On May 6, 2025 IDEAYA Biosciences, Inc. (Nasdaq: IDYA), a precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported a business update and announced financial results for the first quarter that ended March 31, 2025 (Press release, Ideaya Biosciences, MAY 6, 2025, View Source [SID1234652577]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"We have provided an updated cash runway guidance into 2029, and this past quarter we made significant progress on the darovasertib program, including receiving U.S. FDA breakthrough therapy designation, and enrollment is ahead of schedule with over 300 patients in the 1L HLA-A2-negative MUM registrational trial for a targeted median PFS readout by year-end to enable a potential accelerated approval filing next year. We also advanced a broad clinical pipeline of potential first-in-class programs to continue to drive forward our growth strategy, including DLL3 TOP1 ADC IDE849 in lung cancer, Werner Helicase inhibitor IDE275 in MSI-high colorectal and endometrial cancer, and MAT2A inhibitor IDE397 in MTAP-deletion lung and urothelial cancer," said Yujiro S. Hata, President and Chief Executive Officer, IDEAYA Biosciences.

Recent Key Developments and Upcoming Milestones

Research and Clinical Development

Darovasertib: a potential first-in-class PKC inhibitor in Phase 2/3 clinical testing for the treatment of metastatic uveal melanoma (MUM) and as neoadjuvant treatment for primary uveal melanoma (UM).

•
MUM
•
Part 2b with the selected optimal dose for the potential registration-enabling trial evaluating darovasertib and crizotinib in first line (1L) HLA-A2-negative MUM continues enrolling.
•
Median progression-free survival (PFS) readout for Phase 2/3 registration-enabling trial of the darovasertib and crizotinib combination in 1L HLA-A2-negative MUM targeted by year-end 2025. Rapid enrollment in the trial continues with over 300 patients as of May 5, 2025.
•
Phase 2 median overall survival (OS) readout from study IDE196-001 in over 40 1L MUM patients targeted at a medical conference in the second half of 2025. The readout will include both 1L HLA-A2-negative and HLA-A2-positive MUM patients. We continue to enroll additional HLA-A2-positive MUM patients in the IDE196-001 trial.
•
Neoadjuvant UM
•
Successfully completed a Type D meeting with the FDA on Phase 3 registrational trial design for darovasertib as neoadjuvant therapy for primary UM. The Phase 3 study is expected to enroll approximately 520 patients randomized 2:1 to receive darovasertib or control. Two cohorts include enucleation-eligible UM patients (n=120) and plaque brachytherapy (PB)-eligible UM patients (n=400). Primary endpoints, which are supportive of full approval based on the FDA Type D Meeting, include eye preservation rate for enucleation patients and proportion of patients with best corrected visual acuity 15-letter loss from time of randomization and time of completion of PB for the PB cohort, with event-free survival (EFS) as a required secondary endpoint for both cohorts. Commencement of the Phase 3 registration-enabling trial for darovasertib in neoadjuvant UM is targeted for the first half of 2025.
•
U.S. FDA granted breakthrough therapy designation for single agent darovasertib for the neoadjuvant treatment of adult patients with primary uveal melanoma (UM) for whom enucleation has been recommended.
•
Two clinical updates from the Company-sponsored Phase 2 trial targeted at medical conferences in mid-2025 and the second half of 2025. The mid-2025 update will be focused on vision data and the plaque brachytherapy patients, and the update in second half of 2025 will include over 90 UM patients from both the enucleation and plaque brachytherapy eligible cohorts.

IDE397: a potential first-in-class Phase 2 MAT2A inhibitor for the treatment of MTAP-deletion solid tumors.

•
Entered into an additional clinical study collaboration and supply agreement with Gilead to evaluate IDE397, IDEAYA’s MAT2A inhibitor, in combination with Trodelvy (sacituzimab govitecan-hziy), Gilead’s Trop-2 directed ADC, in MTAP-deletion NSCLC.
•
IDEAYA plans to enable the wholly-owned IDE397 and IDE892 (PRMT5MTA) combination in patients with MTAP-deletion non-small cell lung cancer (NSCLC) in the second half of 2025.
IDE849 (SHR-4849): a potential first-in-class Phase 1 DLL3 TOP1i antibody drug conjugate (ADC) targeting small cell lung cancer (SCLC) and neuroendocrine tumors (NETs).

