On August 5, 2025 Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported financial results for the second quarter 2025 and associated Company developments (Press release, Supernus, AUG 5, 2025, View Source [SID1234654798]).

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"Our strong operating performance in the first half of the year was driven by continued strong sales growth of Qelbree and GOCOVRI, which combined accounted for 72% of total net sales in the second quarter of 2025," said Jack Khattar, President and CEO of Supernus. "Our focus for the second half of the year remains on the launch of ONAPGO, the successful integration of Sage Therapeutics, and the continued performance of our growth products," said Jack Khattar, President and CEO of Supernus.

Acquisition of Sage Therapeutics, Inc.

The Company completed the acquisition of Sage Therapeutics on July 31, 2025, strengthening its leading presence in neuropsychiatric conditions with an innovative commercial product, ZURZUVAE (zuranolone), and a novel CNS discovery platform. The acquisition is expected to accelerate mid- to long-term revenue and cash flow growth and further diversify the Company’s revenue base.
Commercial Highlights

Total IQVIA prescriptions(2) for Qelbree were 225,254 for the second quarter 2025, an increase of 23% compared to the same period in the prior year. Qelbree continues to expand its base of prescribers, with approximately 36,000 prescribers in the second quarter of 2025, up by 23% compared to the same period last year.

In April 2025, the Company launched ONAPGO, the first and only subcutaneous apomorphine infusion device for the treatment of motor fluctuations in adults with advanced Parkinson’s disease. ONAPGO demand is exceeding the Company’s expectations, with more than 750 enrollment forms submitted by more than 300 prescribers through the end of the second quarter of 2025.
Product Pipeline Update

SPN-817 – Novel first-in-class highly selective AChE inhibitor for epilepsy

The Phase 2b randomized, double-blind, placebo-controlled study of 3mg and 4mg twice daily doses is ongoing with a targeted enrollment of approximately 258 adult patients with treatment resistant focal seizures.
SPN-820 – Novel first-in-class molecule that increases mTORC1 mediated synaptic function for depression

The Company plans to initiate a follow-on Phase 2b multi-center, randomized, double-blind, placebo-controlled trial in approximately 200 adults with major depressive disorder (MDD). The study will examine the safety and tolerability of SPN-820 2400 mg given intermittently (twice weekly) as an adjunctive treatment to the current baseline antidepressant therapy, as well as assess the rapid onset of improvement in depressive symptoms. The Company expects to initiate the Phase 2b study by the end of 2025.
SPN-443 – Novel stimulant for ADHD/CNS

The Company completed a Phase 1 pharmacokinetic study of two oral formulations in healthy adults. Both formulations of SPN-443 showed adequate bioavailability and were well tolerated. The Company expects to disclose a lead indication for the product candidate by the end of 2025.
Financial Highlights

This section includes information on non-GAAP financial measures. See "Non-GAAP Financial Information" section for information on non-GAAP financial measures. In addition, a reconciliation of applicable GAAP to non-GAAP financial information is included at the end of this press release.

Revenues

The following table provides information regarding total revenues (dollars in millions):

Three Months Ended
June 30, Six Months Ended
June 30,
2025 2024 Change % 2025 2024 Change %
(unaudited)
Net product sales
Qelbree $ 77.6 $ 59.4 31 % $ 142.3 $ 104.5 36 %
GOCOVRI 36.7 31.7 16 % 67.4 58.3 16 %
APOKYN 12.8 17.3 (26 )% 27.8 33.9 (18 )%
Trokendi XR 11.2 17.1 (35 )% 24.0 33.1 (27 )%
Oxtellar XR 11.6 29.5 (61 )% 21.8 56.5 (61 )%
ONAPGO 1.6 — 100 % 1.6 — 100 %
Other(3) 6.5 7.5 (13 )% 15.1 14.7 3 %
Total net product sales 158.0 162.5 (3 )% 300.0 301.0 — %
Royalty, licensing and other revenues(4) 7.5 5.8 29 % 15.3 11.0 39 %
Total revenues $ 165.5 $ 168.3 (2 )% $ 315.3 $ 312.0 1 %

Total revenues excluding Trokendi XR and Oxtellar XR net sales (non-GAAP)(1) $ 142.7 $ 121.7 17 % $ 269.5 $ 222.4 21 %

