On August 11, 2025 Summit Therapeutics Inc. (NASDAQ: SMMT) ("Summit," "we," or the "Company") reported its financial results and provides an update on operational progress for the second quarter ended June 30, 2025 (Press release, Summit Therapeutics, AUG 11, 2025, View Source [SID1234655086]).

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Operational & Corporate Updates

Operational progress continues with ivonescimab (SMT112), an investigational, potentially first-in-class bispecific antibody combining the effects of immunotherapy via a blockade of PD-1 with the anti-angiogenesis effects associated with blocking VEGF into a single molecule:

Since in-licensing ivonescimab (SMT112), from Akeso Inc. (Akeso, HKEX Code: 9926.HK) in January 2023, over 2,800 patients have been treated in clinical studies globally. Summit has rights to develop and commercialize ivonescimab in the United States, Canada, Europe, Japan, Latin America, including Mexico and all countries in Central America, South America, and the Caribbean, the Middle East, and Africa while Akeso retains development and commercialization rights for the rest of the world, including China.
Summit is developing ivonescimab in non-small cell lung cancer ("NSCLC"), specifically conducting Phase III clinical trials in the following proposed indications:
HARMONi: Ivonescimab combined with chemotherapy in patients with epidermal growth factor receptor (EGFR)-mutated, locally advanced or metastatic non-squamous NSCLC who have progressed after treatment with a third-generation EGFR tyrosine kinase inhibitor (TKI)
HARMONi-3: Ivonescimab combined with chemotherapy in first-line patients with metastatic NSCLC
HARMONi-7: Ivonescimab monotherapy in patients with first-line metastatic NSCLC whose tumors have high PD-L1 expression
In May 2025, we announced topline results from our multiregional, double-blinded, placebo-controlled, Phase III study, HARMONi.
At the prespecified primary data analysis, ivonescimab in combination with chemotherapy demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS), with a hazard ratio of 0.52 (95% CI: 0.41 – 0.66; p<0.00001). PFS was measured by blinded independent central radiology review committee (BICR) compared to placebo in combination with chemotherapy.
A clinically meaningful hazard ratio was observed in both Asia and ex-Asia sub-populations. The primary analysis demonstrated the consistency of the magnitude of the PFS benefit between patients randomized in Asia and ex-Asia, as well as the consistency in a single-region study (HARMONi-A) with this multiregional study.
Ivonescimab in combination with chemotherapy showed a positive trend in overall survival (OS) in the primary analysis without achieving a statistically significant benefit with a hazard ratio of 0.79 (95% CI: 0.62 – 1.01; p=0.057). This trend provides further support for its use in 2L+ EGFRm NSCLC, a setting where high unmet need continues to exist with limited approved options in the United States and other western territories. Currently there are no FDA-approved regimens that have demonstrated a statistically significant OS benefit in this patient setting. The median follow-up time for western patients was less than the median OS at the time of the analysis, and these patients may continue to be followed for long-term outcomes. Both Asian and North American patients demonstrated a positive trend in OS. The results of the primary analysis in this multiregional study were consistent with that of the single-region HARMONi-A study, which demonstrated an OS hazard ratio of 0.80 at 52% data maturity in a similar patient population.
The safety profile of ivonescimab in combination with chemotherapy was acceptable and manageable in the context of the observed clinical benefit.
Based on the results of the HARMONi clinical trial, Summit, at present time, intends to file a Biologics License Application (BLA) in order to seek approval for ivonescimab plus chemotherapy in this setting. Based on discussions with the United States Food & Drug Administration (FDA), under our determination and subject to our review, Summit will consider the timing of the filing of this BLA.
A more complete data presentation from HARMONi is intended to be shared at a future major medical conference.
In April 2025, Akeso announced that HARMONi-6 met its primary endpoint of PFS. This trial, conducted in China by our partners at Akeso with all relevant data exclusively generated, managed, and analyzed by Akeso, evaluated ivonescimab combined with platinum-based chemotherapy against tislelizumab, a PD-1 inhibitor, with the same chemotherapy in patients with locally advanced or metastatic squamous NSCLC, regardless of PD-L1 expression. HARMONi-6 showed statistically significant and clinically meaningful improvement in PFS for ivonescimab plus chemotherapy, and no new safety signals were identified. This marks the first known Phase III trial in NSCLC to show significant improvement over PD-(L)1 inhibitor therapy combined with chemotherapy in a head-to-head setting. Following the success of Akeso’s HARMONi-2 study in China, this is the second instance where ivonescimab-based regimens have demonstrated a statistically significant benefit compared to standard-of-care PD-(L)1 inhibitor-based regimens in a Phase III. The full data set for HARMONi-6 is planned to be presented at an upcoming major medical conference.
Also in April 2025, Akeso announced that ivonescimab was approved by the Chinese Health Authorities, the National Medical Products Administration (NMPA), for a second indication based on the results of the Phase III clinical trial, HARMONi-2. HARMONi-2 evaluated monotherapy ivonescimab against monotherapy pembrolizumab in patients with locally advanced or metastatic NSCLC whose tumors have positive PD-L1 expression. In conjunction with the approval announcement, Akeso announced that the results of a NMPA-requested interim OS analysis included a hazard ratio of 0.777. The analysis was conducted at 39% data maturity, with a nominal alpha level of 0.0001. HARMONi-2 is a single region, multi-center, Phase III study conducted in China sponsored by Akeso with all relevant data exclusively generated, managed, and analyzed by Akeso.
Clinical trial collaborations and investigator sponsored trials with leading organizations, including MD Anderson, the Memorial Sloan Kettering Cancer Center, and the Dana Farber Cancer Institute, among others, continue to progress and expand evaluating ivonescimab in solid tumor settings outside of metastatic NSCLC.
In June 2025, we announced a clinical collaboration with Revolution Medicines to evaluate ivonescimab in combination with three RAS(ON) inhibitors, including the multi-selective inhibitor daraxonrasib (RMC-6236), G12D-selective inhibitor zoldonrasib (RMC-9805), and G12C-selective inhibitor elironrasib (RMC-6291), in solid tumor settings with RAS mutations.
Enrollment continues in Summit’s global Phase III trials, HARMONi-3 and HARMONi-7. In addition to the enrollment in multiregional studies conducted and sponsored by Summit, our partners at Akeso are also enrolling several single-region Phase III studies exclusively in China in multiple indications, including biliary-tract cancer, triple-negative breast cancer, head and neck squamous cell carcinoma, microsatellite stable colorectal cancer, and pancreatic cancer.
Financial Highlights

