Genprex Signs Exclusive License to Additional Gene Therapy Technology with UTHealth Houston for the Treatment of Glioblastoma

On May 6, 2025 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported it has entered into an exclusive patent license agreement with UTHealth Houston granting Genprex exclusivity and commercial rights relating to its lead drug candidate, Reqorsa Gene Therapy (quaratusugene ozeplasmid) for the potential treatment of glioblastoma (Press release, Genprex, MAY 6, 2025, View Source [SID1234652574]). The subject patent is co-owned by Genprex and UTHealth Houston, and the license provides Genprex with patent exclusivity.

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"We are pleased to expand our portfolio of licensed patents and add a technology that uses REQORSA to treat glioblastoma," said Thomas Gallagher, Senior Vice President of Intellectual Property and Licensing. "With this license agreement, Genprex has obtained exclusive commercial rights to REQORSA in glioblastoma while also adding to our patent estate. The role of TUSC2 in lung cancer has been well established, and this latest license enables Genprex to expand the use of REQORSA into a new indication, in which the cancer can be difficult to treat and there are unmet medical needs."

REQORSA may be a potential therapeutic treatment for glioblastoma.

REQORSA may be a potential therapeutic treatment for glioblastoma. Genprex previously reported positive preclinical data on the efficacy of REQORSA in glioblastoma in October 2024 at the 2024 EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics. Research collaborators from UTHealth Houston previously reported TUSC2 as a novel tumor suppressor for glioblastoma, the most common and deadliest primary brain tumor in adults which is associated with a poor prognosis. In their latest study, UTHealth Houston researchers used patient-derived glioblastoma (GBM) cell lines and patient-derived glioma stem cell (PD-GSC) lines. REQORSA was used to restore TUSC2 expression.

Researchers at UTHealth Houston observed that REQORSA significantly reduced GBM cell viability, and the results of a migration assay demonstrated that REQORSA suppressed GBM cell migration independent of its ability to suppress cell viability. In conclusion, REQORSA demonstrates promising in vitro efficacy in GBM and PD-GSCs, and these results support further evaluation of its in vivo anti-tumor efficacy in malignant gliomas using mouse models.

About Reqorsa Gene Therapy

REQORSA (quaratusugene ozeplasmid) consists of a plasmid containing the TUSC2 gene encapsulated in non-viral lipid-based nanoparticles in a lipoplex form (the Company’s Oncoprex Delivery System), which has a positive charge. REQORSA is injected intravenously and specifically targets cancer cells. REQORSA is designed to deliver the functioning TUSC2 gene to negatively charged cancer cells while minimizing uptake by normal tissue. Laboratory studies conducted at The University of Texas MD Anderson Cancer Center show that the uptake of TUSC2 in tumor cells in vitro after REQORSA treatment was 10 to 33 times the uptake in normal cells.

Galmed Announces First Time Results in Oncology Studies: Aramchol Significantly Enhances Bayer’s Regorafenib Effect in GI Cancer Models

On May 6, 2025 Galmed Pharmaceuticals Ltd. (NASDAQ: GLMD) ("Galmed" or the "Company"), a clinical-stage biopharmaceutical company for cardiometabolic diseases and GI oncological therapeutics, reported that the first set of oncology studies has shown that Aramchol enhances the liver / colorectal cancer-approved therapeutic Regorafenib effects in liver and colon cancers in-vitro and in-vivo models (Press release, Galmed Pharmaceuticals, MAY 6, 2025, View Source [SID1234652573]).

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Aramchol interacted with the multi-kinase inhibitors Sorafenib, Regorafenib and Lenvatinib, to kill GI tumor cells, with Regorafenib exhibiting the greatest effect. Aramchol enhanced both flux and autolysosome formation caused by Regorafenib, activating ATM and AMPK and inactivating mTORC1 and mTORC2 pathways. In addition, Regorafenib and Aramchol interacted to suppress tumor growth in hepatoma models without normal tissue toxicities.

Paul Dent, Ph.D. Professor School of Medicine Biochemistry and Molecular Biology Virginia Commonwealth University commented: "The key molecular mechanisms by which Aramchol and Regorafenib killed GI tumor cells were defined in the study. Aramchol acts to enhance autophagy through mechanisms that are different to those of Regorafenib. The interaction between Aramchol and Regorafenib, causing more autophagic flux and autolysosome formation, is required for the enhanced killing of tumor cells by the drug combination."