Other Financial Highlights

Operating earnings were $12.1 million and $1.9 million for the three and six months ended June 30, 2025, compared to operating earnings of $22.6 million and $19.4 million for the same periods in 2024. The change was primarily due to higher contingent consideration loss, mainly related to the achievement of ONAPGO-related milestones, and higher selling and marketing expenses.
Adjusted operating earnings (non-GAAP) were $40.9 million and $66.9 million for the three and six months ended June 30, 2025, compared to $45.5 million and $67.7 million for the same periods in 2024.
Net earnings and diluted earnings per share were $22.5 million and $0.40 for the three months and $10.7 million and $0.19 for the six months ended June 30, 2025, respectively, compared to net earnings and diluted earnings per share of $19.9 million and $0.36 for the three months and $20.0 million and $0.36 six months ended June 30, 2024, respectively.
At June 30, 2025, cash, cash equivalents, and current marketable securities were approximately $522.6 million compared to $453.6 million as of December 31, 2024. This increase was primarily due to cash generated from operations.

On August 5, 2025 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a late-stage immunotherapy company targeting cancer and autoimmune diseases, reported it has received positive and constructive feedback from the US Food and Drug Administration (FDA), regarding future clinical development of its first-in-class MHC Class II agonist, eftilagimod alfa ("efti"), for first line treatment of recurrent/metastatic head and neck squamous cell carcinoma (1L HNSCC) patients who have PD-L1 expression below 1 (Combined Positive Score [CPS] <1) (Press release, Immutep, AUG 5, 2025, View Source [SID1234654981]).

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Based on its review of the encouraging data in 1L HNSCC with CPS <1 from the TACTI-003 (KEYNOTE-C34) Phase IIb trial evaluating efti in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD-1 KEYTRUDA (pembrolizumab), the FDA agreed on the potential of efti in combination with KEYTRUDA to address the high unmet need in this CPS <1 patient segment and is supportive of the combination’s further development.

Paths for future clinical development and potential accelerated approval in light of the FDA’s Project FrontRunner include a randomised registrational trial evaluating efti in combination with KEYTRUDA against standard-of-care therapy or alternatively a smaller single-arm study (e.g. 70 – 90 patients) with safety, response rate, and duration of response as key endpoints, followed by a confirmatory randomised study that builds on the existing data.

"We are pleased with the FDA’s feedback and guidance that underscores the high unmet need of head and neck cancer patients whose PD-L1 expression level is below one. The FDA feedback positions Immutep to evaluate options for future collaborative clinical development paths to bring a new, effective and safe treatment option to this underserved patient population," said Marc Voigt, CEO of Immutep.

"Our primary focus clearly remains the pivotal TACTI-004 Phase III evaluating efti as first line therapy for non-small cell lung cancer and we are excited with its progress to date and the consistent, encouraging feedback we hear from physicians. This focus and additional considerations will be reviewed internally and discussed with stakeholders and potential strategic partners in regards to forward paths in head and neck cancer," added Mr Voigt.

Project FrontRunner is an FDA Oncology Center of Excellence (OCE) initiative to encourage drug sponsors to consider when it may be appropriate to develop and seek approval of cancer drugs for advanced/metastatic disease, in an earlier clinical setting rather than the usual approach to develop and seek approval of a drug for treatment of patients who have received numerous prior lines of therapies or have exhausted available treatment options. In this setting, advancing new effective therapies has the greatest potential to significantly improve quantity and quality of patients’ lives.

Patients with CPS <1 in 1L HNSCC represent a treatment population with high unmet medical need. Up to 20% of 1L HNSCC patients have CPS <1 and despite immunotherapy’s progress in fighting cancer, anti-PD-1 therapy alone (without chemotherapy) is only approved for patients who express PD-L1 (CPS >1). All currently available treatment options for patients with PD-L1 CPS <1 include chemotherapy.

On August 5, 2025 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of emavusertib (CA-4948), an orally available, small molecule IRAK4 inhibitor, reported its financial and operating results for the second quarter ended June 30, 2025 (Press release, Curis, AUG 5, 2025, View Source [SID1234654783]).

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"We are pleased with our progress in the TakeAim Lymphoma study and are continuing to enroll PCNSL patients in both Part B (BTKi-experienced patients) and Part C (BTKi-naïve patients) to enable accelerated approval filings in the US and EU. We look forward to providing updated data later this year," said James Dentzer, President and Chief Executive Officer. "We are also excited to add a clinical study of emavusertib in combination with a BTK inhibitor in Chronic Lymphocytic Leukemia, as part of our strategy to expand across CLL and NHL and bring this novel treatment option to broader patient populations with unmet needs. For CLL patients in particular, this could represent a fundamental change in the treatment paradigm, with the potential of an emavusertib-BTKi combination to enable time-limited treatment. We look forward to beginning enrollment later this year."

