On August 4, 2025 Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, reported its financial results for the second quarter ended June 30, 2025, and provided a business update (Press release, Syndax, AUG 4, 2025, View Source [SID1234654731]).

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"Syndax reported another remarkable quarter. With strong Revuforj and Niktimvo sales momentum along with our expectation for stable expenses over the next few years and more than $500 million in cash, we are well on our way to becoming a profitable company with two blockbuster products," said Michael A. Metzger, Chief Executive Officer. "Notably, Revuforj net revenue increased 43% quarter-over-quarter, even with approximately a third of patients pausing treatment to receive a stem cell transplant. Revuforj is poised for continued growth as we further penetrate the KMT2A population and as patient recontinuations following stem cell transplantation build meaningfully. Importantly, we are nearing another major inflection point with the anticipated approval of Revuforj as the first therapy for R/R mNPM1 AML. Longer-term, our leadership in the menin space positions us to be first to the frontline and meaningfully expand the franchise."

Mr. Metzger continued, "Additionally, Niktimvo is off to a very fast start generating $50 million in net revenue in just the first five months of the launch. It is already profitable to Syndax and we expect increasing profitability margins as revenues continue to grow. The impressive results demonstrate the benefit of delivering a novel approach to treating chronic GVHD and the potential for Niktimvo to transform patient care and make a substantial financial impact for Syndax."

Recent Business Highlights and Anticipated Milestones

Revuforj (revumenib)

Revuforj achieved $28.6 million in net revenue in the second quarter of 2025, representing a 43% increase over the first quarter of 2025. Revuforj was launched in the U.S. in November 2024 for the treatment of relapsed or refractory (R/R) acute leukemia with a KMT2A translocation in adult and pediatric patients one year and older.
Announced that the U.S. FDA granted Priority Review to the Company’s supplemental New Drug Application (sNDA) for Revuforj for the treatment of R/R mutant NPM1 (mNPM1) acute myeloid leukemia (AML). The sNDA is being reviewed under the FDA’s Real-Time Oncology Review (RTOR) program and has been assigned a Prescription Drug User Fee Act (PDUFA) target action date of October 25, 2025.
Presented new Revuforj data in R/R mNPM1 and NUP98-rearranged (NUP98r) AML from the AUGMENT-101 trial at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress Meeting in June 2025.
Additional results from all efficacy-evaluable R/R mNPM1 AML patients in the pivotal Phase 2 portion of the AUGMENT-101 trial of revumenib (n=77) show an overall response rate (ORR)1 of 48% (37/77). The complete remission (CR) plus complete remission with partial hematologic recovery (CRh) rate was 26% (20/77). Median duration of CR/CRh response was 4.7 months. Among patients with CR/CRh assessed for measurable residual disease (MRD), 63% (12/19) were MRD negative. Subpopulation analysis showed that a median overall survival (OS) of 23.3 months (95% CI: 8.4-NR) was observed among the 37 patients who achieved an overall response, based on a Kaplan-Meier estimate from this single-arm trial.
Among patients with R/R NUP98r AML included in the Phase 1 portion of AUGMENT-101, the ORR was 60% (3/5). One patient who proceeded to transplant resumed revumenib post-transplant and was in maintenance cycle 10 as of the data cutoff date. The safety profile of revumenib in patients with R/R NUP98r AML was consistent with previous reports observed in KMT2Ar or mNPM1 AML.
Published pivotal data from patients with R/R mNPM1 AML in the Phase 2 portion of the AUGMENT-101 trial in the journal Blood in May 2025. The results published from the primary efficacy analysis (n=64) are consistent with the data presented at the EHA (Free EHA Whitepaper) Annual Congress Meeting in June 2025 from the larger efficacy-evaluable population (n=77).
Multiple trials evaluating revumenib in mNPM1 and KMT2Ar acute leukemia across the treatment landscape are ongoing. These trials include:
EVOLVE-2: A pivotal, Phase 3, randomized, double-blind, placebo-controlled trial evaluating revumenib in combination with venetoclax and azacitidine in newly diagnosed mNPM1 AML patients who are unfit for intensive chemotherapy. The trial is being conducted in collaboration with the HOVON network, a leading cooperative clinical trial group with extensive experience studying novel therapies for hematologic malignancies.
BEAT AML: A Phase 1 trial evaluating the combination of revumenib with venetoclax and azacitidine in newly diagnosed older adults (≥60 years) with mNPM1 or KMT2Ar AML. The trial is being conducted as part of the Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial. Updated data that showed a 67% (29/43) CR rate and 100% (37/37) flow-MRD negativity rate among evaluable responders were published in the Journal of Clinical Oncology and simultaneously presented in an oral presentation at the EHA (Free EHA Whitepaper) Annual Congress Meeting in June 2025.
SAVE: A Phase 1/2 trial evaluating an all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in pediatric and adult patients with R/R AML or mixed-lineage acute leukemia (MPAL) harboring either mNPM1, KMT2Ar, or NUP98r alterations. The trial is being conducted by investigators from MD Anderson Cancer Center. Updated data that showed an ORR of 82% (27/33) and a CR/CRh rate of 48% (16/33) were presented at the 66th ASH (Free ASH Whitepaper) Annual Meeting. The trial is now enrolling a cohort of newly diagnosed patients.
Intensive chemotherapy: A Phase 1 trial evaluating the combination of revumenib with intensive chemotherapy (7+3) followed by revumenib maintenance treatment in newly diagnosed mNPM1 or KMT2Ar acute leukemia patients. The Company expects to report data in the fourth quarter of 2025 supporting pivotal dose selection.
Break Through Cancer: A Phase 2 trial studying whether the combination of revumenib and venetoclax can eliminate MRD in patients with AML and extend progression-free survival. The trial is being conducted by Break Through Cancer, a collaboration between leading U.S. cancer research centers.
INTERCEPT: A Phase 1 trial evaluating the use of novel therapies, including revumenib, to target MRD and early relapse in AML. The trial is being conducted by the Australasian Leukaemia and Lymphoma Group as part of the INTERCEPT AML master clinical trial. Data that showed 54% (6/11) of patients had MRD reduction at any time, including 36% (4/11) who achieved MRD negativity, were presented at the 66th ASH (Free ASH Whitepaper) Annual Meeting.
Start-up activities are underway for two trials, known as the REVEAL trials, that will evaluate revumenib in combination with standard of care regimens in newly diagnosed acute leukemia patients with mNPM1 or KMT2A-rearranged AML who are fit to receive intensive chemotherapy, with trial initiation expected in the fourth quarter of 2025.
The Company is evaluating revumenib in patients with R/R metastatic microsatellite stable (MSS) colorectal cancer (CRC). The Company expects to report data from the trial by the end of 2025.

