2024, this range implies oncology revenue growth of approximately 20%. Oncology clinical volume is expected to accelerate to approximately 25% growth in 2025 compared to 20% growth in 2024.
•Screening revenue is expected to be in the range of $25 to $30 million, driven by 45,000 to 50,000 Shield test volume.
•Biopharma & data revenue growth is expected to be in the low double-digit range.

Guardant Health expects full year 2025 non-GAAP gross margin to be in the range of 62% to 63%, compared to 62% in 2024. Guardant Health expects total non-GAAP operating expenses to be in the range of $815 to $825 million, representing an 8 to 9% increase compared to 2024, due to the commercial ramp to support Shield while both R&D and G&A are expected to be approximately flat year over year. Guardant Health expects free cash flow burn to be in the range of $225 to $235 million in 2025, an improvement compared to $275 million for the full year 2024. This includes approximately $200 million of screening net cash burn. Guardant Health expects the remainder of the business excluding screening to reach free cash flow breakeven in the fourth quarter of 2025.

Webcast Information
Guardant Health will host a conference call to discuss the fourth quarter and full year 2024 financial results after market close on Thursday, February 20, 2025 at 1:30 pm Pacific Time / 4:30 pm Eastern Time. A webcast of the conference call can be accessed at View Source The webcast will be archived and available for replay for at least 90 days after the event.