Relay Therapeutics Reports First Quarter 2025 Financial Results and Corporate Updates

On May 5, 2025 Relay Therapeutics, Inc. (Nasdaq: RLAY), a clinical-stage precision medicine company transforming the drug discovery process by combining leading-edge computational and experimental technologies, reported first quarter 2025 financial results and corporate updates (Press release, Relay Therapeutics, MAY 5, 2025, View Source [SID1234652518]).

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"2025 is a year of execution across a range of high value clinical programs," said Sanjiv Patel, M.D., President and Chief Executive Officer of Relay Therapeutics. "The ongoing changes to our cost base are designed to enable a full funding of key initiatives including generating topline data from the ReDiscover-2 trial and clinical proof-of-concept data in vascular malformations."

Strategic Cost Reductions Implemented to Extend Runway into 2029 and to Fully Fund Key Value Drivers


These reductions help extend operating runway into 2029 and are aimed at funding key objectives, including:
o
Completion of ReDiscover-2 Phase 3 trial of RLY-2608 + fulvestrant in metastatic breast cancer well past topline data readout and additional breast cancer clinical trials
o
Execution of RLY-2608 Phase 1 vascular malformations trial through clinical proof-of-concept data
o
Advance Fabry and NRAS program to Investigational New Drug application (IND) readiness
o
Advance one research-stage program


Specific cost reductions include:
o
Over the past year, focused the research portfolio and platform on the highest value areas resulting in a reduction in the research run rate spend by approximately 80%

Reduced research-stage programs from four to one

Recent reduction in force by approximately 70 people
o
Executed a global out-license of RLY-4008 with Elevar Therapeutics, Inc. (Elevar) with potential for downstream economics
o
Phased the timing of entry into the clinic for Fabry and NRAS targeted programs

RLY-2608 Highlights


Breast Cancer
o
Initiation of Phase 3 ReDiscover-2 trial of RLY-2608 + fulvestrant in PI3Kα-mutated, CDK4/6 pre-treated, HR+/HER2- advanced breast cancer remains on track for mid-2025
o
Abstract accepted to ASCO (Free ASCO Whitepaper) for update of Phase 1b ReDiscover trial of RLY-2608 + fulvestrant

Focus of the abstract is updated 600mg BID (fasted) doublet data with median follow-up now greater than 12 months

Poster Title: Updated efficacy of mutant-selective PI3Kα inhibitor RLY-2608 in combination with fulvestrant in patients with PIK3CA-mutant HR+HER2- advanced breast cancer: ReDiscover trial

Date/Time: Monday, June 2, 10:00-1:00 p.m. ET (9:00-12:00 p.m. CT)
o
Continued advancement of the ongoing triplet cohorts with RLY-2608 + fulvestrant + atirmociclib or ribociclib
o
Planning continues for development of next-generation endocrine therapy combinations with RLY-2608

Vascular Malformations
o
Initiation of Phase 1 vascular malformations clinical trial in the first quarter of 2025

First Quarter 2025 Financial Results

Cash, Cash Equivalents and Investments: As of March 31, 2025, cash, cash equivalents and investments totaled $710.3 million, as compared to $781.3 million as of December 31, 2024. The company expects its current cash, cash equivalents, and investments will be sufficient to fund its operating expenses and capital expenditure requirements into 2029.

Revenue: Revenue was $7.7 million for the first quarter of 2025, as compared to $10.0 million for the first quarter of 2024. The revenue recognized in the first quarter of 2025 was due to completion of all performance obligations under the company’s Exclusive License Agreement with Elevar. The revenue recognized in the first quarter of 2024 was due to a milestone achieved under the Collaboration and License Agreement with Genentech, Inc.

R&D Expenses: Research and development expenses were $73.8 million for the first quarter of 2025, as compared to $82.4 million for the first quarter of 2024. The decrease was primarily due to the series of strategic choices made to streamline the research organization throughout 2024.