Niktimvo (axatilimab-csfr)

Niktimvo achieved $36.2 million in net revenue in the second quarter of 2025, the first full quarter of the U.S. launch, representing significant growth compared to the $13.6 million in net revenue in the first quarter of 2025. Niktimvo was launched in the U.S. in late January 2025 for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs). Syndax and Incyte are co-commercializing Niktimvo. Syndax records 50% of the Niktimvo net commercial profit/loss, defined as net product revenue minus the cost of sales and commercial expenses. For the second quarter of 2025, Syndax’s share of the Niktimvo product contribution, reported as Collaboration revenue, was $9.4 million.
Presented data at the EHA (Free EHA Whitepaper) Annual Congress Meeting in June 2025 highlighting the robust and rapid responses observed in different organs and subgroups of patients with chronic GVHD in the pivotal AGAVE-201 trial that supported the FDA approval of Niktimvo.
Two trials evaluating axatilimab in combination with standard of care therapies in newly diagnosed chronic GVHD patients are ongoing, including:
A Phase 2, open-label, randomized, multicenter trial of axatilimab in combination with ruxolitinib in patients ≥ 12 years of age with newly diagnosed chronic GVHD.
A pivotal Phase 3, randomized, double-blind, placebo-controlled, multi-center trial of axatilimab in combination with corticosteroids in patients ≥ 12 years of age with newly diagnosed chronic GVHD.
Enrollment is ongoing in MAXPIRe, a Phase 2, 26-week randomized, double-blinded, placebo-controlled trial of axatilimab on top of standard of care in patients with idiopathic pulmonary fibrosis (IPF). The Company expects to complete enrollment in the trial in the fourth quarter of 2025 with topline data anticipated in the second half of 2026.
Corporate Updates

In May 2025, the Company announced the appointment of Dr. Nick Botwood as Head of Research and Development and Chief Medical Officer. Dr. Botwood brings 25 years of industry experience leading drug development, R&D strategy, and global commercialization of novel oncology therapeutics.
In June 2025, the Company announced the resignation of William Meury from its Board of Directors after seven years of service due to his acceptance of the role of President and Chief Executive Officer and a member of the Board of Directors at Incyte, the Company’s collaboration partner for Niktimvo.
Second Quarter 2025 Financial Results

As of June 30, 2025, Syndax had cash, cash equivalents, and short and long-term investments of $517.9 million and 86.3 million common shares and prefunded warrants outstanding.