Cash and Cash Equivalents and Short-term Investments

Aggregate cash and cash equivalents and short-term investments were $297.9 million and $412.3 million at June 30, 2025 and December 31, 2024, respectively.
On August 11, 2025, the Company amended its Distribution Agreement with J.P. Morgan Securities LLC, (the "Sales Agent"), pursuant to which the Company may offer and sell, in an at-the-market (ATM) offering, from time to time, through the Sales Agent, additional shares of the Company’s common stock, having an aggregate offering price of up to $360.0 million. The Company filed a prospectus supplement with the SEC on August 11, 2025 in connection with this offer and sale of the shares pursuant to the Distribution Agreement. The Company has no obligation to sell any of the shares under the Distribution Agreement and may at any time suspend solicitations and offers under the Distribution Agreement.
Stock-Based Compensation Modification Expense

On April 29, 2025, the compensation committee of the board of directors approved a modification to the Company’s outstanding unvested performance-based stock option awards for certain employees and executives in order to require only service-based vesting requirements to continue vesting considering the overall performance of the company including achievement of the performance goals related to market capitalization of the company for a sustained period of time. As a result, certain options immediately vested on the date of modification, and the remaining options continue to vest over a designated period of time.
On the modification date, 44.5 million options were valued. These 44.5 million options which were modified represent approximately 6% of total shares outstanding as of June 30, 2025. There had been no prior expense recognized for these unvested performance-based stock options. Based on generally accepted accounting principles in the U.S. (US GAAP), total non-cash stock-based compensation expense for this modification was calculated based on the closing share price of $23.62 on the date of modification.
Non-cash stock-based compensation expense for the stock options which were immediately vested on the modification date was calculated based on their intrinsic value. For the options which will continue to vest over the future service period, non-cash stock-based compensation expense was calculated using the Black-Scholes valuation methodology.
For this modification, total non-cash stock-based compensation expense of $466.6 million was recognized during the three months ended June 30, 2025. The unrecognized non-cash stock-based compensation expense of $454.6 million will be recognized over the future remaining service period.

On August 11, 2025 Biohaven Ltd. (NYSE: BHVN) (Biohaven or the Company), a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies to treat a broad range of rare and common diseases, reported financial results for the second quarter ended June 30, 2025, and provided a review of recent accomplishments and anticipated upcoming developments (Press release, Biohaven Pharmaceutical, AUG 11, 2025, View Source [SID1234655084]).

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Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven, commented, "As we eagerly await a regulatory decision on the VYGLXIA (troriluzole) NDA for spinocerebellar ataxia, Biohaven has made important progress on multiple clinical stage assets this quarter, highlighted by the momentum we showcased at our recent R&D Day, where we unveiled advancements across our innovative therapeutic platforms." Dr. Coric added, "We are excited about the prospects of launching the first treatment for SCA if VYGLXIA is approved by the FDA and our commercial team is taking the appropriate steps to ensure an efficient launch to meet this high unmet need. We are also enthusiastic about the progress made across our Inflammation and Immunology (I&I) platform where we have observed compelling evidence of targeted protein degradation with our MoDE and TRAP degraders, BHV-1300 and BHV-1400. The body of evidence we have presented to date, combined with the safety profiles observed and convenient subcutaneous administration, continues to support our belief in our degrader platform’s ultimate potential in addressing a range of immune-mediated diseases. We are also very pleased with the advancement of another key pillar of our I&I platform — the brain-penetrant, TYK2/JAK1 inhibitor, BHV-8000, which has the potential to revolutionize the treatment of neuroinflammatory and neurodegenerative diseases. We initiated a pivotal Phase 2/3 study in Parkinson’s disease, an unrelenting illness for which there is an urgent need for novel therapies to halt the progression of the disease."