G&A Expenses: General and administrative expenses were $18.7 million for the first quarter of 2025, as compared to $19.8 million for the first quarter of 2024. The decrease was primarily due to a decrease in stock compensation expense, partially offset by costs to obtain the agreement with Elevar, which were expensed commensurate with the timing of revenue recognized.

Net Loss: Net loss was $77.1 million for the first quarter of 2025, or a net loss per share of $0.46, as compared to a net loss of $81.4 million for the first quarter of 2024, or a net loss per share of $0.62.

Recursion Reports First Quarter 2025 Financial Results and Provides Business Update

On May 5, 2025 Recursion (Nasdaq: RXRX), a leading clinical stage TechBio company decoding biology to radically improve lives, reported business updates financial results for its first quarter ending March 31, 2025 (Press release, Recursion Pharmaceuticals, MAY 5, 2025, View Source [SID1234652517]).

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Recursion will host a L(earnings) Call on May 5, 2025 at 8:00 am ET / 6:00 am MT / 1:00 pm BST from Recursion’s X (formerly Twitter), LinkedIn, and YouTube accounts giving analysts, investors, and the public the opportunity to ask questions of the company by submitting question here: View Source

"Recursion’s decade-long investment in AI is driving a decisive, data-led portfolio strategy," said Chris Gibson, Co-Founder and CEO of Recursion. "We are prioritizing high-potential programs to accelerate better treatments to patients, building on our platform’s unique ability to learn and lead this transformative shift in drug discovery. Our deep appreciation goes to the patients and investigators whose participation is invaluable on this journey."

Summary of Business Highlights

Pipeline Updates
"Our portfolio evolution reflects Recursion’s commitment to advancing medicines in areas of high unmet need where we believe we can have the greatest impact," said Najat Khan, Chief R&D Officer and Chief Commercial Officer at Recursion. "As part of our business combination with Exscientia, we are proactively streamlining our portfolio, platform, and operations, making deliberate tradeoffs to focus resources on programs with the strongest scientific rationale and the highest potential for near- and long-term impact. Powered by the integrated Recursion OS platform, we are advancing a focused set of differentiated internal and partnered programs. Our approach is grounded in rigorous science and evidenced by the consistent delivery of key milestones with leading pharmaceutical and technology collaborators."