Total revenue for the second quarter of 2025 was $38.0 million, which consisted of $28.6 million in Revuforj net product revenue and $9.4 million in Niktimvo collaboration revenue. The collaboration revenue is derived from the $36.2 million in Niktimvo net revenue that was previously reported by the Company’s partner Incyte. Syndax records 50% of the Niktimvo net commercial profit/loss, defined as net product revenue (recorded by Incyte) minus the cost of sales and commercial expenses.

Second quarter 2025 research and development expenses increased to $62.2 million from $48.7 million for the comparable prior year period. The increase was due to an increase in revumenib-related costs primarily driven by multiple trials evaluating revumenib in mNPM1 and KMT2Ar acute leukemia across the treatment landscape, milestone paid to AbbVie upon submission of the sNDA, and medical affairs activities supporting Revuforj as well as increased personnel costs to help support commercialization.

Second quarter 2025 selling, general and administrative expenses increased to $43.8 million from $29.1 million for the comparable prior year period. The increase was primarily due to increased employee-related expenses and professional fees to support increased sales and marketing-related expenses related to the U.S. commercial launch of Revuforj.

For the three months ended June 30, 2025, Syndax reported a net loss attributable to common stockholders of $71.8 million, or $0.83 per share, compared to a net loss attributable to common stockholders of $68.1 million, or $0.80 per share, for the comparable prior year period.

Financial Guidance

For the third quarter of 2025, the Company expects total research and development plus selling, general and administrative expenses to be $95 to $100 million, excluding non-cash stock compensation expense. For the full year of 2025, the Company expects total research and development plus selling, general and administrative expenses to be $370 to $390 million, excluding non-cash stock compensation expense. The full year 2025 guidance represents no change to prior guidance, which was $415 to $435 including $45 million in estimated non-cash stock compensation expense. The Company is not providing revenue guidance at this time.

Syndax expects that its operating expense base will remain stable over the next few years. As a result, Syndax expects that its cash, cash equivalents and short- and long-term investments, combined with its anticipated product revenue and interest income, will enable the company to reach profitability.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Monday, August 4, 2025.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website. Alternatively, the conference call may be accessed through the following:

Conference ID: Syndax2Q25
Domestic Dial-in Number: 800-590-8290
International Dial-in Number: 240-690-8800
Live webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company’s website at www.syndax.com approximately 24 hours after the conference call and will be available for 90 days following the call.

About Revuforj (revumenib)

Revuforj (revumenib) is an oral, first-in-class menin inhibitor that is FDA approved for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients one year and older.

The FDA has granted Priority Review to Syndax’s supplemental New Drug Application (sNDA) seeking the approval of revumenib for the treatment of R/R mNPM1 AML. The sNDA is being reviewed under the FDA’s Real-Time Oncology Review (RTOR) program, which allows for a more efficient review and close engagement between the sponsor and FDA. The sNDA is supported by positive pivotal data from the AUGMENT-101 trial of revumenib in patients with R/R mNPM1 AML. Results from this population were published in the journal Blood and presented at the 2025 European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress Meeting.

Additionally, multiple trials of revumenib are ongoing or planned across the treatment landscape, including in combination with standard of care therapies in newly diagnosed patients with mNPM1 or KMT2Ar AML.

Revumenib was previously granted Orphan Drug Designation for the treatment of AML, ALL and acute leukemias of ambiguous lineage (ALAL) by the U.S. FDA and for the treatment of AML by the European Commission. The U.S. FDA also granted Fast Track designation to revumenib for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation and Breakthrough Therapy Designation for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement.

About Niktimvo (axatilimab-csfr)

Niktimvo (axatilimab-csfr) is a first-in-class colony stimulating factor-1 receptor (CSF-1R)-blocking antibody approved for use in the U.S. for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs).

In 2016, Syndax licensed exclusive worldwide rights to develop and commercialize axatilimab from UCB. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab in chronic GVHD and any future indications.

Axatilimab is being studied in frontline combination trials in chronic GVHD, including a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids (NCT06585774). Axatilimab is also being studied in an ongoing Phase 2 trial in patients with idiopathic pulmonary fibrosis (NCT06132256).

