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]).

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"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 PTC Therapeutics, Inc., (NASDAQ: PTCT) reported a corporate update and financial results for the first quarter ended March 31, 2025 (Press release, PTC Therapeutics, MAY 6, 2025, View Source [SID1234652592]).

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"Following a year of outstanding execution across every part of the Company, we have built on this positive momentum with solid revenue performance in the first quarter, allowing us to narrow our full-year revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. "Our strong cash balance supports all of our planned commercial and R&D activities and provides the ability to reach cashflow breakeven without raising additional capital. The positive CHMP opinion for Sephience kickstarts our anticipated global launch for what we see as a significant revenue opportunity."

Key Corporate Updates:

First quarter 2025 total net product and royalty revenue of $190 million.
First quarter 2025 revenue for the DMD franchise of $134 million, including net product revenue for Translarna of $86 million and for Emflaza of $48 million.
Key Clinical and Regulatory Milestones:

Sephience
Positive CHMP opinion on Sephience MAA for adult and pediatric PKU patients received on April 25, 2025; the opinion includes a broad label inclusive of all ages and disease severities. PTC expects the EC to adopt the opinion in approximately two months
NDA currently under review by the FDA, with a target regulatory action date of July 29, 2025
Japan regulatory submission under review and a decision is expected in Q4 2025
Vatiquinone
NDA for pediatric and adult patients with Friedreich’s ataxia accepted and granted Priority Review by the FDA, with a target regulatory action date of August 19, 2025
Translarna
NDA currently under review by the FDA
PTC518
Phase 2 PIVOT-HD study results announced on May 5, 2025:
Met primary endpoint of dose-dependent blood HTT lowering at Week 12
Dose-dependent trends of clinical benefit in Stage 2 patients at Month 12
Signals of dose-dependent clinical benefit relative to matched natural history cohort and dose-dependent lowering of NfL in Stage 2 subjects at Month 24
Continued favorable safety and tolerability with no treatment-related NfL spikes
Plans to complete additional analyses and discuss next development and regulatory steps, including potential for accelerated approval
First Quarter 2025 Financial Highlights:

Total net product and royalty revenue was $189.9 million for the first quarter of 2025, compared to $208.8 million for the first quarter of 2024.
Total revenue includes net product revenue across the commercial portfolio of $153.4 million for the first quarter of 2025, compared to $177.6 million for the first quarter of 2024. Total revenue also includes royalty, collaboration and license, and manufacturing revenue of $1,022.7 million for the first quarter of 2025, compared to $32.5 million for the first quarter of 2024. The first quarter of 2025 collaboration and license revenue includes $986.2 million, related to the PTC518 license and collaboration agreement with Novartis, which closed in January 2025.
Translarna net product revenues were $86.2 million for the first quarter of 2025, compared to $103.6 million for the first quarter of 2024.
Emflaza net product revenues were $47.8 million for the first quarter of 2025, compared to $57.5 million for the first quarter of 2024.
Roche reported Evrysdi full year 2025 sales of approximately 420 CHF million, resulting in royalty revenue of $36.4 million to PTC for first quarter 2025, as compared to $31.2 million for first quarter 2024.
Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $109.0 million for the first quarter of 2025, compared to $116.1 million for the first quarter of 2024.
Non-GAAP R&D expenses were $100.3 million for the first quarter of 2025, excluding $8.7 million in non-cash, stock-based compensation expense, compared to $107.2 million for the first quarter of 2024, excluding $9.0 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $81.0 million for the first quarter of 2025, compared to $73.3 million for the first quarter of 2024.
Non-GAAP SG&A expenses were $71.6 million for the first quarter of 2025, excluding $9.4 million in non-cash, stock-based compensation expense, compared to $63.9 million for the first quarter of 2024, excluding $9.4 million in non-cash, stock-based compensation expense.
Net income was $866.6 million for the first quarter of 2025, compared to net loss of $91.6 million for the first quarter of 2024.
Cash, cash equivalents, and marketable securities were $2,027.2 million as of March 31, 2025, compared to $1,139.7 million as of December 31, 2024.
Shares issued and outstanding as of March 31, 2025 were 79,225,276.
PTC Updates Full-Year 2025 Financial Guidance:

PTC now anticipates full-year 2025 revenue to be between $650 million and $800 million, which includes in-line products, potential new product launches, and royalty revenue from Evrysdi.
PTC anticipates full-year 2025 GAAP R&D and SG&A expense to be between $805 and $835 million.
PTC anticipates full-year 2025 non-GAAP R&D and SG&A expense to be between $730 and $760 million, excluding estimated non-cash, stock-based compensation expense of $75 million.

On May 6, 2025 Intensity Therapeutics, Inc. (Nasdaq: INTS), ("Intensity" or "the Company") a late-stage clinical biotechnology company focused on the discovery and development of proprietary, novel immune-based intratumoral cancer therapies designed to kill tumors and increase immune system recognition of cancers, and The Swiss Group for Clinical Cancer Research SAKK ("SAKK"), a decentralized academic research institute that has been conducting clinical trials of cancer treatments in all major Swiss hospitals since 1965, reported that the European Medicines Agency ("EMA") has authorized the initiation of the INVINCIBLE-4 (SAKK 66/22) ("INVINCIBLE-4 Study") (NCT06358573) in France in collaboration with Unicancer (Press release, Intensity Therapeutics, MAY 6, 2025, View Source [SID1234652609]).