•Preliminary Program Data:
▪REC-4881 (MEK1/2): Preliminary data presented at DDW as a late-breaking oral presentation on May 4, 2025 from the ongoing Phase 1b/2 TUPELO study of REC-4881 in familial adenomatous polyposis (FAP):
•In the Phase 2 open-label study, REC-4881 (4 mg QD) led to a preliminary median 43% reduction (n=6 patients) in polyp burden at the week 13 assessment at time of data cutoff.
•Five of six patients (83%) experienced reductions in polyp burden ranging from 31% to 82%, however, one patient showed a substantial increase from baseline.
•At Week 13, 50% of patients (3 out of 6) achieved ≥1-point improvement in Spigelman stage, a measure of upper GI disease severity.
•The early safety profile of REC-4881 was generally consistent with that of prior MEK1/2 inhibitors; among 19 patients across Phase 1b and 2, most treatment-related adverse events were Grade 1 or 2, with Grade 3 events in 16% of patients and no Grade ≥4 TRAEs reported to date.
▪REC-7735 (PI3Kα H1047R): Candidate profiling ongoing targeting PI3Kα H1047R mutant breast cancer; DC nomination expected 2H25:
•Highly selective and structurally differentiated molecule to reduce dose-limiting hyperglycemia.
•REC-7735 showed dose-dependent tumor regression in PI3Kα H1047R CDX models, with no elevation in insulin levels or hyperglycemia markers in wild-type mice, unlike standard-of-care PI3K inhibitors.
•Demonstrated dose-dependent tumor regression in preclinical models, with low-dose REC-7735 outperforming high-dose capivasertib (AKT inhibitor) in efficacy and tolerability.
▪REV102 (ENPP1): IND-enabling studies ongoing for hypophosphatasia (HPP) in development with Rallybio; Phase 1 initiation expected 2H26:
•Highly selective and orally bioavailable molecule supports QD or BID dosing.
•In vivo data in early-onset HPP model shows improved survival while treatment in late-onset HPP model improves bone defects.
•Preliminary data supports first-in-class potential for adult-onset HPP.
◦Additional Strategic Pipeline Programs
▪Focus on highest value programs in core therapeutic areas (oncology and rare disease):
•REC-617 (CDK7): Phase 1/2 ELUCIDATE study ongoing in advanced solid tumors; molecule designed to maximize therapeutic index; best-in-class potential.
•REC-1245 (RBM39): Phase 1/2 DAHLIA study with dose-escalation ongoing for biomarker-selected solid tumors and R/R lymphomas; novel mechanism of action identified to modulate DDR; first-in-class potential.
•REC-3565 (MALT1): Phase 1 EXCELERIZE study recently initiated for B cell malignancies; designed to avoid UGT1A1 on-target toxicity; best-in-class potential.
•REC-4539 (LSD1): Precision designed for reversibility and CNS penetration in solid tumors (e.g.; SCLC); strategic pause to ensure a competitive Target Product Profile.
•Continued focus on advancing additional discovery programs that meet key criteria.
▪As part of this prioritization, the Company will discontinue development and/or pursue partnering opportunities for the following clinical programs:
•REC-2282 (NF2): Totality of data supports the discontinuation of the study
◦New findings: Phase 2 passed the futility threshold primarily driven by the 40mg cohort, however the 60mg and combined dose arms did not pass the futility criteria.
◦Limited overall tumor shrinkage and clinical activity across all arms.
•REC-994 (CCM): Totality of data supports the discontinuation of study
◦Early data suggested potential promising trends in exploratory efficacy endpoints at 400mg (mean volume reduction, mRS), negative trends in efficacy at 200mg (data were not statistically significant).
◦New findings: Long-term extension results showed no promising trends in MRI or functional outcomes in the placebo-to-400mg crossover, and the 400mg-to-400mg arm did not continue prior trends and was indistinguishable from natural history.
•REC-3964 (C. difficile): The company will consider out-licensing opportunities
◦Evolved treatment options result in low recurrence rates (~5%); thus limiting unmet need.
◦Strategic decision to focus on other areas with greater unmet need.

◦Upcoming milestones:
▪REC-617 (CDK7): On track to initiate CDK7 combination studies in 1H25, additional monotherapy data expected in 2H25.
▪REC-4881 (MEK1/2): Additional data in FAP from TUPELO expected in 2H25.

▪REC-7735 (PI3Kα H1047R): preclinical studies ongoing with development candidate expected in 2H25.
▪REC-1245 (RBM39): Early Phase 1 safety and PK monotherapy data expected in 1H26.
▪REC-3565 (MALT1): Early Phase 1 safety and PK monotherapy data expected in 2H26.
▪REV102 (ENPP1): Phase 1 initiation expected in 2H26.

Partnerships Updates
Recursion and Sanofi advanced their fourth partnered program through a significant discovery milestone. This milestone involved the Recursion OS identifying differentiated, orally active small molecule leads against a high-interest immune cell target. These leads exhibit potential best-in-class properties, addressing significant liabilities seen in other candidates. As a result of this milestone, Recursion has received a $7 million milestone payment with the potential for over $300 million in additional milestone payments for this program. In total, the partnership has generated $130 million of cash inflows (including an upfront payment) to Recursion to date.

Recursion’s collaboration within Neuroscience and a GI Oncology Indication for Roche and Genentech continues to bring unbiased novel biological insights to potential programs. To date, the collaboration has built five phenomaps derived from a vast dataset of over one trillion iPSC-derived cells, one hundred billion GI Oncology relevant cells, alongside around 5,000 transcriptomes representing approximately 171 TB of data. Our approach continues to integrate high-throughput screens of genetics and small molecules with detailed cell measurements, informing our AI/ML models. Looking ahead, we are actively building additional maps and are focused on leveraging the Recursion OS and collaborating with Roche and Genentech to identify new novel programs that will fuel program advancement in both a GI Oncology indication and within Neuroscience.