About the Real-Time Oncology Review Program (RTOR)

RTOR provides a more efficient review process for oncology drugs to ensure that safe and effective treatments are available to patients as early as possible, while improving review quality and engaging in early iterative communication with the applicant. Specifically, it allows for close engagement between the sponsor and the FDA throughout the submission process and it enables the FDA to review individual sections of modules of a drug application rather than requiring the submission of complete modules or a complete application prior to initiating review. Additional information about RTOR can be found at: View Source

On August 4, 2025 Mural Oncology plc (Nasdaq: MURA), reported its financial results for the second quarter of 2025 and provided a business update (Press release, Mural Oncology, AUG 4, 2025, View Source [SID1234654729]). On April 15, 2025, Mural announced that it was discontinuing all clinical development of its lead product candidate, nemvaleukin alfa, and was commencing the exploration of strategic alternatives focused on maximizing shareholder value. The Company is continuing to explore strategic alternatives.

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Following its announcement on April 15, 2025, Mural has taken steps to conserve its remaining cash, including the implementation of a reduction in workforce by approximately 90%, the discontinuation of the clinical development of nemvaleukin alfa, and the termination of its other research and development activities, including the research and development of its IL-18 and IL-12 programs. As of June 30, 2025, the company had approximately $77.1 million in cash and cash equivalents. The company estimates that, if it has not consummated a transaction or other strategic alternative by December 31, 2025, its cash and cash equivalents as of such date will total approximately $43 to $48 million. This estimate reflects anticipated costs to be incurred in connection with finalization of the discontinuation of the company’s remaining activities and expected residual operating expenses (e.g. lease expense, salary and benefits for remaining employees, insurance costs). This cash guidance is subject to a number of assumptions and actual cash balances may differ materially, particularly if the Company consummates a transaction or other strategic alternative prior to December 31, 2025. Mural has not included in its cash guidance, additional costs that may be incurred as a result of, or in connection with, any strategic alternative it may pursue, including, but not limited to, an offer for or other acquisition of the company, merger, business combination or other transaction, including a possible wind-down and liquidation of the company (e.g., legal and financial advisor fees and severance costs for remaining employees).

Further updates and developments will be disclosed as appropriate or where necessary under regulatory requirements. There can be no assurance that the company’s exploration of strategic alternatives will result in the company pursuing a transaction or that any acquisition or other transaction involving the company will be completed, nor as to the terms on which any acquisition or other transaction will occur, if at all.

Financial Results for the Quarter Ended June 30, 2025

Cash Position: As of June 30, 2025, cash and cash equivalents were $77.1 million.

R&D Expenses: Research and development expenses were $23.3 million for the second quarter of 2025 compared to $27.5 million for the second quarter of 2024. This decrease was primarily due to decreased employee-related expenses following the reduction-in-force implemented by the company during the second quarter of 2025 and the decreased spend on the ARTISTRY-7 clinical trial of nemvaleukin due to the termination of the development of nemvaleukin. These decreases were partially offset by an increase in spend on early discovery programs upon completion of certain manufacturing activities and on the ARTISTRY-6 trial of nemvaleukin related to the top-line read-out and wind-down of the trial during the second quarter of 2025.

G&A Expenses: General and administrative expenses were $8.1 million for the second quarter of 2025 compared to $6.7 million for the second quarter of 2024. This increase in G&A expenses was primarily due to increased employee-related expenses associated with employee termination and retention benefits and increased legal expenses associated with corporate activities, partially offset by decreased share-based compensation expense resulting from an increase in the awards that were forfeited or were expected to be forfeited following the reduction-in-force.

Restructuring and Impairment Expenses: Mural incurred $17.5 million in restructuring and impairment charges during the second quarter of 2025, consisting of severance and termination benefits related to the reduction-in-force, an impairment charge related to lab equipment sold during the second quarter of 2025 and contract termination and write-offs related to the termination of the company’s research and development programs.

Net Loss: Net loss was $48.0 million for the second quarter of 2025 compared to $31.6 million for the second quarter of 2024. The increase in net loss was primarily driven by the restructuring and impairment charges incurred during the second quarter of 2025.

Cash Guidance: Mural estimates that its cash and cash equivalents will be approximately $43.0 million – $48.0 million as of December 31, 2025, if it has not consummated a transaction or other strategic alternative by such date.

