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SpringWorks Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Highlights Recent Business Updates

On February 20, 2025 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, reported financial results for the fourth quarter and full year periods ended December 31, 2024 and provided an update on recent company developments (Press release, SpringWorks Therapeutics, FEB 20, 2025, View Source [SID1234650420]).

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"We are very pleased with the strong execution of OGSIVEO in 2024 and believe that we are still in the early stages of realizing the full potential of our opportunity to serve the desmoid tumor community. With the recent FDA approval of GOMEKLI for adults and children with NF1-PN, we believe we are ready to deliver another strong launch and are delighted that the broad label enables us to help patients throughout their treatment journey," said Saqib Islam, Chief Executive Officer of SpringWorks. "In parallel with our U.S. launches, we are working with urgency to bring our medicines to patients globally and are advancing a diversified pipeline across a variety of indications that provide the potential for us to develop important therapeutic advances for patients who are currently underserved."

Recent Business Highlights and Upcoming Milestones

OGSIVEO (Nirogacestat)

Continued strong commercial execution of the OGSIVEO launch in the U.S. with fourth quarter and full-year 2024 U.S. net product revenue for OGSIVEO of $61.5 million and $172.0 million, respectively.
A Marketing Authorization Application (MAA) for nirogacestat for the treatment of adult patients with desmoid tumors is under review with the European Medicines Agency (EMA). If approved, SpringWorks expects to launch OGSIVEO following reimbursement authorization in individual EU countries, beginning with Germany in mid-2025.
Presented long-term follow-up data from the Phase 3 DeFi trial of nirogacestat in adults with progressing desmoid tumors at the 2024 Connective Tissue Oncology Society Annual Meeting, which highlighted further reductions in tumor size, increase in objective response rate, sustained improvement in desmoid tumor symptoms, and consistent safety profile. SpringWorks expects to publish these data in a peer-reviewed journal in 2025.
SpringWorks expects to report initial data from the Phase 2 trial evaluating nirogacestat as a monotherapy in patients with ovarian granulosa cell tumors in the first half of 2025.
SpringWorks continues to support several industry and academic collaborator studies evaluating nirogacestat as part of B-cell maturation antigen (BCMA) combination therapy regimens across treatment lines in patients with multiple myeloma.
GOMEKLI (Mirdametinib)

On February 11, 2025, SpringWorks received U.S. Food and Drug Administration (FDA) approval for GOMEKLI, an oral MEK inhibitor, for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. GOMEKLI is the first and only medicine approved for both adults and children with NF1-PN. With the approval, SpringWorks was granted a rare pediatric disease priority review voucher (PRV) by the FDA.
GOMEKLI is now available through a specialty pharmacy and specialty distributor network in the United States.
An MAA for mirdametinib for the treatment of adults and children with NF1-PN is under review with the EMA. If approved, SpringWorks expects to begin its initial launch in the European Union in 2025.
A Phase 2 study evaluating mirdametinib in pediatric and young adult patients with low-grade gliomas (LGG) is ongoing and enrolling patients.
Emerging Pipeline

A Phase 1b trial of brimarafenib and Amgen’s EGFR inhibitor, panitumumab, in colorectal and pancreatic cancer patients with known MAPK pathway mutations is ongoing. Brimarafenib is an investigational, selective RAF dimer inhibitor being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene, Ltd.
SpringWorks is continuing to enroll patients in a Phase 1 trial of SW-682, an investigational novel, oral, potent, and selective pan-TEAD inhibitor, in Hippo-mutant solid tumors.
SpringWorks obtained an exclusive, global license from Rappta Therapeutics Oy for a first-in-class molecular glue of specific Protein Phosphatase 2A (PP2A) complexes. PP2A mutations represent a class of targetable oncogenic drivers in molecularly defined subsets of uterine cancer patients with high unmet need. In preclinical models of PP2A mutant uterine cancer, SW-3431 (formerly RPT04402) showed rapid, deep and durable tumor regressions as a monotherapy. SpringWorks expects to file an Investigational New Drug (IND) application for SW-3431 by the end of 2025.
Fourth Quarter and Full Year 2024 Financial Results

Product Revenues: OGSIVEO net product revenues were $61.5 million and $172.0 million in the fourth quarter of 2024 and full year 2024, respectively.
Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $77.1 million and $256.7 million for the fourth quarter and full year 2024, respectively, compared to $59.8 million and $197.6 million for the comparable periods of 2023. The increase in SG&A expense for the fourth quarter and the full year 2024 were largely attributable to commercial readiness activities to support the U.S. launch of GOMEKLI as well as commercial activity supporting the U.S. launch of OGSIVEO.
Research and Development (R&D) Expenses: R&D expenses were $60.2 million and $200.5 million for the fourth quarter and full year 2024, respectively, compared to $43.7 million and $150.5 million for the comparable periods of 2023. The increase in R&D expense for the fourth quarter and year ended 2024 was primarily attributable to an increase in external costs related to licensing fees, drug manufacturing, clinical trials, other research, consulting and professional services.
Net Loss Attributable to Common Stockholders: SpringWorks reported a net loss of $77.3 million, or $1.04 per share, for the fourth quarter of 2024 and a net loss of $258.1 million, or $3.48 loss per share, for the year ended December 31, 2024. This compares to a net loss of $94.3 million, or $1.44 per share, for the fourth quarter of 2023 and a net loss of $325.1 million, or $5.15 per share for the year ended December 31, 2023.
Cash Position: Cash, cash equivalents and marketable securities were $461.9 million as of December 31, 2024.
Additional Information

Additional information on the Company’s results can be found on the Investors and Media section of the SpringWorks website at View Source The previously scheduled conference call and webcast has been cancelled.

SELLAS Announces Positive Data from Phase 2a Trial of SLS009 in Combination with Zanubrutinib in DLBCL

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]).

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."

PTC Therapeutics to Participate in Upcoming Investor Conferences

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]):

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.

First patient treated with new extended dosing of OVM-200 cancer vaccine

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.

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.

OXC-101 has been granted ODD status by the FDA in AML

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]).

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.