Operational Highlights

NHL/CLL

Presented at the 2025 Annual Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting the poster titled, Analysis of genetic mutation profile and CNS pharmacokinetics in relapsed/refractory primary CNS lymphoma patients responding to novel emavusertib (IRAK4i) and BTKi combination.
Continued to enroll relapsed/refractory Primary Central Nervous System Lymphoma (R/R PCNSL) patients in the Company’s TakeAim Lymphoma study which, as a result of discussions with the EMA and FDA, is now enrolling patients to support registration. Emavusertib has been granted orphan drug designation by both the FDA and EMA in PCNSL.
Adding a proof-of-concept clinical study of emavusertib in combination with a BTKi in 20-30 patients with R/R CLL with the goal of improving upon the current standard of care (BTKi) with its limitations of partial responses and life-long therapy. The combination of emavusertib with a BTKi has the potential to enable patients to achieve complete remission or minimal residual disease (MRD) negativity and time-limited treatment, which would be a paradigm shift in the management of CLL.
Leukemia

Completed enrollment in the TakeAim Leukemia Phase 1/2 study of emavusertib as a monotherapy in patients with R/R Acute Myeloid Leukemia (AML) or R/R Myelodysplastic Syndrome (MDS) and are following several patients still on study treatment.
Engaged clinical trial investigators and key opinion leaders at ASCO (Free ASCO Whitepaper) and European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress on the design of a head-to-head registrational trial vs. gilteritinib in R/R AML based upon encouraging data, Preliminary Safety, Efficacy and Molecular Characterization of Emavusertib (CA-4948) in Relapsed/Refractory Acute Myeloid Leukemia Patients, presented by Dr. Eric Winer at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.
Engaged clinical trial investigators and key opinion leaders at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) on the design of a Phase 1/2 study in frontline high-risk MDS (hrMDS) in combination with azacitidine based upon encouraging R/R MDS clinical data at ASH (Free ASH Whitepaper) 2024 in the poster, Preliminary Safety, Efficacy and Molecular Characterization in Higher-Risk Myelodysplastic Syndrome Patients Treated with Single Agent Emavusertib (CA-4948).
Ongoing triplet study evaluating different dosing regimens of emavusertib, venetoclax, and azacitidine in frontline AML, with data expected at the 67th ASH (Free ASH Whitepaper) Annual Meeting in December.
Corporate

Completed a registered direct offering and concurrent private placement extending cash runway to the first quarter of 2026.
Upcoming Presentations and Conferences

Cantor Global Healthcare Conference 2025 – September 2-5, 2025
H.C. Wainwright 27th Annual Global Investment Conference – September 8-10, 2025
Upcoming Milestones

Additional data in R/R PCNSL patients from the TakeAim Lymphoma study is expected later this year.
Second Quarter 2025 Financial Results

For the second quarter of 2025, Curis reported a net loss of $8.6 million or $0.68 per share on both a basic and diluted basis as compared to $11.8 million or $2.03 per share on both a basic and diluted basis, for the same period in 2024. Curis reported a net loss of $19.2 million or $1.82 per share on both a basic and diluted basis, for the six months ended June 30, 2025, as compared to a net loss of $23.7 million or $4.08 per share on both a basic and diluted basis for the same period in 2024.

Revenues for the second quarter of 2025 were $2.7 million as compared to $2.5 million for the same period in 2024. Revenues were $5.1 million for the six months ended June 30, 2025, as compared to $4.6 million for the same period in 2024. Revenues consist of royalty revenues from Genentech/Roche’s sales of Erivedge.

Research and development expenses were $7.5 million for the second quarter of 2025, as compared to $10.3 million for the same period in 2024. The decrease was primarily attributable to lower employee related, research, consulting, and clinical costs. Research and development expenses were $16.0 million for the six months ended June 30, 2025, as compared to $19.9 million for the same period in 2024.

General and administrative expenses were $3.5 million for the second quarter of 2025, as compared to $4.8 million for the same period in 2024. The decrease was primarily attributable to lower employee-related and legal costs. General and administrative expenses were $7.5 million for the six months ended June 30, 2025, as compared to $9.7 million for the same period in 2024.