On August 4, 2025 XOMA Royalty Corporation ("XOMA Royalty") (NASDAQ: XOMA) and LAVA Therapeutics N.V. ("LAVA") (NASDAQ: LVTX) reported they have entered a definitive share purchase agreement (the "Purchase Agreement" and the transactions set forth in the Purchase Agreement, the "Transactions") whereby XOMA Royalty will acquire LAVA for (i) USD between $1.16 and $1.24 per share in cash, consisting of (A) USD $1.16 (the "Base Price Per Share") in cash per share (the "LAVA common stock"), plus (B) an additional amount of cash of up to $0.08 per Share (such amount as finally determined in accordance with the Purchase Agreement, the "Additional Price Per Share," and together with the Base Price Per Share, the "Cash Amount"), plus (ii) a non-transferable contingent value right ("CVR") per share representing the right to receive 75% of the net proceeds related to LAVA’s two partnered assets and 75% of any net proceeds from any out license or sale of LAVA’s unpartnered programs (Press release, Lava Therapeutics, AUG 4, 2025, View Source [SID1234654728]).

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"We believe the structure of this transaction has the potential to benefit both LAVA and XOMA Royalty shareholders over time," stated Owen Hughes, Chief Executive Officer of XOMA Royalty. "We are adding economics related to LAVA’s partnered programs investigating the utility of gamma delta bispecific antibodies, which hold significant promise for patients."

"The Purchase Agreement with XOMA Royalty announced today is the result of a thorough and wide-ranging strategic review process, conducted with the support of our legal and financial advisors, aimed at maximizing shareholder value while participating in the sustained success of LAVA’s business," said Steve Hurly, Chief Executive Officer of LAVA.

In accordance with its fiduciary duties under Dutch law, LAVA’s Board of Directors (the "Board") has unanimously determined that the Transactions are in the best interests of LAVA and the sustainable success of its business, having carefully considered the interests of LAVA shareholders, employees, and all other relevant stakeholders and has approved the Purchase Agreement. The Board unanimously recommends that shareholders support the Offer, accept the Offer and vote in favor of the resolutions to be proposed to LAVA’s shareholders’ meeting, as noted below.

Transaction Terms

Pursuant and subject to the terms of the Purchase Agreement, XOMA Royalty will commence a tender offer (the "Offer") by August 15, 2025, to acquire all outstanding shares of LAVA common stock. The closing of the Offer is subject to certain conditions, including the tender of LAVA common stock representing at least 80% (or, in certain cases, 75%) of LAVA’s issued and outstanding shares, the condition that certain resolutions are adopted by LAVA’s shareholders meeting; a minimum cash balance at closing, and other customary closing conditions. Following a subsequent offering period, LAVA will undergo a corporate reorganization designed to result in XOMA Royalty acquiring 100% of the shares in LAVA’s successor and all then-remaining LAVA shareholders (other than XOMA Royalty) receiving the same cash and CVR consideration per share as is provided in the tender offer, subject to applicable withholding taxes. LAVA will hold a shareholder’s meeting in connection with the Transactions. The closing of the Transactions is expected in the fourth quarter of 2025.

In connection with the Transactions, the Company plans to discontinue its Phase 1 clinical trial of LAVA-1266 for acute myeloid leukemia and myelodysplastic syndrome and initiate the wind-down of the LAVA-1266 program.

Advisors

XOMA Royalty was represented by Gibson, Dunn & Crutcher LLP and Loyens & Loeff N.V, who acted as U.S. and Dutch legal advisors, respectively. Leerink Partners is acting as exclusive financial advisor to LAVA, Cooley LLP is acting as U.S. legal advisor to LAVA and NautaDutilh N.V. is acting as Dutch legal advisor to LAVA.

On August 4, 2025 Intensity Therapeutics, Inc. (Nasdaq: INTS) ("Intensity" or "the Company"), a late-stage clinical biotechnology company focused on the discovery and development of novel intratumoral cancer therapies that are designed to kill tumors and increase immune system recognition of cancers using its proprietary non-covalent conjugation technology, reported that in July 2025 the Company added $6.6 million in gross proceeds ($6.3 million net) by selling 19,868,658 shares of its common stock via its At-the-Market offering (the "ATM") at an average price of $0.3323 per share (Press release, Intensity Therapeutics, AUG 4, 2025, View Source [SID1234654727]). Following such sales of common stock pursuant to the ATM, the Company has 46,035,081 shares of common stock issued and outstanding as of July 31, 2025.

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"We were able to take advantage of strong liquidity and favorable prices in our stock last month. The proceeds from these ATM sales strengthen our balance sheet considerably and allow us to continue to advance the clinical trials into the second half of 2026," said Lewis H. Bender, President and CEO of Intensity. "We are also pleased to announce that the average price per share for these ATM sales was more than 10% higher than our recently completed June 2025 public offering, and the costs to raise this incremental capital were much lower. We will continue to be selective and strategic in the deployment of the remainder of the ATM."