Dr. Coric continued, "We also continue to take bold steps in other therapeutic areas to address important unmet medical needs for patients including in our Ion Channel and Oncology platforms. Our Kv7 platform continues to advance clinical programs toward completion in epilepsy and depression, and we are pleased to hear the promising early observations of a DEE pediatric patient successfully transitioned from ezogabine to opakalim. Finally, our Oncology platform is also generating early promising clinical data, demonstrating tumor reduction in the first 6 out of 6 patients treated with BHV-1510 plus cemiplimab. Our oncology team has also been the first to advance an FGFR3-directed ADC with potential application in urothelial cancers into clinical testing."

"Biohaven is committed to following cutting edge science to attempt to help patients across multiple areas of high unmet need. For the balance of the year, we expect to deliver continued excellence in study execution and patient enrollment across key trials in our portfolio, as well as prepare for the potential commercialization of VYGLXIA in SCA if approved. We believe the SCA data supports approval in this rare, progressive and fatal indication for which no other treatments are available. Biohaven is well-positioned to execute on our commitment to transforming the treatment landscape for patients with serious and underserved diseases and we are excited to deliver on our promise to advance our programs for patients, caregivers, and shareholders in the balance of the year."

Second Quarter 2025 and Recent Business Highlights

Released new data with MoDE (Molecular Degrader of Extracellular Proteins) program: In May 2025, the Company released new positive data from the Phase 1 study of BHV-1300, a MoDE initially being developed for the treatment of common immune-mediated diseases, such as Graves’ disease and rheumatoid arthritis. In the Phase 1 multiple-dose study, subcutaneous administered BHV-1300 achieved IgG reductions up to 87%. Median maximum reductions of 83% were achieved within 18 days. The range of IgG lowering enabled by different dose levels of BHV-1300 potentially offers tunability and flexibility in dosing paradigm, with higher doses planned for management of acute conditions, and lower, less frequent dosing planned for the management of chronic disease.
Released new data with TRAP (Targeted Removal of Aberrant Protein) degrader program: In May 2025, the Company announced further data from the Phase 1 study of BHV-1400, a TRAP degrader initially being developed to target Gd-IgA1, the aberrant immunoglobulin that drives IgA Nephropathy. In the Phase 1 study, a single dose of BHV-1400 was subcutaneously administered at a dose of 500 mg and achieved rapid, deep and sustained reductions in Gd-IgA1 of up to 81%, with a median reduction of 66%. Reductions occurred within hours of each dose, were progressive, and were sustained for weeks after a single dose administration. Effects were selective, with no significant reductions observed in other immunoglobulins (IgA, IgG, IgE, or IgM).
Demonstrated early clinical activity and favorable PK profile with BHV-1510: In May 2025, the Company announced early clinical results from a Phase 1 study of BHV-1510, a next-generation Trop2-directed ADC incorporating the proprietary TopoIx payload. The data demonstrated early signs of clinical activity and a differentiated, manageable safety profile, both as monotherapy and in combination with Regeneron’s anti-PD-1, cemiplimab. Partial responses were observed across multiple tumor types with BHV-1510 monotherapy, accompanied by low rates of payload-related toxicities. The most common adverse event was stomatitis, an anticipated and manageable class effect associated with Trop2-targeted therapies. Notably, tumor reduction was seen in all six of the first patients treated with the BHV-1510 and cemiplimab combination, including confirmed partial responses. The combination regimen was well tolerated, with no dose-limiting toxicities or cases of interstitial lung disease reported in these initial cohorts. These encouraging early clinical data, along with the favorable pharmacokinetic profile and proprietary stable linker technology, support continued investigation of BHV-1510 as monotherapy and in combination with cemiplimab in difficult-to-treat tumor types, including potential evaluation in earlier lines of therapy for patients with advanced or metastatic disease.
First patient dosed with Biohaven’s TopoIx ADC, BHV-1530: In May 2025, the Company commenced dosing with BHV-1530, a potential first-in-class fibroblast growth factor receptor 3 (FGFR3)-directed ADC which utilizes the proprietary Topolx payload. BHV-1530 has potential in cancer indications driven by FGFR3 alterations and/or upregulated FGFR3 protein expression, including urothelial cancers and other solid tumors.
Phase 2/3 PD trial initiated: In May 2025, the Company commenced enrollment in a global, pivotal, Phase 2/3 study of the first-in-clinic, orally-administered, brain-penetrant, and highly selective TYK2/JAK1 inhibitor, BHV-8000, for the treatment of early Parkinson’s disease (PD).
Compassionate use of opakalim (BHV-7000) in a child with intractable epilepsy due to Kv7 gene mutation (KCNQ2 Developmental and Epileptic Encephalopathy [KCNQ2-DEE]) provides early evidence of potential clinical benefit. A child with KCNQ2-DEE and a history of intractable epilepsy who was previously maintained on ezogabine, as well as other antiepileptics, was successfully transitioned to treatment with opakalim, Biohaven’s next-generation Kv7 activator. Opakalim was administered after receiving a compassionate use request, under a single patient IND approved by the FDA, as the child was being withdrawn from ezogabine treatment. The child had multiple unsuccessful attempts in the past to taper ezogabine, leading to severe seizure exacerbations requiring admission to the hospital intensive care unit. Dosing of opakalim in this pediatric patient was selected to achieve comparable exposures as the 75mg dose being investigated in ongoing Phase 2/3 clinical trials. Following the transition, the patient demonstrated signs of therapeutic benefit as assessed by initial seizure control and a favorable side effect profile. Although generalizability of these observations is limited, given it represents a single case report of treating a KCNQ2-DEE patient and a short initial follow-up period after transition from ezogabine, the early clinical experience after initiation of opakalim, a selective Kv7 activator, is promising for its observed antiseizure effects and favorable tolerability.
Phase 3 trial in OCD with troriluzole was completed with no efficacy signal detected. The OCD development program is being ended to allow resources to be applied to other development programs. Study results will be presented at an upcoming academic meeting.
Expected Upcoming Milestones:

We believe Biohaven is well positioned to achieve significant milestones in 2025 and 2026 across numerous programs:

MoDE Platform

IgG MoDE Degraders (1300/1310): Initiated Phase 1b study in Graves’ disease in 2H 2025, with potentially registrational study expected to initiate in 2H 2025.
Phase 1 studies in healthy volunteers with BHV-1400 and BHV-1600 concluding, with BHV-1400 Phase 1 studies expanding to include patients with IgA nephropathy. BHV-1400 potentially registrational study expected to initiate in 2026.
Four additional degrader molecules advancing, including: IgG4 degrader, PLA2R autoantibody degrader, pro-insulin autoantibody degrader, and TSH receptor autoantibody degrader.
Kv7 Activator (BHV-7000):

Pivotal major depressive disorder topline results expected in 2H 2025.
Focal epilepsy study pivotal topline results expected in 1H 2026.
Glutamate Modulator (VYGLXIA):

Priority Review of SCA NDA ongoing, with PDUFA expected in 4Q 2025. Preparing for potential commercial launch in all-genotype SCA if approved by FDA.
Myostatin (Taldefgrobep alfa):

Continue ongoing Health Authority interactions to discuss Spinal Muscular Atrophy ("SMA") registrational path in the U.S. and Europe.
Expect to initiate Phase 2 study in obesity in 2H 2025.
TYK2/JAK1 Inhibitor (BHV-8000):

Continue advancing enrollment in Phase 2/3 study in Parkinson’s disease.
Advance Alzheimer’s disease, multiple sclerosis ("MS") and amyloid-related imaging abnormalities ("ARIA") programs.
Next Generation ADC Platform:

Continue advancing Phase 1/2 study with BHV-1510 as monotherapy and combination therapy with cemiplimab in epithelial tumors in 2025.
Continue advancing Phase 1 study with BHV-1530, FGFR3-directed ADC utilizing proprietary Topolx payload with potential applications in urothelial cancers and other solid tumors.
Advance additional preclinical ADCs, including Merus and GeneQuantum collaborations (undisclosed targets) in 2025.
Capital Position:

Cash, cash equivalents, marketable securities and restricted cash as of June 30, 2025 totaled approximately $408.2 million.

Second Quarter 2024 Financial Highlights:

Research and Development (R&D) Expenses: R&D expenses, including non-cash share-based compensation costs, were $184.4 million for the three months ended June 30, 2025, compared to $314.8 million for the three months ended June 30, 2024. The decrease of $130.5 million was primarily due to a one-time non-cash expense during the three months ended June 30, 2024, paid to Knopp for a milestone and royalty buyback related to the BHV-7000 and broader Kv7 platform. The decrease was partially offset by increased direct program costs for advancing clinical trials and preclinical research programs in 2025, including one-time developmental milestone payments of $15.0 million and $10.0 million for our BHV-8000 and BHV-1530 programs, respectively, as well as increased non-cash share-based compensation expense. Non-cash share-based compensation expense was $13.1 million for the three months ended June 30, 2025, an increase of $6.0 million as compared to the same period in 2024. Non-cash share-based compensation expense was higher in 2025 primarily due to our annual equity incentive awards granted in the first quarter of 2025.