Allen Baharaff, President and CEO of Galmed Pharmaceuticals commented: "Targeting lipid metabolism with Aramchol, a potent SCD1 inhibitor, is a promising emerging strategy to overcome TKIs, such as Sorafenib, Regorafenib or Lenvatinib, for therapy resistance in HCC and colorectal cancers. A combination of Bayer’s Regorafenib and Aramchol could potentially become a cost-effective first line treatment for HCC and other liver and colorectal cancers. The mechanisms by which the combination of Aramchol and Regorafenib kills tumor cells provide the scientific foundation for a Phase Ib clinical trial in GI tumors. We look forward to taking these findings forward in a new clinical program, alongside the recently announced Semaglutide GLP-1 sublingual development."

Day One Reports First Quarter 2025 Financial Results and Corporate Progress

On May 6, 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 first quarter 2025 financial results and highlighted recent corporate achievements (Press release, Day One, MAY 6, 2025, View Source [SID1234652572]).

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"We continued our successful launch of OJEMDA in 2025 and are energized by the growing impact we are bringing to patients every day," said Jeremy Bender, Ph.D., chief executive officer of Day One. "This is an exciting time of growth for Day One. We remain focused on creating value at every level of the organization, and we are executing on our objectives with urgency and fiscal discipline."

Program Highlights


OJEMDA net product revenue was $30.5 million in the first quarter of 2025.
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U.S. OJEMDA net product revenue increased 11% from the fourth quarter of 2024.
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OJEMDA prescriptions increased to over 900 (+16%) in the first quarter of 2025.


Achieved $87.7 million in OJEMDA net product revenue since launch, representing over 2,500 OJEMDA prescriptions.


In April 2025, Ipsen announced the regulatory filing for tovorafenib was accepted by the European Medicines Agency for review in the European Union, marking an important step forward for tovorafenib’s development outside of the U.S.


DAY301, our PTK7-targeted ADC, cleared the first dose cohort (a single-patient accelerated titration cohort) in the Phase 1a portion of the Phase 1a/b clinical trial.


Day One’s pivotal Phase 3 FIREFLY-2 clinical trial is expected to be fully enrolled in the first half of 2026.


Day One presented two posters at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in Chicago, April 25-30, 2025.

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A preclinical study provided evidence for the efficacy of sequencing of MEK inhibitor treatment after tovorafenib by demonstrating mice bearing AGK::BRAF fusion tumors maintained significant tumor regression with no emergent tumors when treated with tovorafenib (25 mg/kg) once daily followed by MEK inhibitor treatment. Additionally, long-term efficacy was maintained with continuous dosing of tovorafenib in vivo; significant tumor regression was maintained with no emergent tumors indicating no evidence of emerging resistance. The study results demonstrate that anti-tumor activity with MEK inhibitors is observed after treatment with tovorafenib and suggest that acquired resistance may be less common in BRAF fusion-driven tumors treated with tovorafenib. (Abstract 1727: Sequencing MEK inhibitor therapy after tovorafenib in BRAF fusion-driven cancers: Preclinical evidence of sustained tumor regression)
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A clinical trial-in-progress poster on DAY301-001 the first-in-human Phase 1a/1b trial with DAY301 to evaluate its safety, tolerability, pharmacokinetics (PK) and antitumor activity in patients with locally advanced or metastatic solid tumors. (Abstract CT196: Phase I dose escalation and expansion study of the PTK-7 targeted antibody-drug conjugate DAY301 in patients with locally advanced or metastatic solid tumors)

First Quarter 2025 Financial Highlights


Product Revenue, Net: OJEMDA net product revenue was $30.5 million for the first quarter of 2025.


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


R&D Expenses: Research and development expenses were $39.6 million for the first quarter of 2025 compared to $40.2 million for the first quarter of 2024. The difference was primarily due to a decrease in license agreement payments and employee compensation costs.


SG&A Expenses: Selling, general and administrative expenses were $29.3 million for the first quarter of 2025 compared to $26.6 million for the first quarter of 2024. The increase was primarily due to employee compensation costs and professional service expenses to support the launch of OJEMDA.


Net Loss: Net loss totaled $36.0 million for the first quarter of 2025 with non-cash stock-based compensation expense of $12.9 million, compared to a net loss of $62.4 million for the first quarter of 2024, with non-cash stock-based compensation expense of $12.6 million.


Cash Position: The Company’s cash, cash equivalents and short-term investments totaled $473.0 million as of March 31, 2025.

Upcoming Events


Bank of America Securities 2025 Health Care Conference
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Management will participate in a fireside chat on Tuesday, May 13 at 8:00 a.m. PT. A live and archived audio webcast of the discussion will be available by visiting the Events section of the Company’s website.