About At the Market Transactions

"At-the-Market" (ATM) offerings, also known as "ATM" programs, refer to a method where a public company sells its newly issued shares directly into the existing trading market at the prevailing market price, rather than through a traditional underwritten offering. This approach allows companies to raise capital opportunistically and incrementally, as needed, with minimal disruption to the market and typically lower costs.

About INT230-6

INT230-6, Intensity’s lead proprietary investigational product candidate, is designed for direct intratumoral injection. INT230-6 was discovered using Intensity’s proprietary DfuseRx℠ technology platform. The drug consists of two proven, potent anti-cancer agents, cisplatin and vinblastine sulfate, and a diffusion and cell penetration enhancer molecule ("SHAO") that facilitates the dispersion of potent cytotoxic drugs throughout tumors, allowing the active agents to diffuse into cancer cells. These agents remain in the tumor, resulting in a favorable safety profile. In addition to local disease control and direct tumor killing, INT230-6 causes a release of a bolus of neoantigens specific to the malignancy, leading to immune system engagement and systemic anti-tumor effects. Importantly, these effects are mediated without immunosuppression, which often occurs with systemic chemotherapy.

On August 4, 2025 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the second quarter ended June 30, 2025 (Press release, CRISPR Therapeutics, AUG 4, 2025, View Source [SID1234654716]).

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"We are entering the second half of the year with momentum across both our commercial and clinical programs," said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. "The activation of 75 authorized treatment centers for CASGEVY has been achieved, marking a meaningful step in expanding patient access, while clinical trials across multiple other programs continue to advance. Looking ahead, we expect several key milestones including the presentation of complete Phase 1 data for CTX310, as well as updates across our oncology and autoimmune portfolios. Our focus remains on delivering transformative therapies for patients with critical unmet needs."

Recent Highlights and Outlook

Hemoglobinopathies and CASGEVY (exagamglogene autotemcel [exa-cel])

CASGEVY is a non-viral, ex vivo, CRISPR/Cas9 gene-edited cell therapy for eligible patients with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT), designed to eliminate both vaso-occlusive crises (VOCs) and transfusion requirements. CASGEVY is approved in the U.S., Great Britain, the EU, the Kingdom of Saudi Arabia (KSA), the Kingdom of Bahrain (Bahrain), Qatar, Canada, Switzerland and the United Arab Emirates (UAE) for the treatment of both SCD and TDT. Building on the foundational launch in 2024, significant progress is being made to bring this transformative therapy to patients worldwide.
The target of activating 75 authorized treatment centers (ATCs) globally has been achieved, marking an important milestone in the commercial rollout of CASGEVY. Since launch through June 30, approximately 115 patients have completed their first cell collection, and 29 patients have received infusions of CASGEVY, including 16 infused in the second quarter. The launch of CASGEVY is building strong momentum, positioning the program for significant growth and broader impact.
Through reimbursement agreements, Vertex has secured access for eligible SCD and TDT patients in 10 countries. Recent agreements include Northern Ireland, Scotland and Denmark. Efforts are ongoing with government and reimbursement authorities globally to secure access for eligible patients.
CRISPR Therapeutics continues to advance its next-generation approaches designed to significantly broaden the addressable patient population for SCD and TDT. The Company’s internally developed targeted conditioning program, an anti-CD117 (c-Kit) antibody-drug conjugate (ADC), remains on track in preclinical development. In parallel, the Company is making continued progress in its in vivo editing platform aimed at enabling direct editing of hematopoietic stem cells (HSC) without the need for conditioning. By potentially eliminating the need for conditioning, this approach could unlock access to transformative therapies for a significantly larger patient population.
Immuno-Oncology and Autoimmune Disease Programs