Platform Updates
◦The platform is continuing to expand its ClinTech focus including high-quality, linked data assets, to industrialize clinical development, reduce costs, and accelerate the development of novel therapeutics. Updates include:
▪Leveraging Tempus data across Recursion’s oncology programs to expand therapeutic areas, which the Company believes will enrich patient population subgroups, and help increase likelihood of response for oncology clinical programs.
▪Signing an agreement with HealthVerity to integrate de-identified data for over 340 million covered lives within the US into Recursion OS, allowing for deeper insights into patient populations, enhanced trial design and feasibility assessments, as well as clinical operations workflows.
◦The collaboration with Enamine, leveraging Recursion’s massive data layer of predicted protein-small molecule interactions, resulted in the generation of enriched screening libraries to target 100 key and clinically relevant drug targets. The screening libraries are now available for purchase from Enamine.

Financial and Corporate Updates
"Recursion is stronger as a combined company, allowing us to not only deliver on operational goals more efficiently, but also be nimble in periods of uncertainty," stated Ben Taylor, CFO of

Recursion and President Recursion UK. "We have been able to significantly lower costs without cutting important platform capabilities by decreasing overall capacity. We maintain the ability to restore capacity in the future to respond to market needs. This aligns closely with our fundamental goal of using AI and automation to make drug discovery more flexible, efficient and effective."

First Quarter 2025 Financial Results

•Cash Position: Cash and cash equivalents and restricted cash were $509 million as of March 31, 2025 compared to $603 million as of December 31, 2024.
•Revenue: Total revenue, consisting primarily of revenue from collaborative agreements, was $15 million for the first quarter of 2025, compared to $14 million for the first quarter of 2024 due to the timing of projects from the Company’s Sanofi, Roche and Merck KGaA, Darmstadt, Germany collaborations.
•Research and Development Expenses: Research and development expenses were $130 million for the first quarter of 2025, compared to $68 million for the first quarter of 2024. The increase was primarily driven by the Company’s agreement with Tempus as well as our business combination with Exscientia in November 2024. This includes $27 million in non-cash expenses for use of Tempus’ patient-centric multimodal oncology data for Recursion programs.
•General and Administrative Expenses: General and administrative expenses were $55 million for the first quarter of 2025, compared to $31 million for the first quarter of 2024. The increase compared to prior period was primarily due to the inclusion of G&A expenses from the business combination with Exscientia.
•Net Loss: Net loss was $202.5 million for the first quarter of 2025, compared to a net loss of $91.4 million for the first quarter of 2024.
•Net Cash: Net cash used in operating activities was $132 million for the first quarter of 2025, compared to net cash used in operating activities of $102 million for the first quarter of 2024. The difference was primarily driven by higher costs incurred for R&D and G&A due to the Company’s business combination with Exscientia, in addition to $16 million of one-time transaction related costs in the first quarter of 2025.

Integration update and guidance
•Operational teams have been functioning as consolidated groups since immediately after closing. Core integration plans are completed or on schedule across the company.
•Expected cash burn* excluding partnering or financing inflows for 2025 of equal to or less than $450 million, excluding the benefit of potential cash inflows from existing or new partnerships. In 2024, the combined cash burn excluding partnering or financing inflows was approximately $606 million, including $203 million of change in cash from Exscientia prior to the business combination and $403 million from Recursion, excluding $49 million of respective partnership inflows.
•1Q25 cash burn excluding partnering or financing inflows of approximately $118 million, excluding transaction related costs
•Projected cash runway into mid 2027 based on current business plan.
•Primary areas of combination synergies and operational savings beyond pipeline prioritization:
◦Duplicated corporate expenses
◦Reduction in capacity of drug discovery operations
◦Utilization of broader platform capabilities to reduce project costs
◦Increasing administrative efficiency
◦Rationalization of facilities and office locations
◦Greater purchasing power with vendors
◦Spin-out of Austrian operations

*Cash burn is a non-GAAP financial measure. See "Non-GAAP Financial Measures" below for additional information regarding cash burn and for a reconciliation of cash burn to net cash used in operating activities for historical periods, the most directly comparable GAAP financial measure. With respect to the expected cash burn for 2025, certain items that affect the calculation of the GAAP financial measure for net cash used by operating activities are not available on a forward-looking basis because such items cannot be reasonably calculated without unreasonable effort due to the unpredictability of the amounts and timing of events affecting the items we exclude from cash burn. Consequently, the Company is unable to provide a reconciliation of net cash used in operating activities to cash burn for the Company’s fiscal 2025 guidance.