General and Administrative (G&A) Expenses: G&A expenses, including non-cash share-based compensation costs, were $27.3 million for the three months ended June 30, 2025, compared to $19.0 million for the three months ended June 30, 2024. The increase of $8.4 million was primarily due to increased non-cash share-based compensation expense and increased expenses related to fees incurred in connection with the Note Purchase Agreement with Beetlejuice SA LLC, an affiliate of Oberland Capital Management LLC, entered into during the second quarter of 2025 (the Note Purchase Agreement) and other legal costs. Non-cash share-based compensation expense was $7.7 million for the three months ended June 30, 2025, an increase of $2.5 million as compared to the same period in 2024. Non-cash share-based compensation expense was higher in 2025 primarily due to our annual equity incentive awards granted in the first quarter of 2025.

Other Income, Net: Other income, net was $13.8 million for the three months ended June 30, 2025, compared to other income, net of $14.2 million for the three months ended June 30, 2024. The decrease of $0.4 million was primarily due to decreased investment income and an increase in non-cash losses related to changes in fair value of our notes payable liability under the Note Purchase Agreement during the second quarter of 2025, partially offset by an increase in gains recorded for the non-cash changes in the fair value of our forward contract and derivative liability recorded in connection with the amendment to our Membership Interest Purchase Agreement with Knopp Biosciences LLC in May 2024 (the Knopp Amendment).

Net Loss: Biohaven reported a net loss for the three months ended June 30, 2025 of $198.1 million, or $1.94 per share, compared to $319.8 million, or $3.64 per share, for the same period in 2024. Non-GAAP adjusted net loss for the three months ended June 30, 2025 was $166.4 million, or $1.63 per share, compared to $308.6 million, or $3.52 per share, for the same period in 2024. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges and losses from the change in fair value of derivatives. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below.

On August 11, 2025 Theriva Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, reported financial results for the second quarter ended June 30, 2025, and provided a corporate update (Press release, Theriva Biologics, AUG 11, 2025, View Source [SID1234655083]).

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"We are highly encouraged by the progress we’ve made so far in the first half of 2025," said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "We have achieved a significant milestone with the positive readout from the Phase 2b VIRAGE trial, where our lead asset VCN-01 (zabilugene almadenorepvec) achieved its primary survival and safety endpoints in metastatic pancreatic cancer patients treated with first-line standard of care chemotherapy gemcitabine/nab-paclitaxel. We are now preparing a study protocol for a potential Phase 3 clinical trial and advancing VCN-01’s manufacturing scale-up using our propietary suspension cell line. Leveraging our clinical and manufacturing momentum, we have also initiated strategic outreach to identify potential partners for VCN-01’s late-stage clinical development."

Recent Highlights and Anticipated Milestones

VCN-01

Metastatic Pancreatic Ductal Adenocarcinoma (PDAC):

Positive topline results from the VIRAGE Phase 2b trial were announced in May. PDAC patients treated with VCN-01 (zabilugene almadenorepvec) plus gemcitabine/nab-paclitaxel standard-of-care (SoC) chemotherapy had increased overall survival (OS), progression free survival (PFS), and duration of response (DOR) compared to patients treated with gemcitabine/nab-paclitaxel SoC.
Theriva hosted a virtual event featuring eminent pancreatic cancer clinician/researchers to review and discuss the data from the VIRAGE trial of VCN-01. To access the replay of the event, click HERE.
Theriva and VIRAGE trial investigators participated in an off-site meeting during the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2025 Annual Meeting to review the topline data and discuss the conclusions relevant to the design and execution of a potential Phase 3 clinical trial in metastatic pancreatic ductal adenocarcinoma (PDAC).
Expanded data from the VIRAGE trial are to be presented at a mini oral session at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2025 Congress. The presentation entitled "VIRAGE trial: randomized Phase IIb, open-label, study of Nab-Paclitaxel and Gemcitabine with/without intravenous VCN-01 in Patients with Metastatic Pancreatic Cancer (mPDAC)" (abstract 2216MO) is planned to be presented on Monday, October 20, 2025 by Dr. Rocío García-Carbonero, Hospital 12 de Octubre, Madrid, Spain and subsequently reviewed by an invited Discussant.
Retinoblastoma:

As previously announced, safety and clinical outcomes of Phase 1 study (NCT03284268) of VCN-01 (zabilugene almadenorepvec) in refractory retinoblastoma patients were presented during a poster session at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, Illinois on Saturday, May 31, 2025. The abstract can be viewed here.
Second Quarter Ended June 30, 2025 Financial Results

General and administrative expenses increased to $11.2 million for the three months ended June 30, 2025, from $1.5 million for the three months ended June 30, 2024. This increase of 662% is primarily comprised of the increase in fair value of the contingent consideration adjustment of $9.2 million due to the VIRAGE Phase 2b clinical trial of VCN-01 in PDAC achieving its primary endpoint and increased registration fees. The charge related to stock-based compensation expense was $97,000 for the three months ended June 30, 2025, compared to $114,000 for the three months ended June 30, 2024.