Clinical trials are ongoing for the Company’s next-generation allogeneic CAR T product candidates, CTX112 and CTX131, targeting CD19 and CD70, respectively, across multiple indications. Both candidates incorporate novel potency edits designed to significantly enhance CAR T cell expansion and cytotoxicity, positioning them as potential best-in-class therapies.
CTX112, targeting CD19, is in development for hematologic malignancies and autoimmune diseases. Preliminary clinical data support a differentiated profile with strong clinical benefit combined with the convenience of an "off-the-shelf" therapy.
In relapsed or refractory B-cell malignancies, encouraging results from the ongoing Phase 1/2 clinical trial led to the FDA granting Regenerative Medicine Advanced Therapy (RMAT) designation for CTX112 in relapsed or refractory follicular lymphoma and marginal zone lymphoma.
A Phase 1 trial of CTX112 is ongoing in autoimmune indications, including systemic lupus erythematosus (SLE), systemic sclerosis and inflammatory myositis. Preliminary safety, pharmacokinetic, and pharmacodynamic data from oncology trials support its potential in autoimmune indications.
The Company plans to provide a broad update for CTX112 in oncology and autoimmune disease in the second half of 2025.
CTX131, targeting CD70, is in development for both solid tumors and hematologic malignancies. Clinical trials for CTX131 are ongoing, with an update expected in 2025.
CRISPR Therapeutics’ immuno-oncology and autoimmune disease efforts are supported by a wholly-owned, U.S. manufacturing facility located in Framingham, MA. This investment enables the production of clinical and commercial-stage good manufacturing practice (GMP) materials across the Company’s allogeneic cell therapy programs.
In Vivo Liver Editing Programs

CRISPR Therapeutics is advancing a pipeline of in vivo gene editing candidates targeting major unmet needs in cardiovascular and metabolic diseases using its proprietary lipid nanoparticle (LNP) delivery platform.
CTX310 is in an ongoing Phase 1 clinical trial targeting ANGPTL3 in patients with homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (SHTG), heterozygous familial hypercholesterolemia (HeFH), or mixed dyslipidemias. ANGPTL3 loss-of-function mutations are linked to reduced in low-density lipoprotein cholesterol (LDL-C), triglycerides (TG), and a lower risk of atherosclerotic cardiovascular disease (ASCVD), without adverse effects on overall health. In the U.S., more than 40 million patients have elevated LDL, severely elevated TGs or both. CTX310 is initially focused on a high-risk subset of this group with the greatest unmet medical need and limited effective treatment options.
In June, the Company reported data for CTX310, demonstrating dose-dependent reductions in ANGPTL3, TG, and LDL following a single administration. As dose-range finding continues, data to date demonstrate peak reductions of up to 82% in TG and LDL reductions of up to 86% at DL4 without any clinically significant changes in liver enzymes and a safety and tolerability profile consistent with previous findings.
These initial results represent a significant milestone in the advancement of CRISPR Therapeutics’ proprietary LNP delivery technologies for gene editing in the liver. The Company anticipates presenting the complete Phase 1 data for CTX310 at a medical meeting in the second half of 2025.
CTX320 is in an ongoing Phase 1 clinical trial targeting the LPA gene in patients with elevated lipoprotein(a) [Lp(a)], a genetically determined risk factor associated with an increased incidence of major adverse cardiovascular events (MACE). Elevated Lp(a) levels affect up to 20% of the global population and remain unaddressed by current therapies. The Company plans to provide an update in the first half of 2026.
CRISPR Therapeutics continues to advance two preclinical programs: CTX340, targeting angiotensinogen (AGT) for the treatment of refractory hypertension, and CTX450, targeting 5’ aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias (AHP). Both candidates are currently in IND/CTA-enabling studies.
SRSD107

In May, the Company entered a strategic collaboration with Sirius Therapeutics to jointly develop and commercialize small interfering RNA (siRNA) therapies, beginning with SRSD107, a long-acting Factor XI (FXI) siRNA. Under the partnership, development and commercialization will be shared, with CRISPR Therapeutics leading efforts in the U.S. and Sirius in Greater China. The agreement also grants CRISPR Therapeutics the option to nominate two additional siRNA targets for future development. This collaboration expands CRISPR Therapeutics’ capabilities, enabling the development of a broader range of transformative gene-based medicines beyond its current gene-editing programs in the clinic.
In July, the European Medicines Agency (EMA) authorized the initiation of a Phase 2 clinical trial of SRSD107 for thromboembolic disorders. The study is designed to evaluate the safety and efficacy of SRSD107 in preventing post-operative venous thromboembolism in patients undergoing total knee arthroplasty and aims to confirm its anticoagulant potential.
Regenerative Medicine Programs

CRISPR Therapeutics continues to advance its regenerative medicine efforts in Type 1 diabetes (T1D). In addition to CTX211, the Company is developing next-generation programs focusing on induced pluripotent stem cell (iPSC) derived, allogeneic, gene-edited, beta islet cell precursors. These approaches aim to enable insulin independence in T1D patients without the need for chronic immunosuppression. The Company expects to provide an update in 2025.
Second Quarter 2025 Financial Results