Expanded Board:
•Namandjé Bumpus, Ph.D, and Elaine Sun have been appointed to Recursion’s Board of Directors, effective as of March 15th.
•Dr. Bumpus brings deep experience in scientific innovation and regulatory strategy, while Elaine Sun adds extensive leadership in life sciences finance and corporate strategy.

Orna Therapeutics Announces Presentation at the American Society of Gene and Cell Therapy Annual Meeting Supporting its in vivo CAR Therapy Approach in Autoimmune Diseases

On May 5, 2025 Orna Therapeutics, a biotechnology company dedicated to engineering immune cells in vivo to treat autoimmune and oncology diseases, reported an upcoming presentation at the 28th American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting being held May 13-17, 2025, in New Orleans, Louisiana (Press release, Orna Therapeutics, MAY 5, 2025, View Source [SID1234652516]). The presentation will highlight data supporting Orna’s in vivo CAR therapy approach in autoimmune diseases.

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"We are excited to present new data at the upcoming ASGCT (Free ASGCT Whitepaper) annual meeting as we advance our panCAR programs towards the clinic," said Joseph Bolen, Ph.D., Chief Executive Officer of Orna Therapeutics. "Our platform, which pairs our circular (oRNA) technology and best-in-class lipid nanoparticle (LNP) delivery has the potential to deliver a pipeline in a product and treat multiple diseases and therapeutic areas. The data to be presented will highlight the ability of our platform to enable sustained pharmacodynamic effects at low doses, durable protein expression, and repeat dosing. We look forward to advancing our CD19 autoimmune panCAR program into the clinic in 2026, followed by our BCMA panCAR program in oncology."

Presentation details are as follows:

Title: In Vivo panCAR Therapy Using Circular RNA for the Treatment of Autoimmune Disease

Speaker: Megan Hoban, Ph.D., panCAR Program Lead, Orna Therapeutics

Date/Time: Thursday, May 15, 2025, 8:00 AM – 9:45 AM CDT

Session Name: Cellular and Gene Therapies for Autoimmune Disease

Location: Room 388-390

By leveraging its leading oRNA technology and potentially best-in-class LNP delivery, Orna’s in vivo CD19 panCAR platform holds the potential to benefit patients across multiple B cell driven autoimmune diseases. New data to be presented at ASGCT (Free ASGCT Whitepaper) will highlight the ability of Orna’s CD19 panCAR platform to generate deep and sustained B cell depletion in non-human primates across multiple doses.

ORIC® Pharmaceuticals Reports First Quarter 2025 Financial Results and Operational Updates

On May 5, 2025 ORIC Pharmaceuticals, Inc. (Nasdaq: ORIC), a clinical stage oncology company focused on developing treatments that address mechanisms of therapeutic resistance, reported financial results and operational updates for the quarter ended March 31, 2025 (Press release, ORIC Pharmaceuticals, MAY 5, 2025, View Source [SID1234652515]).

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"In the first quarter, we made significant progress across our pipeline, announced focused registrational development plans for our two lead programs, extended our cash runway, and accelerated key corporate milestones," stated Jacob M. Chacko, M.D., president and chief executive officer. "Looking ahead, we expect to share multiple clinical data updates across both programs over the next fifteen months. We remain on track to initiate the first Phase 3 trial of ORIC-944 in mCRPC in the first half of 2026, with registrational development of ORIC-114 in first-line NSCLC expected to begin later that year."