•
U.S. IND clearance obtained for IDE849 Phase 1 study in small cell lung cancer (SCLC).
•
IDE849 currently being evaluated by Hengrui Pharma in an ongoing Phase 1 trial in China in SCLC patients. In January 2025, partner Hengrui Pharma selected expansion doses for the study. Clinical efficacy and safety data from over 40 SCLC patients in the multi-site open label Phase 1 trial, including the dose escalation and multiple expansion doses, will be presented at a medical conference in Q3 2025.
•
Initiation of evaluation of IDE849 and IDE161 combination targeted in the second half of 2025.
IDE275 (GSK959): a potential first-in-class and best-in-class Phase 1 Werner Helicase inhibitor for the treatment of high microsatellite instability (MSI-High) tumors.

•
Phase 1 dose escalation trial ongoing in MSI-H solid tumors with GSK.
•
IDE275 highlighted in an oral presentation in the New Drugs on the Horizon series, and three poster presentations, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2025 Annual Meeting. The preclinical data demonstrated the molecule’s selectivity to treat MSI-H solid tumors and potential to be developed clinically as both a monotherapy agent and in combination with anti-PD1. A Trial in Progress poster for the ongoing SYLVER Phase 1/2 study (NCT06710847) was also presented at AACR (Free AACR Whitepaper) 2025.
IDE161: a potential first-in-class Phase 1 PARG inhibitor for the treatment of solid tumors.
•
Phase 1 monotherapy dose optimization is ongoing. The clinical focus for the IDE161 program moving forward will be on enrollment with combination with IDE849.
•
Preclinical data on immune checkpoint inhibitor (ICI)-driven anti-tumor immunity was presented at AACR (Free AACR Whitepaper) 2025
•
Targeting to present preclinical combination mechanism and synergy efficacy data of IDE161 with TOP1-payload based ADCs at a medical conference in the third quarter of 2025
IDE705 (GSK101): a potential first-in-class Phase 1 Pol Theta Helicase Inhibitor in combination with PARP inhibitor for the treatment of HRD solid tumors.

•
Targeting Phase 2 expansion in HRD solid tumors, which would trigger a potential $10 million milestone payment from GSK.
IDE892: a potential best-in-class MTA-cooperative PRMT5 inhibitor to enable wholly-owned combination with IDE397.

•
IND filing targeted for mid-year 2025.
•
Preclinical data providing insights into metabolite kinetics and PRMT5 dysregulation in MTAP-deficient cancers was presented at AACR (Free AACR Whitepaper) 2025.
IDE034: a potential first-in-class B7H3/PTK7 TOP1i bispecific ADC with combination potential with IDE161.

•
IND filing targeted for the second half of 2025.
IDE574: a potential first-in-class KAT6/7 dual inhibitor development candidate with combination opportunities with multiple programs in the Company’s pipeline.

•
IND filing targeted for the second half of 2025.
•
Preclinical data on dual inhibition’s impact on epigenetics and adaptive drug resistance was presented at AACR (Free AACR Whitepaper) 2025.
Corporate Development and Operations

•
Formed a research collaboration with ATTMOS to develop a physics-based computational platform for small molecule discovery, aimed at swiftly unlocking oncology targets traditionally considered undruggable. The collaboration will integrate IDEAYA’s differentiated and proven capabilities in structural biology and pharmaceutical drug discovery across multiple first-in-class oncology targets with ATTMOS’s capabilities in computational chemistry method development, high performance computing, and software development.

•
Joshua Bleharski, Ph.D., joined IDEAYA as Chief Financial Officer. Dr. Bleharski joins from J.P. Morgan, serving most recently as Managing Director and Global Co-Head of Biopharma in the Healthcare Investment Banking group. Josh spent nearly 17 years at J.P. Morgan advising clients in the biopharma sector on capital markets transactions, corporate strategy and other investment banking services representing more than $65 billion of value for biotechnology companies worldwide.
•
Shanthakumar Tyavanagimatt, Ph.D., joined IDEAYA as Senior Vice President, Technical Operations, where he will lead IDEAYA’s darovasertib global commercial supply chain readiness activities, as well as the technical operations activities across IDEAYA’s preclinical and clinical-stage pipeline. Prior to IDEAYA, Shanthakumar brings over 20-years of technical operations experience to IDEAYA, including approximately 9-years at CTI Biopharma (acquired by SOBI, Inc.) where he led the technical operations function for multiple commercial product launches.
•
Updated cash runway guidance into 2029 based on current operating plan.
Financial Results

As of March 31, 2025, IDEAYA had cash, cash equivalents and marketable securities of approximately $1.05 billion. This compared to cash, cash equivalents and marketable securities of approximately $1.08 billion as of December 31, 2024 The decrease in the balance as of March 31, 2025 was primarily driven by net cash used in operations which was offset by $25.0 million in net proceeds from the sale of common stock shares through at-the-market financings during the quarter.