Research and development expenses decreased to $2.0 million for the three months ended June 30, 2025, from approximately $3.0 million for the three months ended June 30, 2024. This decrease of 34% is primarily the result of lower clinical trial expenses related to the Company’s VIRAGE Phase 2b clinical trial of VCN-01 in PDAC, lower indirect cost related to decreased VCN-01 manufacturing costs and lower clinical trial expenses related to its Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic HCT recipients, offset by higher patent expenses related to SYN-020. Theriva anticipates research and development expense to increase as it completes its VIRAGE Phase 2b clinical trial of VCN-01 and plans for its potential Phase 3 clinical trial of VCN-01 in PDAC, advances its VCN-01 program in retinoblastoma, expands GMP scale-up manufacturing activities for VCN-01, and continues supporting the Company’s other preclinical and discovery initiatives. The charge related to stock-based compensation expense was $76,000 for the three months ended June 30, 2025, compared to $58,000 related to stock-based compensation expense for the three months ended June 30, 2024.

Other income was $74,000 for the three months ended June 30, 2025 compared to other income of $172,000 for the three months ended June 30, 2024. Other income for the three months ended June 30, 2025 is primarily comprised of interest income of $54,000 and an exchange gain of $20,000. Other income for the three months ended June 30, 2024 is primarily comprised of interest income of $173,000 and exchange loss of $1,000.

Cash and cash equivalents totaled $12.1 million as of June 30, 2025, compared to $11.6 million as of December 31, 2024. The Company expects that its cash position of $9.5 million as of early August 2025, will be able to fund its operations into the first quarter of 2026.

About VCN-01

VCN-01 (zabilugene almadenorepvec) is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to 142 patients to date in clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection). More information on these clinical trials is available at Clinicaltrials.gov.

About Pancreatic Ductal Adenocarcinoma

Cancer of the pancreas consists of two main histological types: cancer that arises from the ductal (exocrine) cells of the pancreas or, much less often, cancers may arise from the endocrine compartment of the pancreas. Pancreatic ductal adenocarcinoma ("PDAC") accounts for more than 90% of all pancreatic tumors. It can be located either in the head of the pancreas or in the body/tail. Pancreatic cancer usually metastasizes to the liver and peritoneum. Other less common metastatic sites are the lungs, brain, kidney and bone. In its early stages, pancreatic cancer does not typically result in any characteristic symptoms, so in most cases it is diagnosed in its late stages (locally advanced non-metastatic or metastatic disease) when surgical resection and possibly curative treatment is not possible. It is generally assumed that only 10% of cases are resectable at presentation, whereas 30-40% of patients are diagnosed at local advanced/unresectable stage and 50-60% present with distant metastases.

About VIRAGE

VIRAGE was a two-arm, Phase 2b open-label, randomized, controlled, multicenter clinical trial in patients with histologically confirmed, newly-diagnosed metastatic PDAC. Patients were enrolled at 5 sites in the U.S. and 9 sites in Spain. In both the control and VCN-01 (zabilugene almadenorepvec) treatment arms, patients received gemcitabine/nab-paclitaxel standard-of-care chemotherapy in repeated 28-day cycles until disease progression. In the VCN-01 treatment arm only, patients were also administered intravenous VCN-01 seven-days prior to starting the first and fourth cycles of gemcitabine/nab-paclitaxel treatment (study days 1 and ~92 respectively). Primary endpoints for the trial include overall survival and VCN-01 safety/tolerability. Additional endpoints include progression free survival, duration of response, and measures of VCN-01 biodistribution, replication, and immune response. More information about the trial is available on Clinicaltrials.gov (NCT05673811), through the Spanish Clinical Trials Registry and European Union Drug Regulating Authorities Clinical Trials Database (EudraCT Number: 2022-000897-24).

About Retinoblastoma

Retinoblastoma is a tumor that originates in the retina and is the most common type of eye cancer in children. It occurs in approximately 1/14,000 – 1/18,000 live newborns and accounts for 15% of the tumors in the pediatric population < 1 year old. The average age of pediatric patients at diagnosis is 2, and it rarely occurs in children older than 6. In Europe, retinoblastoma has an estimated incidence rate of 1 per 13,844 live births (14.1 per million children under the age of 5) with approximately 300 children diagnosed per year (Stacey et al. 2021). Preserving life and preventing the loss of an eye, blindness, and other serious effects of treatment that reduce the patient’s life span or the quality of life remains a challenge. In addition, children with retinoblastoma have been more likely to lose their eye and die of metastatic disease in low-resource countries.