Society for Neuro-Oncology’s 8th Biennial Pediatric Neuro-Oncology Conference, May 15-17, 2025
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A poster sponsored by Day One, "Managing side effects of MAPK inhibitor therapies in pediatric populations: a Delphi consensus initiative," will be presented on May 16 at 6:15 p.m. PT.


2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, May 30-June 3, 2025
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Abstract #10029 titled "Growth recovery in patients with BRAF altered pediatric low-grade gliomas (LGG) after discontinuation of tovorafenib" will be presented in a poster session on Saturday, May 31 from 9:00-12:00 p.m. CDT in Hall A.
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Abstract #10037 titled "Post hoc analysis of rashes reported in patients (pts) with BRAF-altered relapsed/refractory (r/r) pediatric low-grade glioma (pLGG) treated with the type II RAF inhibitor tovorafenib in FIREFLY-1" will be presented in a poster session on Saturday, May 31 from 9:00-12:00 p.m. CDT in Hall A.


46th Annual Goldman Sachs Global Healthcare Conference
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Management will participate in a fireside chat on Tuesday, June 10 at 8:40 a.m. ET. A live and archived audio webcast of the discussion will be available by visiting the Events section of the Company’s website.

Conference Call

Day One will host a conference call and webcast today, May 6 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.

Curis Provides First Quarter 2025 Business Update

On May 6, 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 business update and financial results for the first quarter ended March 31, 2025 (Press release, Curis, MAY 6, 2025, View Source [SID1234652571]).

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"I am pleased to announce the expansion of the Curis Executive Team with the addition of Dr. Ahmed Hamdy as our Chief Medical Officer. Dr. Hamdy is a well-known and respected leader in the industry and brings a wealth of experience as CMO of Pharmacyclics, CMO and CEO of Acerta, and CEO of Vincerx. We look forward to his contributions to Curis."

"Also, as we announced at the end of March, we have made exciting progress with both the FDA and EMA on the potential for accelerated approval of emavusertib in R/R PCNSL. We are currently enrolling patients in the TakeAim Lymphoma study to enable accelerated approval filings in both the EU and US. We expect to provide additional data for both BTK-naïve and BTK-experienced patients from the TakeAim Lymphoma study at ASH (Free ASH Whitepaper) later this year," said James Dentzer, President and CEO of Curis.

"Curis has made great progress developing IRAK4 as a novel oncology target and emavusertib as the first-in-class IRAK4 inhibitor. I am excited to be joining the Curis team at this critical time of advancement toward regulatory filing in PCNSL in the US and EU and expansion beyond PCNSL into additional indications in NHL, AML, and solid tumors. Given my experience developing both ibrutinib and acalabrutinib, I have a special appreciation for the potential of emavusertib in combination with BTK inhibition in NHL, and the importance of targeting IRAK4 and the toll-like receptor pathway in NHL, AML and solid tumors. I look forward to working with the Curis Team to bring this novel therapy to patients," said Dr. Hamdy.

Operational Highlights

TakeAim Lymphoma

Successfully concluded meetings with both the U.S. Food and Drug Administration (FDA) and the Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA) on the suitability of using the ongoing TakeAim Lymphoma study (NCT 03328078) to support a potential accelerated approval path in PCNSL.
Announced emavusertib had been granted Orphan Drug Designation for PCNSL in both the U.S. and Europe.
Provided data update for 27 patients enrolled in the ongoing TakeAim Lymphoma study in relapsed/refractory (R/R) PCNSL, 20 BTK-experienced patients and 7 BTK-naïve patients.
Ema-Ven-Aza Triplet Study in Frontline AML

Announced successful completion of the first dosing cohort and the commencement of dosing in the second cohort. Enrollment is currently ongoing.
Corporate

Completed a registered direct offering and concurrent private placement with net proceeds of approximately $8.8 million.
First Quarter 2025 Financial Results

For the first quarter of 2025, Curis reported a net loss of $10.6 million or $1.25 per share on both a basic and diluted basis as compared to $11.9 million or $2.05 per share on both a basic and diluted basis for the same period in 2024.

Revenues for the first quarter of 2025 were $2.4 million, as compared to $2.1 million for the same period in 2024. Revenues for both periods consist of royalty revenues from Genentech/Roche’s sales of Erivedge.

Research and development expenses were $8.5 million for the first quarter of 2025, as compared to $9.6 million for the same period in 2024. The decrease was primarily attributable to lower employee related costs.

General and administrative expenses were $4.0 million for the first quarter of 2025, as compared to $4.9 million for the same period in 2024. The decrease was primarily attributable to lower employee related and professional, legal and consulting costs.