IDEAYA projects that the $1.05 billion in cash, cash equivalent and marketable securities balance as of March 31, 2025 will be sufficient to fund its planned operations into 2029 based on its current operating plan. We have updated our operating plan costs with further pipeline prioritization, including focusing: 1) the IDE161 clinical program on the combination study with DLL3 TOP1 ADC IDE849, 2) the IDE397 and PRMT5 mechanism clinical combination activities to wholly-owned PRMT5 inhibitor IDE892 (PRMT5MTA), and 3) the clinical dose escalation and expansion data for the IDE397 and Trodelvy combination in MTAP-deletion UC, to be utilized for the MTAP-deletion NSCLC indication clinical expansion activities.

There was no collaboration revenue for the three months ended March 31, 2025, compared to $7.0 million in collaboration for the three months ended December 31, 2024. Collaboration revenue for the three months ended December 31, 2024 was related to a milestone payment from GSK that was earned for the IND clearance of IDE275 (GSK959) in October 2024.

Research and development (R&D) expenses for the three months ended March 31, 2025, totaled $70.9 million compared to $140.2 million for the three months ended December 31,2024. The decrease was primarily due to a one-time $75.0 million upfront payment under the license agreement for IDE849 with Hengrui Pharma that occurred in December 2024, offset by higher clinical trial, consulting and personnel-related expenses to support our pipeline.

General and administrative (G&A) expenses for the three months ended March 31, 2025 totaled $13.5 million compared to $11.0 million for the three months ended December 31, 2024. The increase was primarily due to higher personnel-related, consulting and legal patent expenses to support our growth.

The net loss for the three months ended March 31, 2025, was $72.2 million compared to the net loss of $130.3 million for the three months ended December 31, 2024. Total stock compensation expense for the three months ended March 31, 2025, was $10.2 million compared to $9.5 million for the three months ended December 31, 2024.

On May 6, 2025 Heron Therapeutics, Inc. (Nasdaq: HRTX) ("Heron" or the "Company"), a commercial-stage biotechnology company, reported financial results for the three months ended March 31, 2025, and highlighted recent corporate updates (Press release, Heron Therapeutics, MAY 6, 2025, View Source [SID1234652576]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"We are off to a strong start in 2025, achieving record adjusted EBITDA for the first quarter. Building on our efforts to strengthen our financial foundation, we are well positioned for future growth, with strong tailwinds for our lead product, ZYNRELEF. These include the expanded label indications, the approval of the NOPAIN Act, the launch of the VAN, and the partnership with Crosslink Network, LLC," said Craig Collard, Chief Executive Officer.

Financial Guidance for 2025

Item

2025 Full-Year Guidance for Net Revenue and Adjusted EBITDA
(in millions)

Original

Q1 Updated Guidance

Net Revenue

$153.0 to $163.0

Adjusted EBITDA

$0.0 to $8.0

$4.0 to $12.0

Business Highlights

Net Revenue growth of 12.2% Q1 2025 over Q1 2024, primarily driven by the acute care franchise which increased revenue by 89.4%; ZYNRELEF grew 60.4%.
Settlement reached with Mylan Pharmaceuticals, Inc. ("Mylan"), wherein the Company has granted Mylan a license to market generic versions of CINVANTI and APONVIE in the United States beginning June 1, 2032, or earlier under certain customary circumstances.
Non-Opioid Policy for Pain Relief took effect April 1, providing separate payment for non-opioids like ZYNRELEF by the Centers for Medicare & Medicaid Services with significant awareness among health care providers being recognized.
Successful launch of the VAN for ZYNRELEF continues to progress, offering a more efficient aseptic preparation, streamlining operations within the surgical setting.
Cash, cash equivalents, and short-term investments were $50.7 million as of March 31, 2025.
ZYNRELEF device transition for product preparation for use from the Vented Vial Spike ("VVS") to the Vial Access Needle ("VAN") proceeds smoothly with an orderly and efficient draw down of the VVS inventory.
ZYNRELEF development of the ready to use Prefilled Syringe ("PFS") continues with a projected early 2027 launch.
Net Revenue Performance – Quarter Ended March 31

2025

2024

Dollar Change

Percentage Change

Acute Care

$10,302

$5,438

$4,864

89.4 %

APONVIE

$2,260

$425

$1,835

431.8 %

ZYNRELEF

$8,042

$5,013

$3,029

60.4 %

Oncology

$28,601

$29,232

($631)

(2.2 %)

CINVANTI

$25,742

$25,617

$125

0.5 %

SUSTOL

$2,859

$3,615

($756)

(20.9 %)

Total Net Revenue

$38,903

$34,670

$4,233

12.2 %

Conference Call and Webcast

Heron will host a conference call and live webcast on Tuesday, May 6, 2025, at 8:00 a.m. ET. The conference call can be accessed by phone by utilizing the following registration link which will provide participants with dial-in details. To avoid delays, we encourage participants to dial into the conference call fifteen minutes ahead of the scheduled start time. The conference call will also be available via webcast under the Investor Relations section of Heron’s website at www.herontx.com. An archive of the teleconference and webcast will also be made available on Heron’s website for sixty days following the call.