On August 11, 2025 Aptevo Therapeutics Inc. (Nasdaq:APVO), a clinical-stage biotechnology Company focused on developing novel immune-oncology therapeutics based on its proprietary ADAPTIR and ADAPTIR-FLEX platform technologies, reported financial results for the quarter ended June 30, 2025, and provided a business update (Press release, Aptevo Therapeutics, AUG 11, 2025, View Source [SID1234655082]).

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Second Quarter Highlights

Pipeline Expansion – Introduced APVO455, a Nectin-4 x CD3 bispecific for solid tumors, expanding the Company’s suite of differentiated CRIS-7-derived CD3 – engaging molecules in development, including lead candidate, mipletamig

Mipletamig for acute myeloid leukemia (AML) – Achieved 85% remission* rate (11/13 patients) in frontline AML patients across two trials in combination therapy

Increased Liquidity – Completed financings that raised a total of $15.9 million, extending cash runway into late 4Q25

Secured an equity line of credit for up to an additional $25 million in capital

*Remission = complete remission (CR) and, complete remission with blood markers that have not yet recovered (CRi).

"The second quarter marked a period of decisive progress for Aptevo across both clinical and corporate fronts," said Marvin White, President and CEO of Aptevo. We are energized by the expansion of our differentiated CD3 bispecific portfolio – anchored by mipletamig in AML and APVO442 in prostate cancer – with the introduction of APVO455, a Nectin-4 x CD3 bispecific designed to address a broad range of solid tumor types. Broadening our reach into solid tumors, beyond prostate cancer, with APVO455 underscores Aptevo’s commitment to advancing differentiated, next-generation immunotherapies in areas of significant unmet need."

Mr. White continued, "We’re pleased to report that our lead asset, mipletamig, continues to perform as designed in the clinic. In frontline acute myeloid leukemia (AML), 85% of evaluable patients achieved remission when treated in combination with standard of care across two independent trials. These encouraging results validate mipletamig’s differentiated mechanism of action and highlight its potential as a transformative therapy for AML, anchoring our CRIS-7-derived CD3 portfolio. Just as important, we strengthened our financial position with a $15.9 million raise, extending cash runway into late Q4 2025, and ensuring we are well-positioned to reach additional clinical and business milestones in the months ahead."

APVO455: A New T-Cell Engager Targeting Solid Tumors

APVO455 is a preclinical Nectin-4 x CD3 bispecific T-cell engager designed for tumors such as bladder, breast, NSCLC, and head and neck cancers, where nectin-4 is highly expressed. Unlike other approaches that restrict activity to acidic tumor environments or rely on activated T-cells, APVO455 is designed to avoid binding to or triggering T-cell activation in the periphery and do so only in the presence of nectin-4 positive tumor cells. This offers the potential for a broader therapeutic window, more consistent immune activation and the favorable safety and tolerability characteristics of Aptevo’s clinical stage CD3 engager, mipletamig.

CRIS-7-Derived CD3 Portfolio

With this APVO455 addition to the pipeline, Aptevo now has a suite of three CD3-engaging molecules in development. All three molecules share the same CRIS-7-derived CD3 binding domain in the same orientation from within our ADAPTIR/ADAPTIR-FLEX platform that we believe drives the safety results, especially regarding our intention to reduce cytokine release syndrome, as is being delivered in the clinic by mipletamig. In addition, APVO455 and APVO442 contain CD3 binding domains that are optimized for localizing to solid tumors. All three molecules are designed to drive tumor-specific immune activation while limiting harmful side effects to the patient. The suite includes:

Mipletamig, a CD123 x CD3 bispecific for frontline AML, currently in a Phase 1b/2 trial

APVO442, a PSMA x CD3 bispecific targeting prostate cancer, currently in preclinical development

And now APVO455, a Nectin-4 x CD3 bispecific developed to address multiple solid tumor types

Additional Pipeline Candidates

Aptevo continues to develop clinical candidate ALG.APV-527 ( 4-1BB x 5T4) and preclinical candidates APVO711, a checkpoint inhibitor with added functionality and APVO603, a 4-1BB x OX40 molecule. All targeting multiple solid tumor types.

Mipletamig Data Highlights

Results from the RAINIER trial, reported to date demonstrate mipletamig’s increasingly impressive clinical profile, and highlight its potential as a differentiated, highly targeted immunotherapy with a compelling safety profile. As evidence grows, the Company believes that mipletamig is emerging as a strong candidate to reshape frontline AML treatment for unfit patients and potentially advance the standard of care.