Other expense, net was $0.5 million for the first quarter of 2025, as compared to other income, net of $0.6 million for the same period in 2024. The decrease was primarily attributable to an increase in the expense related to the sale of future royalties and a decrease in interest income.

Curis’s cash and cash equivalents totaled $20.3 million as of March 31, 2025, and the Company had approximately 10.5 million shares of common stock outstanding. Curis expects its existing cash and cash equivalents will enable its planned operations into the fourth quarter of 2025.

Conference Call Information

Curis management will host a conference call today, May 6, 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, or login here shortly before 8:30 a.m. ET. The conference call can also be accessed on the Curis website at the Events and Presentations section of the Investors page.

CRISPR Therapeutics Provides First Quarter 2025 Financial Results and Announces Positive Top-Line Data from Phase 1 Clinical Trial of CTX310™ Targeting ANGPTL3

On May 6, 2025 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the first quarter ended March 31, 2025 (Press release, CRISPR Therapeutics, MAY 6, 2025, View Source [SID1234652570]).

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"CRISPR Therapeutics remains focused on executing our strategic priorities and advancing our portfolio of innovative therapies. We are highly encouraged by the initial data from our Phase 1 trial for CTX310, which demonstrates the power of our in vivo gene editing platform to deliver paradigm changing medicines to patients with serious cardiovascular disease," said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. "Additionally, we are pleased with the continued progress of Casgevy and the broader pipeline, and we look forward to sharing further clinical updates in the months ahead."

Recent Highlights and Outlook

In Vivo Liver Editing Programs

CTX310 targets ANGPTL3, a gene that encodes for key protein involved in the regulation of low-density lipoprotein (LDL) and triglyceride (TG) levels – both well-established risk factors for atherosclerotic heart disease (ASCVD). Loss-of-function mutations in ANGPTL3 are associated with significantly reduced levels of LDL and TGs, as well as reduced risk of ASCVD, without adverse effects on overall health. In the U.S. alone, more than 40 million patients are affected by elevated LDL, severely elevated TGs or both – representing a large addressable patient population. CTX310 is initially focused on a high-risk subset of this group with the greatest unmet medical need and limited effective treatment options.

CTX310 is in an ongoing Phase 1 first-in-human dose escalation clinical trial targeting ANGPTL3 in four patient groups with elevated LDL, TG or both including homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (sHTG), heterozygous familial hypercholesterolemia (HeFH), or mixed dyslipidemias (MDL) with levels of TG (>300 mg/dL) and/or LDL-C (>100 mg/dL); >70 mg/dL for subjects with ASCVD. TG and LDL, both of which are validated as surrogate endpoints for clinical benefit and accepted by regulatory agencies, were assessed at various timepoints.

Top-line data reported today are from the first 10 patients across the first four cohorts (lean body weight-based doses of DL1 [0.1 mg/kg], DL2[0.3 mg/kg], DL3 [0.6 mg/kg] and DL4 [0.8 mg/kg]) with at least 30 days of follow-up for each participant as of a data cutoff date of April 16, 2025.

A single dose of CTX310 demonstrated dose-dependent decreases in ANGPTL3, TGs, and LDL. Based upon ANGPTL3 knockdown, DL1 and DL2 were minimally active doses, whereas treatment at DL3 and DL4 resulted in reductions of up to 75% of baseline levels in ANGPTL3. CTX310 has been well-tolerated, with no treatment-related severe adverse events (SAEs) and no grade ≥3 adverse events (AEs) reported. No clinically significant changes in alanine aminotransferase (ALT), aspartate aminotransferase (AST), bilirubin, or platelets were observed at any dose level. There were no dose-dependent trends in any of these laboratory measurements.

Mean % Change from Baseline at Day 30 post-infusion
(+/- SEM)
Dose Level (DL) DL1 + DL2
0.1 + 0.3 mg/kg
(n=6) DL3
0.6 mg/kg
(n=3) DL4
0.8 mg/kg
(n=1)
Patient type HeFH (4), MDL, sHTG MDL (2), HeFH sHTG
Triglycerides -10.6% ± 13.1% -55.7% ± 8.0% -81.9%
LDL 34.8% ± 27.0% -28.5% ± 24.4% -64.6%

Compelling individual patient responses highlight the therapeutic potential of CTX310: a DL4 patient with sHTG had an 82% reduction in triglycerides from a baseline of 1073 mg/dL at day 30, and a DL3 patient with HeFH had an 81% reduction in LDL-C from a baseline of 256 mg/dL at day 90 – supporting the potential for targeted efficacy in high-risk populations.