Cash Position: Cash, cash equivalents, and marketable securities were $1,721.2 million as of June 30, 2025, compared to $1,903.8 million as of December 31, 2024. The decrease in cash was primarily driven by operating expenses, as well as the $25.0 million upfront cash payment made as part of the Sirius Agreement, offset by proceeds from interest income and proceeds from the issuance of common shares and option exercise activity.
R&D Expenses: R&D expenses were $69.9 million for the second quarter of 2025, compared to $80.2 million for the second quarter of 2024. The decrease in R&D expense was primarily driven by a decrease in employee-related expenses, including stock-based compensation expenses.
Acquired In-Process R&D Expenses: Acquired in-process R&D expenses were $96.3 million for the second quarter of 2025 related to costs incurred upon entering the Sirius Agreement during the second quarter of 2025.
G&A Expenses: General and administrative expenses were $18.9 million for the second quarter of 2025, compared to $19.5 million for the second quarter of 2024.
Collaboration Expense: Collaboration expense, net, was $45.2 million for the second quarter of 2025, compared to $52.1 million for the second quarter of 2024. The decrease in collaboration expense, net, was primarily attributable to an increase in CASGEVY revenue, as well as a decrease in operating expenses for the program.
Net Loss: Net loss was $208.5 million for the second quarter of 2025, compared to a net loss of $126.4 million for the second quarter of 2024.
About CASGEVY (exagamglogene autotemcel [exa-cel])
CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate recurrent vaso-occlusive crises (VOCs) for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for certain indications in multiple jurisdictions for eligible patients.

About the CRISPR Therapeutics – Vertex Collaboration for CASGEVY
CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CASGEVY represents the first potential treatment to emerge from the joint research program. Under an amended collaboration agreement, Vertex now leads global development, manufacturing, and commercialization of CASGEVY and splits program costs and profits worldwide 60/40 with CRISPR Therapeutics. Vertex is the manufacturer and exclusive license holder of CASGEVY.

About CTX112
CTX112 is being developed for both oncology and autoimmune indications. CTX112 is a next-generation, wholly-owned, allogeneic CAR T product candidate targeting Cluster of Differentiation 19, or CD19, which incorporates edits designed to evade the immune system, enhance CAR T potency, and reduce CAR T exhaustion. CTX112 is being investigated in an ongoing clinical trial designed to assess safety and efficacy of the product candidate in adult patients with relapsed or refractory B-cell malignancies who have received at least two prior lines of therapy. In addition, CTX112 is being investigated in an ongoing clinical trial designed to assess the safety and efficacy of the product candidate in adult patients with systemic lupus erythematosus, systemic sclerosis, and inflammatory myositis.

About CTX131
CTX131 is being developed for both solid tumors and hematologic malignancies, including T cell lymphomas (TCL). CTX131 is a next-generation, wholly-owned, allogeneic CAR T product candidate targeting Cluster of Differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX131 incorporates edits designed to evade the immune system, prevent fratricide, enhance CAR T potency, and reduce CAR T exhaustion. CTX131 is being investigated in ongoing clinical trials designed to assess the safety and efficacy of the product candidate in adult patients with relapsed or refractory solid tumors and hematologic malignancies, including TCL.

About In Vivo Programs
CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) platform for the delivery of CRISPR/Cas9 to the liver. The Company’s in vivo portfolio includes its lead investigational programs, CTX310 (directed towards angiopoietin-related protein 3 (ANGPTL3)) and CTX320 (directed towards LPA, the gene encoding apolipoprotein(a) (apo(a)), a major component of lipoprotein(a) [Lp(a)]). Both are validated therapeutic targets for cardiovascular disease. CTX310 and CTX320 are in ongoing clinical trials in patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia, and in patients with elevated lipoprotein(a), respectively. In addition, the Company’s research and preclinical development candidates include CTX340 and CTX450, targeting angiotensinogen (AGT) for refractory hypertension and 5’-aminolevulinate synthase 1 (ALAS1) for acute hepatic porphyria (AHP), respectively.

About SRSD107
SRSD107 is a novel double-stranded small interfering ribonucleic acid (siRNA). Developed initially by Sirius Therapeutics, SRSD107 specifically targets the human coagulation factor XI (FXI) mRNA and inhibits FXI protein expression, thereby blocking the intrinsic coagulation pathway and promoting anticoagulant/anti-thrombotic effects.