First Quarter 2025 and Other Recent Highlights

ORIC-944: a potent and selective allosteric inhibitor of PRC2

Reported encouraging early safety and efficacy data in ongoing dose escalation trial for ORIC-944 in combination with apalutamide in patients with metastatic castration resistant prostate cancer (mCRPC).
Presented preclinical ORIC-944 data demonstrating synergistic activity and improved progression-free survival (PFS) when combined with androgen receptor pathway inhibitors (ARPIs) in models of prostate cancer at the 2025 AACR (Free AACR Whitepaper) Annual Meeting.
Announced updated program milestones and development plans to initiate first Phase 3 registrational trial for ORIC-944 in mCRPC in 1H 2026.
ORIC-114: a brain penetrant, orally bioavailable, irreversible EGFR/HER2 inhibitor

Announced a clinical trial collaboration and supply agreement with Johnson & Johnson and initiated a trial to evaluate ORIC-114 in combination with subcutaneous (SC) amivantamab for the 1L treatment of patients with non-small cell lung cancer (NSCLC) harboring EGFR exon 20 insertion mutations.
Announced updated program milestones and registrational development plans to focus ORIC-114 in 1L NSCLC and plans to initiate first Phase 3 trial in 2026.
Corporate Highlights:

Extended projected cash runway into 2027 (from previous guidance of late 2026), and accelerated/augmented corporate milestones, based upon favorable enrollment and focused registrational clinical development plans for two lead programs.
Anticipated Program Milestones:

ORIC anticipates the following upcoming milestones:

ORIC-944 (mCRPC):
1H 2025: Combination dose escalation data with AR inhibitors(s)
2H 2025: Updated combination dose escalation data with AR inhibitors(s)
4Q 2025 / 1Q 2026: Combination dose optimization data with AR inhibitor(s)
ORIC-114 (NSCLC):
2H 2025: 1L EGFR exon 20, 2L EGFR exon 20, 2L+ HER2 exon 20 and 2L+ EGFR atypical data
Mid-2026: 1L EGFR exon 20 combination with SC amivantamab and 1L EGFR atypical data
First Quarter 2025 Financial Results

Cash, Cash Equivalents and Investments: Cash, cash equivalents and investments totaled $223.8 million as of March 31, 2025, which is expected to fund the current operating plan into 2027.
R&D Expenses: Research and development (R&D) expenses were $24.6 million for the three months ended March 31, 2025, compared to $22.0 million for the three months ended March 31, 2024, an increase of $2.7 million. The increase was due to a net increase in external expenses related to the advancement of product candidates, as well as higher personnel costs, including additional non-cash stock-based compensation.
G&A Expenses: General and administrative (G&A) expenses were $8.1 million for the three months ended March 31, 2025, compared to $7.0 million for the three months ended March 31, 2024, an increase of $1.0 million. The increase was primarily due to higher personnel costs, including additional non-cash stock-based compensation.

Nanobiotix Announces Full Results From Completed Phase 1 Study Evaluating JNJ-1900 (NBTXR3) in Pancreatic Cancer

On May 5, 2025 NANOBIOTIX (Euronext: NANO –– NASDAQ: NBTX – the ‘‘Company’’), a late-stage clinical biotechnology company pioneering nanotherapeutic approaches to improve treatment outcomes for patients with cancer, reported the presentation of full results from the completed dose escalation and dose expansion phases of a Phase 1 study evaluating JNJ-1900 (NBTXR3) in patients with locally advanced or borderline resectable pancreatic cancer (Press release, Nanobiotix, MAY 5, 2025, View Source [SID1234652514]). The study, conducted by The University of Texas MD Anderson Cancer Center ("MD Anderson"), was presented by principal investigator Dr. Eugene Koay at the 2025 Annual Meeting of the European Society for Radiotherapy and Oncology (ESTRO 2025).

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Pancreatic ductal adenocarcinoma (PDAC) remains one of the most lethal malignancies, driven by aggressive tumor biology and limited responsiveness to standard therapies. For patients with locally advanced ("LAPC") or borderline resectable ("BRPC") disease, the current standard-of-care ("SOC")—induction chemotherapy followed by chemoradiation—rarely delivers curative outcomes, underscoring the need for novel treatment approaches.