These initial results represent a significant milestone in the advancement of CRISPR Therapeutics’ proprietary lipid nanoparticle (LNP) delivery technologies for gene editing in the liver. The Company plans to present the CTX310 Phase 1 data 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 increased incidence of major adverse cardiovascular events (MACE). Elevated Lp(a) levels are prevalent in up to 20% of the global population. Dose escalation is ongoing, with an update expected in the second quarter of 2025.

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.

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

CASGEVY is approved in the U.S., Great Britain, the EU, the Kingdom of Saudi Arabia (KSA), the Kingdom of Bahrain (Bahrain), Canada, Switzerland and the United Arab Emirates (UAE) for the treatment of both SCD and TDT, and launches are ongoing. Building on the foundational launch in 2024, significant progress is being made to bring this transformative therapy to patients worldwide.

As of May 1, more than 65 authorized treatment centers (ATCs) have been activated globally and approximately 90 patients have had their first cell collection. The number of new patients initiating cell collection is expected to grow significantly throughout 2025.

Vertex has secured a formal reimbursement agreement with NHS England, enabling access to CASGEVY for patients with SCD. This follows an earlier agreement, reaching in August 2024, providing access for eligible patients with TDT. A similar reimbursement agreement has been established in Wales for eligible SCD and TDT patients. Following a positive assessment, national reimbursement was finalized in Austria. In the Middle East, reimbursement was also finalized across the majority of Emirates, following regulatory approval in the UAE.

A manufacturing license application has been submitted to the U.S. Food and Drug Administration (FDA), with commercial production in Portsmouth, New Hampshire expected to begin in the second half of 2025. This submission is part of the planned ramp-up of CASGEVY manufacturing capacity as demand for the therapy increases.

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 its next-generation allogeneic CAR T product candidates, CTX112 and CTX131, targeting CD19 and CD70, respectively, across multiple indications. Both candidates incorporate novel potency edits which can lead to significantly higher CAR T cell expansion and cytotoxicity, potentially establishing them as best-in-class allogeneic CAR T products for their respective targets. CTX112 is being developed for hematologic malignancies and autoimmune diseases and has the potential to be best-in-class based on preliminary data.

Encouraging clinical data from the ongoing Phase 1/2 clinical trial of CTX112 in relapsed or refractory B-cell malignancies supported the FDA’s decision to grant Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of relapsed or refractory follicular lymphoma and marginal zone lymphoma.

CTX112 is also in an ongoing Phase 1 clinical trial in autoimmune diseases, including indications such as 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 an update for both oncology and autoimmune disease in mid-2025.

Clinical trials for CTX131 are ongoing in both solid tumors and hematologic malignancies, with updates expected in 2025. In parallel, an Investigational New Drug (IND) application for glypican-3 (GPC3)-targeted gene-edited autologous CAR T program for the treatment of hepatocellular carcinoma has been opened by our partner, Roswell Park Comprehensive Cancer Center.

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.

Regenerative Medicine Programs

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

Upcoming Events

The Company will participate in the following events in May:

3rd Annual H.C. Wainwright BioConnect Investor Conference, May 20
2025 RBC Capital Markets Global Healthcare Conference, May 20

First Quarter 2025 Financial Results

Cash Position: Cash, cash equivalents, and marketable securities were $1,855.3 million as of March 31, 2025, compared to $1,903.8 million as of December 31, 2024. The decrease in cash was primarily driven by operating expenses, offset by proceeds from interest income and employee option exercises.

R&D Expenses: R&D expenses were $72.5 million for the first quarter of 2025, compared to $76.2 million for the first quarter of 2024. The decrease in R&D expense was primarily driven by a decrease in employee-related expenses, including stock-based compensation expenses.

G&A Expenses: General and administrative expenses were $19.3 million for the first quarter of 2025, compared to $18.0 million for the first quarter of 2024.

Collaboration Expense: Collaboration expense, net, was $57.5 million for the first quarter of 2025, compared to $47.0 million for the first quarter of 2024. The increase in collaboration expense, net, was primarily attributable to costs related to CASGEVY and collaboration expenses related to in vivo HSC editing, offset by CASGEVY product sales.

Net Loss: Net loss was $136.0 million for the first quarter of 2025, compared to a net loss of $116.6 million for the first 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 Collaboration and Vertex
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 CTX211
CTX211 is an allogeneic, gene-edited, stem cell-derived investigational therapy for the treatment of type 1 diabetes (T1D), which incorporates gene edits that aim to make cells hypoimmune and enhance cell fitness. This immune-evasive cell replacement therapy is designed to enable patients to produce their own insulin in response to glucose. A Phase 1 clinical trial for CTX211 for the treatment of T1D is ongoing.