Other expense was $0.3 million for the second quarter of 2025, as compared to other income of $0.7 million for the same period in 2024. The decrease was primarily attributable to a decrease in interest income and an increase in the expense related to the sale of future royalties. Other expense was $0.8 million for the six months ended June 30, 2025, compared to other income of $1.3 million for the same period in 2024.

Curis’s cash and cash equivalents totaled $10.1 million as of June 30, 2025, and the Company had approximately 10.7 million shares of common stock outstanding. We believe that our existing cash and cash equivalents, together with the net proceeds of $6.0 million from the July 2025 Offerings, should enable us to fund our existing operations into the first quarter of 2026.

Conference Call and Webcast Information

Curis management will host a conference call today, August 5, 2025, at 8:30 a.m. ET, to discuss the business update and these financial results.

To access the live conference call, please dial 1-800-836-8184 from the United States or 1-646-357-8785 from other locations. To access the webcast login here shortly before 8:30 a.m. ET. The webcast can also be accessed on the Curis website at the Events and Presentations section of the Investors page.

On August 5, 2025 SynOx Therapeutics Limited ("SynOx"), a late-stage clinical biopharmaceutical company developing emactuzumab for Tenosynovial Giant Cell Tumours (TGCT), reported completion of patient enrolment in the TANGENT study (Press release, SynOx Therapeutics, AUG 5, 2025, View Source [SID1234654799]). TANGENT is the company’s global, multi-centre, randomized, double-blind, placebo-controlled registrational Phase 3 trial of emactuzumab in patients with TGCT who are not amenable to or who would not benefit from surgery. SynOx expects to report top-line data from the study in the first quarter of 2026.

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Emactuzumab is a potentially best-in-class CSF-1 receptor (CSF-1R) inhibiting monoclonal antibody (mAb). Clinical evaluation to date has demonstrated the compound to be a promising, next-generation mAb therapy with the potential to offer a short treatment cycle, rapid onset and long duration of response that differentiates it from chronically administered oral agents. This potentially best-in-class profile helped drive strong interest in the TANGENT trial among patients and clinical investigators across sites in the United States, Canada, Europe and Asia, leading to rapid study enrollment.

"Completion of enrollment in our registrational TANGENT trial marks an important milestone for SynOx and for the TGCT community," said Elyse Seltzer, M.D., Chief Medical Officer of SynOx Therapeutics. "We are deeply grateful to the patients, families, investigators, and clinical sites whose dedication has made this study possible and allowed SynOx to fully enroll the trial significantly ahead of our projected timeline. We remain committed, data permitting, to delivering a much-needed new treatment option for this debilitating condition."

"This is a transformational time for SynOx as we advance emactuzumab through its pivotal Phase 3 program," said Ray Barlow, Chief Executive Officer of SynOx. "We’re excited about the next steps and look forward to delivering top-line data that we hope demonstrate how emactuzumab can transform care for patients with this grievous disease."

About the TANGENT Study

TANGENT (ClinicalTrials.gov Identifier: NCT05417789) is a randomized, double-blind, placebo-controlled Phase 3 trial evaluating the efficacy and safety of emactuzumab in patients with TGCT who are not amenable to or would not benefit from surgery. Patients who consent and meet eligibility criteria are randomized in a 2:1 fashion to receive either emactuzumab (1000 mg every two weeks for five doses) or matched placebo.

The primary endpoint is objective response rate (ORR) as assessed by RECIST criteria at six months post-randomization. Key secondary endpoints include patient-reported outcomes (PROMIS-PF), functional assessments of range of motion, pain, stiffness, durability of response, and safety. Patients are followed for two years from randomization, with those demonstrating disease progression after the six-month assessment eligible to receive open-label emactuzumab during follow-up.

On August 5, 2025 Day One Biopharmaceuticals, Inc. (Nasdaq: DAWN) ("Day One" or the "Company"), a biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, reported its second quarter 2025 financial results and highlighted recent corporate achievements (Press release, Day One, AUG 5, 2025, View Source [SID1234654784]).

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"We have strong momentum going into the second half of 2025. We continue to focus on our three core priorities: accelerating revenue growth with OJEMDA, advancing our pipeline, and pursuing value-driving portfolio expansion anchored in financial discipline," said Jeremy Bender, Ph.D., chief executive officer of Day One. "With strong execution across the organization and a solid financial foundation, we’re building a company that aims to deliver meaningful value to patients and to shareholders."