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The INVINCIBLE-4 Study is a randomized open-label, multicenter study to determine the clinical activity, safety, and tolerability of INT230-6 in patients with early-stage, operable triple-negative breast cancer ("TNBC") who undergo standard of care neoadjuvant immunochemotherapy ("SOC") treatment and SOC alone. The primary endpoint is pathological complete response ("pCR") in the primary tumor and affected lymph nodes. Patients will be randomized one-to-one to receive a regimen of either two doses of INT230-6 followed by SOC, which consists of pembrolizumab, anthracyclines, carboplatin, cyclophosphamide, and paclitaxel (i.e., the Keynote-522 regimen), or the SOC alone. The study is already recruiting patients in Switzerland and is expected to enroll 54 patients.

"We are encouraged to see high levels of tumor necrosis from the MRI scans and evidence of tumor inflammation after two INT230-6 injections and prior to initiation of the SOC in our first patients," said Ursina Zürrer, M.D. Chief Physician for Genetic Counseling, Department of Medical Oncology and Hematology Cantonal Hospital Winterthur, Switzerland, and the Coordinating Investigator for the INVINCIBLE-4 Study. "If the immunological cancer cell death and the ignition of an anti-cancer immune response without increased toxicity in patients receiving INT230-6 shows a meaningful increase in pCR, it would be a major advance for the neoadjuvant treatment of breast cancer and potentially other cancers."

"The acceptance of the INVINCIBLE-4 by the EMA and our expansion of the trial into France is expected to increase our enrollment rate starting in the second quarter of 2025. We should almost double the number of sites actively screening patients. We are excited to work with Unicancer, a group accredited by the French National Cancer Institute with centers of academic excellence and strong operational capability throughout France." Said Lewis H. Bender, President and CEO of Intensity Therapeutics."

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

About Triple Negative Breast Cancer in the Presurgical Setting
Women with aggressive forms of breast cancer, such as TNBC, are often counseled to undergo pre-surgical (neoadjuvant) systemic therapy in advance to reduce the risk of the disease returning. Having a pathological complete response, meaning the absence of live cancer at the time of surgery, has been shown to result in a lower risk of recurrence. Approximately 11-17% of breast cancers test negative for estrogen receptors (ER), progesterone receptors (PR), and overexpression of human epidermal growth factor receptor 2 (HER2) protein, qualifying them as triple negative. There are approximately 56,000 new cases of TNBC in the US and 420,000 Worldwide diagnosed each year, the majority of which are local to the breast. TNBC is considered to be more aggressive and has a poorer prognosis than other types of breast cancer, because there are fewer available targeted medicines. Most patients with local TNBC typically receive immune/chemotherapy before surgery. Since the publication of Keynote-522, the standard neoadjuvant treatment for TNBC includes systemic chemotherapy (anthracyclines, cyclophosphamide, paclitaxel, carboplatin) and the anti-PD-1 monoclonal antibody pembrolizumab. pCR rates are 65%, with rates generally lower in the larger-sized tumors or with lymph node metastasis. The toxicity of the Keynote-522 regimen is high, with 80% of patients experiencing grade 3 or higher treatment-related AEs, including treatment-related adverse events that lead to death in 0.5% of patients.

On May 5, 2025 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company focused on delivering innovative immunology therapeutics, reported financial results for the first quarter ended March 31, 2025, and provided a business update (Press release, AnaptysBio, MAY 5, 2025, View Source [SID1234652496]).

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"Our lead program, rosnilimab, delivered impressive three-month Phase 2b efficacy, safety and tolerability data in rheumatoid arthritis (RA), with data through six months surpassing those of competitor all-active, head-to-head trials. We will report updated clinical and translational RA data in the first week of June, as well as initial Phase 2 ulcerative colitis (UC) data in Q4 2025, further defining rosnilimab’s game-changing potential," said Daniel Faga, president and chief executive officer of Anaptys. "With ANB033 and ANB101 progressing through Phase 1 trials, our autoimmune portfolio promises multiple catalysts over the next couple of years. We remain well-capitalized as we execute on our broad development plan for all three programs, while concurrently executing our $75 million stock repurchase program which are both further supported by substantial royalties and milestone payments anticipated from our GSK financial collaboration."