On May 6, 2025 Halozyme Therapeutics, Inc. (NASDAQ: HALO) ("Halozyme" or the "Company") reported its financial and operating results for the first quarter ended March 31, 2025, provided an update on its recent corporate activities and raised its 2025 financial guidance (Press release, Halozyme, MAY 6, 2025, View Source [SID1234652575]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"2025 is off to a strong start with our current three blockbuster brands, Darzalex SC, Phesgo and VYVGART Hytrulo, continuing to demonstrate strong growth in their currently approved indications. Our four recently launched products, Ocrevus Zunovo in U.S. and Europe, Tecentriq Hybreza in U.S. and Europe, Opdivo Qvantig in U.S. and Rybrevant SC in Europe are just beginning their contributions as our partners focus on gaining and expanding coverage and reimbursement. This broadened portfolio is resulting in an unprecedented set of 11 additional growth catalysts that have happened recently or are expected to happen in the coming months. These opportunities include multiple new European and U.S. product approvals, multiple new indication approvals and multiple key reimbursement milestones supporting access for an even greater number of patients, all creating new near and longer-term growth opportunity. As a result of this momentum, I am pleased to announce we are increasing our full year 2025 financial guidance ranges and a new $250 million share buyback," said Dr. Helen Torley, president and chief executive officer, of Halozyme.

"In addition, our long-term growth prospects have never been better with additional growth opportunities projected to result from our pipeline, where two products, BMS’ nivolumab plus relatlimab SC and Takeda’s 20% immune globulin SC, continue in Phase 3 and where ViiV and Acumen announced development progress and data in the quarter. I am also pleased to announce that we have signed our first HVAI development agreement with a current ENHANZE partner and that a different ENHANZE partner is now moving our SVAI into clinical testing," concluded Dr. Torley.

First Quarter and Recent Corporate Highlights:
•On May 6, Halozyme announced a second $250 million share repurchase under the $750 million approved program from February 2024.
•In April 2025, Halozyme filed a patent infringement lawsuit against Merck Sharp & Dohme Corp. ("Merck") in the U.S. District Court in New Jersey alleging that Merck is using Halozyme’s patented MDASE subcutaneous drug delivery technology to develop Subcutaneous ("SC") Keytruda. Halozyme is seeking damages and injunctive relief to stop Merck’s infringement of Halozyme’s MDASE intellectual property.
•In March 2025, Halozyme completed the first $250 million Accelerated Share Repurchase of its common stock under the $750 million approved program from February 2024.

First Quarter and Recent Partner Highlights:
•In April 2025, Roche received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use ("CHMP") recommending an update to the European Union ("EU") label for Phesgo for human epidermal growth factor receptor 2 ("HER2")-positive breast cancer. Administration of Phesgo outside of a clinical setting (such as in a person’s home) by a healthcare professional will be possible, once safely established in a clinical setting.
•In April 2025, argenx received a positive opinion from the CHMP recommending European Commission approval of VYVGART 1000mg (efgartigimod alfa) developed with ENHANZE for SC injection as a monotherapy for the treatment of adult patients with progressive or relapsing active chronic inflammatory demyelinating polyneuropathy ("CIDP") after prior treatment with corticosteroids or immunoglobulins.
•In April 2025, argenx received U.S. Food and Drug Administration ("FDA") approval of VYVGART Hytrulo prefilled syringe for self-injection for the treatment of adult patients with generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive and adult patients with CIDP.
•In April 2025, Janssen received European Commission marketing authorization of the SC formulation of RYBREVANT (amivantamab) with ENHANZE, in combination with LAZCLUZE (lazertinib), for the first-line treatment of adult patients with advanced non-small cell lung cancer ("NSCLC") with epidermal growth factor receptor ("EGFR") exon 19 deletions or exon 21 L858R substitution mutations. Additionally, RYBREVANT (amivantamab) is approved as a monotherapy for adult patients with advanced NSCLC with activating EGFR exon 20 insertion mutations after the failure of platinum-based therapy. This represents the 10th partner product with ENHANZE to be commercialized.
•In April 2025, Janssen received European Commission approval for an indication extension of DARZALEX SC in combination with bortezomib, lenalidomide, and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma regardless of transplant eligibility.
•In March 2025, Bristol Myers Squibb received a positive CHMP opinion recommending approval of Opdivo (nivolumab) with ENHANZE across multiple solid tumor indications.
•In March 2025, Acumen announced top-line results from a Phase 1 study of sabirnetug (ACU193) with ENHANZE comparing the pharmacokinetics between SC and intravenous administrations in healthy volunteers that demonstrated weekly SC administration of sabirnetug was well-tolerated with systematic exposure supporting further clinical development.
•In March 2025, ViiV announced results from a Phase 2b study demonstrated N6LS administered every four months SC with ENHANZE in combination with cabotegravir successfully maintained viral suppression in adults living with HIV who were already stable on treatment.