OJEMDA Commercial Performance

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OJEMDA net product revenue was $33.6 million in the second quarter of 2025, an increase of 310% from the second quarter of 2024.

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U.S. OJEMDA net product revenue increased 10% from the first quarter of 2025.

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OJEMDA prescriptions exceeded 1,000 in the second quarter of 2025, representing a 15% increase compared to the first quarter of 2025 and a 346% increase compared to the second quarter of 2024.

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Achieved $113.1 million in OJEMDA net product revenue for the most recent 12-month period ended June 30, 2025.

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The Company is providing 2025 net product revenue guidance of $140 to $150 million.

Program Highlights

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DAY301, the Company’s PTK7-targeted ADC, is actively enrolling patients in the Phase 1a portion of the Phase 1a/b clinical trial; the trial is progressing as planned.

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Day One expects to present 3-year follow-up data from the FIREFLY-1 clinical trial in the fourth quarter of 2025.

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Day One published additional data characterizing growth velocity recovery and effective rash management at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

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Abstract 10029: Growth recovery in patients with BRAF-altered pediatric low-grade gliomas (pLGGs) after discontinuation of tovorafenib
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Abstract 10037: Post hoc analysis of rashes reported in patients with BRAF-altered relapsed/refractory pediatric low-grade glioma treated with the type II RAF inhibitor tovorafenib in FIREFLY-1

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Patient enrollment in the pivotal Phase 3 FIREFLY-2 clinical trial is on track to achieve completion of trial enrollment in the first half of 2026.

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Day One terminated its research collaboration and license agreement with Sprint Bioscience AB following careful consideration of the current development status for the VRK1 program and the Company’s overall strategic objectives.

Corporate Highlights

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Industry leader Michael Vasconcelles, M.D., joined Day One in June 2025 as Head of Research and Development. Dr. Vasconcelles brings more than 25 years of extensive oncology research and development experience to the Company, most recently as Executive Vice President and Head of Research, Development and Medical Affairs at ImmunoGen.

Second Quarter 2025 Financial Highlights

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Product Revenue, Net: OJEMDA net product revenue was $33.6 million for the second quarter of 2025 compared to $8.2 million for the second quarter of 2024.

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License Revenue: License revenue from the sale of ex-U.S. commercial rights for tovorafenib was $0.3 million for the second quarter of 2025.

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R&D Expenses: Research and development expenses were $36.1 million for the second quarter of 2025 compared to $92.1 million for the second quarter of 2024. The decrease was primarily due to the MabCare Therapeutics license agreement upfront payment of $55.0 million in the second quarter of 2024.

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SG&A Expenses: Selling, general and administrative expenses were $29.0 million for the second quarter of 2025 compared to $30.2 million for the second quarter of 2024. The decrease was primarily due to lower employee compensation costs.

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Net Loss: Net loss totaled $30.3 million for the second quarter of 2025 with non-cash stock-based compensation expense of $10.9 million, compared to a net loss of $4.4 million for the second quarter of 2024, with non-cash stock-based compensation expense of $13.0 million and gain from sale of priority voucher of $108.0 million.

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Cash Position: The Company’s cash, cash equivalents and short-term investments totaled $453.1 million as of June 30, 2025.

Conference Call

Day One will host a conference call and webcast today, August 5 at 4:30 p.m. ET. To access the live conference call by phone, dial 877-704-4453 (domestic) or 201-389-0920 (international), and provide the access code 13745150. Live audio webcast will be accessible from the Day One Media & Investors page. To ensure a timely connection to the webcast, it is recommended that participants register at least 15 minutes prior to the scheduled start time. An archived version of the webcast will be available for replay on the Events section of the Day One Investors & Media page for 30 days following the event.

About OJEMDA

OJEMDA (tovorafenib) is a Type II RAF kinase inhibitor of mutant BRAF V600, wild-type BRAF, and wild-type CRAF kinases.

OJEMDA is indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Tovorafenib was granted Breakthrough Therapy and Rare Pediatric Disease designations by the FDA for the treatment of patients with pLGG harboring an activating RAF alteration, and it was evaluated by the FDA under priority review. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma and from the European Commission for the treatment of glioma.

For more information, please visit www.ojemda.com.