"Patients with locally advanced or borderline resectable pancreatic cancer face a particularly urgent unmet need for therapeutic innovation that can provide a meaningful survival benefit with an acceptable safety profile," said Eugene Koay, MD, PhD, Associate Professor of Radiation Oncology at MD Anderson. "We are encouraged by the results from the completed cohorts and look forward to the continued evaluation of JNJ-1900 (NBTXR3) in combination with standard-of-care chemoradiation after induction chemotherapy."

PRESENTATION #E25-2265: NANORAY Pancreas: A Phase 1 Study of NBTXR3 (JNJ-1900) Activated by Radiotherapy for Locally Advanced or Borderline Resectable Pancreatic Cancer (LAPC or BRPC)
Koay EJ, Liu S, Guerrero P, Stokes E, Katz MHG, Ikoma N, Snyder RA, Tzeng CD, Overman MJ, Pant S, Wolff RA, Javle M, Holliday EB, Ludmir EB, Das P, Noticewala S, Koong AC, Tamm EP, Bhutani M

This MD Anderson-sponsored Phase 1 study evaluated the potential of radiotherapy("RT")-activated JNJ-1900 (NBTXR3) activated by radiation therapy (45 Gy in 15 fractions) to overcome inherent radioresistance in patients with LAPC or BRPC. The majority of patients in the study (20/22) were diagnosed with locally advanced, unresectable disease (LAPC). For clarity, patients with LAPC or BRPC are traditionally treated with induction chemotherapy followed by concurrent chemoradiation. The treatment regimen in the completed dose escalation and dose expansion parts of this Phase 1 study replaced concurrent chemoradiation with RT-activated JNJ-1900 (NBTXR3) after induction chemotherapy.

Key Results:

Favorable safety profile and injection feasibility were observed (n=22)
Median overall survival ("mOS"): 23 months from diagnosis [95% CI; 17 months – not reached]
For context, an MD Anderson historical review of 144 patients with LAPC treated at the same center showed a mOS of 19.2 months. Patients in the historical review received induction chemotherapy followed by RT with or without concurrent or maintenance chemotherapy (80% received RT with concurrent chemotherapy)
Median local progression-free survival ("mLPFS"): 13.3 months from completion of radiation
Two LAPC patients achieved R0 surgical resection
Exploratory Biomarker Analyses:

Of the 20 patients for whom circulating Tumor Mutational Burden (cTMB) data was available, a notable proportion (40%; 8/20) exhibited increased cTMB, and investigators observed an association between increased cTMB and improved LPFS and OS
Normalization of CA19-9, a surrogate for overall survival benefit, was observed in 59% of patients (11/22) and was associated with longer survival in the study.
For context, an MD Anderson historical review of 243 patients with LAPC treated at the same center showed normalization of CA19-9 in approximately 17% of patients treated with the standard of care who had elevated CA19-9 levels at diagnosis
Based on the safety and preliminary efficacy findings, investigators concluded that further evaluation of JNJ-1900 (NBTXR3) is warranted in a randomized study.

"Our collaboration with MD Anderson has always been driven by a shared commitment to exploring bold new approaches for patients with high unmet need," said Louis Kayitalire, MD, Chief Medical Officer at Nanobiotix. "Given the extremely poor survival rates in LAPC and BRPC, the results from this Phase 1 study give us confidence in the potential of JNJ-1900 (NBTXR3) to serve as a meaningful addition to the treatment landscape. We are particularly excited about the potential to further enhance outcomes through combination of JNJ-1900 (NBTXR3) with SOC chemoradiation in the study’s new active cohort, and we look forward to advancing this program in pancreatic cancer."

MD Anderson received FDA clearance to expand the study to include a new cohort that combines of JNJ-1900 (NBTXR3) with SOC concurrent chemoradiation after induction chemotherapy. The first patient in the new cohort has been injected, and recruitment is ongoing.