Efficacy Data Continues to Outperform Benchmarks

To date, 85% frontline patients across two trials have achieved remission

Of these remissions, multiple patients were considered to have poor prognosis at screening based on the genetic biomarkers of their disease and achieved CR, including one patient who was taken off study and successfully received a stem cell transplant. This is the most favorable outcome because transplant offers the best chance for a cure, although it is a rare occurrence in the unfit AML patient population

Stand Out Safety and Tolerability Outcomes Continue

No CRS has been reported in frontline patients treated to date

Q2 2025 Financial Highlights

Cash Position: Aptevo had cash and cash equivalents as of June 30, 2025, totaling $9.4 million. In Q2 2025, we raised $15.9M in gross proceeds from various equity offerings.

Research and Development Expenses: Research and development expenses decreased by $0.3 million, from $3.6 million for the three months ended June 30, 2024, to $3.3 million for the three months ended June 30, 2025. The decrease was primarily due to lower preclinical and ALG.APV-527 spending due to escalation phase ramping down and was offset by higher mipletamig trial costs from clinical trial patient enrollment.

General and Administrative Expenses: General and administrative expenses increased by $0.5 million, from $2.4 million for the three months ended June 30, 2024, to $2.9 million for the three months ended June 30, 2025. The increase is primarily due to higher consulting costs.

Net Loss: Aptevo had a net loss of $6.2 million or $8.40 per share for the three months ended June 30, 2025, compared to a net loss of $5.9 million or $1,236.96 per share for the corresponding period in 2024.

On August 11, 2025 Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company with a pipeline of first-in-class1 targeted and immune-mediated therapeutics to fight cancer, reported financial results for the quarter ended June 30, 2025 and provided a corporate update (Press release, Tempest Therapeutics, AUG 11, 2025, View Source [SID1234655077]).

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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

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"We are pleased to see the continued progress of our clinical oncology portfolio, including the recent clearance in China to initiate a pivotal trial of amezalpat combination therapy in first-line HCC, expanding on similar clearances we received in the U.S. and Europe from the FDA and EMA," said Stephen Brady, president and chief executive officer of Tempest. "We believe these milestones reflect both the promise of our therapies and the dedication of the team who brought the programs to this point. We remain actively engaged in our strategic alternatives process with the goal of maximizing value for stockholders and patients."

____________________
1 If approved by the U.S. Food and Drug Administration (FDA).

Recent Highlights

Amezalpat (TPST-1120) (clinical PPARα antagonist):
Received clearance to proceed with pivotal trial of amezalpat combination therapy for first-line HCC in China.
Granted orphan drug designation from the EMA for amezalpat for the treatment of patients with HCC.
Reported new data at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, supporting the immune component of amezalpat’s dual mechanism of action and reinforcing its potential as a novel cancer treatment.
Granted both Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA) for amezalpat for the treatment of patients with HCC.
TPST-1495 (clinical dual EP2/4 prostaglandin receptor antagonist):
Granted Orphan Drug designation by the FDA to treat patients with FAP.
Corporate:
Announced cost-cutting measures and plans to explore a full range of strategic alternatives to advance the company’s promising clinical-stage programs and maximize stockholder value.
Strengthened cash position with completion of $4.6 million registered direct offering of common stock in June 2025.
Financial Results

Second Quarter 2025

Tempest ended the quarter with $14.3 million in cash and cash equivalents, compared to $30.3 million on December 31, 2024. The decrease was primarily due to cash used in operating activities, offset by $4.1 million in net proceeds from the June 2025 registered direct offering, as well as $2.8 million in net proceeds from the company’s at-the-market offering program.
Net loss and net loss per share for the quarter were $7.9 million and $2.07, respectively, compared to $9.6 million and $5.52, respectively, for the same period in 2024.
Research and development expenses for the quarter were $3.9 million, compared to $5.8 million for the same period in 2024. The $1.9 million decrease was primarily due to a decrease in costs incurred as a result of re-prioritizing efforts towards exploring strategic alternatives.
General and administrative expenses for the quarter were $4.1 million, compared to $3.7 million for the same period in 2024. The $0.4 million increase was primarily related to one-time separations costs for employees terminated during the period.
Year-to-Date

Cash used in operating activities for the six months ended June 30, 2025 was $16.5 million.
Net loss and net loss per share for the six months ended June 30, 2025 were $18.7 million and $5.17, respectively, compared to $17.5 million and $10.15, respectively, for the same period in 2024.
Research and development expenses for the six months ended June 30, 2025 were $11.5 million, compared to $10.2 million for the same period in 2024. The $1.3 million increase was primarily due to an increase in costs incurred from contract research and manufacturing organizations in preparation for the company’s pivotal Phase 3 trial of amezalpat for the treatment of first-line HCC.
General and administrative expenses for the six months ended June 30, 2025 were $7.4 million, compared to $7.4 million for the same period in 2024, and were primarily related to employee compensation costs, inclusive of one-time separation costs for employees terminated during the quarter ended June 30, 2025, as well as consulting, professional services and facilities costs.