•In March 2025, Takeda announced Health Canada expanded the marketing authorization for HYQVIA to include CIDP as a maintenance therapy after stabilization with intravenous immunoglobulin to prevent relapse of neuromuscular disability and impairment in adults.

First Quarter 2025 Financial Highlights:
•Revenue was $264.9 million, compared to $195.9 million in the first quarter of 2024. The 35% year-over-year increase was primarily driven by royalty revenue growth and an increase in sales of bulk rHuPH20. Revenue for the quarter included $168.2 million in royalties, an increase of 39% compared to $120.6 million in the first quarter of 2024, primarily attributable to increases in revenue of VYVGART Hytrulo, DARZALEX SC, and Phesgo.
•Cost of sales was $48.4 million, compared to $28.3 million in the first quarter of 2024. The increase in cost of sales was primarily due to an increase in product sales.
•Amortization of intangibles expense remained flat at $17.8 million, compared to the first quarter of 2024.
•Research and development expense was $14.8 million, compared to $19.1 million in the first quarter of 2024. The decrease in research and development expense was primarily due to lower compensation expense driven by resource optimization and labor allocation initiatives, and timing of planned investments in ENHANZE related to the development of our new high-yield rHuPH20 manufacturing process.
•Selling, general and administrative expense was $42.4 million, compared to $35.1 million in the first quarter of 2024. The increase was primarily due to an increase in consulting and professional service fees and compensation expense.
•Operating income was $141.5 million, compared to $95.5 million in the first quarter of 2024.
•Net income was $118.1 million, compared to $76.8 million in the first quarter of 2024.
•EBITDA and Adjusted EBITDA were $162.0 million, compared to $115.7 million in the first quarter of 2024.1
•GAAP diluted earnings per share was $0.93, compared to $0.60 in the first quarter of 2024. Non-GAAP diluted earnings per share was $1.11, compared to $0.79 in the first quarter of 2024.1
•Cash, cash equivalents and marketable securities were $747.9 million on March 31, 2025, compared to $596.1 million on December 31, 2024. The increase was primarily a result of cash generated from operations.

Financial Outlook for 2025
The Company is raising its financial guidance for 2025. Note that the guidance reflects tariffs that are currently implemented.
For the full year 2025, the Company expects:

•Total revenue of $1,200 million to $1,280 million, representing growth of 18% to 26% over 2024 total revenue, primarily driven by increases in royalty revenue. Revenue from royalties of $750 million to $785 million, representing growth of 31% to 37% over 2024.
•Adjusted EBITDA of $790 million to $840 million, representing growth of 25% to 33% over 2024.
•Non-GAAP diluted earnings per share of $5.30 to $5.70, representing growth of 25% to 35% over 2024. The Company’s earnings per share guidance does not consider the impact of potential future share repurchases.

Table 1. 2025 Financial Guidance

Previous Guidance Range
New Guidance Range
Total Revenue
$1,150 to $1,225 million
$1,200 to $1,280 million
Royalty Revenue
$725 to $750 million
$750 to $785 million
Adjusted EBITDA
$755 to $805 million
$790 to $840 million
Non-GAAP Diluted EPS
$4.95 to $5.35
$5.30 to $5.70

1 Adjusted EBITDA and non-GAAP Diluted EPS are non-GAAP financial measures. See "Note Regarding Use of Non-GAAP Financial Measures" below for an explanation of these measures.

Webcast and Conference Call
Halozyme will host its Quarterly Update Conference Call for the first quarter ended March 31, 2025 today, Tuesday, May 6, 2025, at 1:30 p.m. PT/4:30 p.m. ET. The conference call may be accessed live with pre-registration via link: View Source The call will also be webcast live through the "Investors" section of Halozyme’s corporate website and a recording will be made available following the close of the call. To access the webcast and additional documents related to the call, please visit Halozyme.com.

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"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.

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

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."