PORTFOLIO UPDATES

Rosnilimab (PD-1 depleter and agonist)

Announced in February that subcutaneously administered rosnilimab, including two once-monthly doses, achieved positive results in 424-patient Phase 2b RA trial and highest-ever reported clinical disease activity index (CDAI) low disease activity (LDA) response over 6 months
Full press release can be found here
Anaptys to host an investor call featuring Anaptys management and key opinion leaders in the first week of June to present rosnilimab’s updated Phase 2b clinical and translational data

Enrollment ongoing for global Phase 2 trial in moderate-to-severe UC
132-patient trial assessing two dose levels of subcutaneously administered rosnilimab vs. placebo (randomized 1:1:1)
Primary statistical analysis at Week 12 on well-established endpoints, including the primary endpoint of change from baseline in modified Mayo score (mMS) and supportive secondary endpoints of clinical response on mMS, clinical remission on mMS and endoscopic remission
All patients in all three study arms treat-through to Week 24 and remain blinded to treatment arm. Placebo-treated patients who achieved clinical response on partial modified Mayo score (pmMS) at Week 12 remain on placebo, while placebo-treated patients who are non-responders are crossed over to the high-dose rosnilimab treatment arm
Patients who are in clinical response on pmMS at Week 24 are eligible for an additional 26-week (50 weeks of total treatment) blinded treatment extension period (TEP)
Initial Phase 2 data anticipated in Q4 2025
ANB033 (CD122 antagonist)

Enrollment ongoing for Phase 1a trial in healthy volunteers
Phase 1b indication to be disclosed at H2 2025 R&D event
ANB101 (BDCA2 modulator)

Enrollment initiated for Phase 1a trial in healthy volunteers
COLLABORATION UPDATES

GSK Immuno-Oncology Financial Collaboration

GSK announced strong commercial performance for Jemperli ($220 million in Q1 2025 sales) with >15% quarter-over-quarter growth
GSK announced the EMA approval of Jemperli plus chemotherapy for all adult patients with primary advanced or recurrent endometrial cancer in January 2025

Anticipate receipt of a $75 million commercial sales milestone payment from GSK in either 2025 or 2026 once Jemperli achieves $1 billion in worldwide net sales in a calendar year

GSK anticipates top-line data in mid-2025 from COSTAR Lung Phase 3 trial in patients with advanced NSCLC who have progressed on prior anti-PD-(L)1 therapy and platinum-based chemotherapy comparing docetaxel alone to cobolimab, a TIM-3 antagonist, plus dostarlimab, a PD-1 antagonist, plus docetaxel and to dostarlimab plus docetaxel

Recent data published in The New England Journal of Medicine (NEJM) and presented at American Association for Cancer Research (AACR) (Free AACR Whitepaper) demonstrated neoadjuvant treatment with dostarlimab resulted in organ preservation in a high proportion of patients (80% of 103 patients), including 100% (rectal; n=49), 100% (bladder; n=6), and 82% (colon; n=22) complete responses in April 2025
GSK anticipates top-line data in 2026 from AZUR-1 pivotal Phase 2 trial of dostarlimab monotherapy in patients with untreated stage II/III dMMR/MSI-H locally advanced rectal cancer
Jemperli received U.S. FDA Breakthrough Therapy Designation for this indication in December 2024
Vanda Imsidolimab Collaboration

Announced an exclusive $15 million global out-license agreement with Vanda Pharmaceuticals to develop and commercialize imsidolimab (IL-36R antagonist), with Anaptys eligible to receive up to $35 million for future regulatory approvals and sales milestones, in addition to 10% royalty on global net sales
FDA BLA submission for generalized pustular psoriasis (GPP) expected in 2025
Full press release can be found here
FINANCIAL UPDATES

Stock Repurchase Program and Cash Runway

Authorized a Stock Repurchase Program in March 2025 of $75.0 million of the Company’s outstanding common stock

Cash and investments of $383.0 million as of March 31, 2025, and reiterating cash runway through year-end 2027
First Quarter 2025 Financial Results

Cash, cash equivalents and investments totaled $383.0 million as of March 31, 2025, compared to $420.8 million as of December 31, 2024, for a decrease of $37.8 million due primarily to operating activities and $4.4 million in shares repurchased offset by $15.0 million received from Vanda Pharmaceuticals for the license of imsidolimab.

Collaboration revenue was $27.8 million for the three months ended March 31, 2025, compared to $7.2 million for the three months ended March 31, 2024. The increase is due to a $11.0 million increase in royalties recognized for sales of Jemperli and $9.6 million in revenue recognized for the Vanda license agreement.

Research and development expenses were $41.2 million for the three months ended March 31, 2025, compared to $37.0 million for the three months ended March 31, 2024. The increase was due primarily to development costs relating to the Phase 2 trials in RA and UC for rosnilimab, and the Phase 1 trials for ANB033 and ANB101, offset by a decrease in development costs for imsidolimab and ANB032. The R&D non-cash, stock-based compensation expense was $4.4 for the three months ended March 31, 2025 as compared to $3.5 million in the same period in 2024.

General and administrative expenses were $14.1 million for the three months ended March 31, 2025, compared to $12.3 million for the three months ended March 31, 2024. The increase was due primarily to transaction costs associated with the Vanda Pharmaceuticals license agreement. The G&A non-cash, stock-based compensation expense was $4.8 million for the three months ended March 31, 2025 as compared to $6.7 million in the same period in 2024.

Net loss was $39.3 million for the three months ended March 31, 2025, or a net loss per share of $1.28, compared to a net loss of $43.9 million for the three months ended March 31, 2024, or a net loss per share of $1.64.

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.