Deciphera Pharmaceuticals Announces Nature Medicine Publication of Results from Exploratory ctDNA Analysis from INTRIGUE Phase 3 Study Demonstrating Substantial Clinical Benefit of QINLOCK® in 2L GIST Patients with Mutations in KIT Exon 11 and 17/18

On January 5, 2024 Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer, reported that Nature Medicine has published results from a circulating tumor DNA (ctDNA) analysis of the INTRIGUE Phase 3 study of QINLOCK (ripretinib) in GIST patients with mutations in KIT exon 11 and 17/18 only previously treated with imatinib (Press release, Deciphera Pharmaceuticals, JAN 5, 2024, View Source [SID1234639007]).

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The article, titled "Ripretinib versus sunitinib in gastrointestinal stromal tumor: ctDNA biomarker analysis of the phase 3 INTRIGUE trial" is now available online and will be published in a future print issue of Nature Medicine.

"The results published in Nature Medicine provide compelling evidence that QINLOCK may provide progression-free and overall survival benefit to second-line (2L) GIST patients in whom a liquid biopsy reveals primary KIT exon 11 mutations plus secondary mutations restricted to KIT exons 17 and 18. It is the first test that measures heterogeneity of resistance and may allow for a more optimized and targeted treatment plan for people living with this disease," said Sebastian Bauer, M.D., Medical Oncologist at the West German Cancer Center in Essen and senior author of the manuscript. "This analysis is leading us to consider a new approach in GIST treatment using sensitive and minimally invasive blood tests to identify the specific secondary mutational profile for individual patients in order to tailor their therapy based on the differential activity of QINLOCK and sunitinib seen in the INTRIGUE subgroup analysis."

"In second-line GIST patients with KIT exon 11 + 17/18 mutations only, treatment with QINLOCK resulted in a 78% reduction in the risk of disease progression and a 66% reduction in the risk of death compared to sunitinib, representing a substantial clinical benefit for these patients," said Matthew L. Sherman, M.D., Chief Medical Officer of Deciphera. "Our ongoing INSIGHT pivotal Phase 3 study is designed to confirm the exceptional efficacy we observed in this exploratory analysis from INTRIGUE. The INSIGHT study is now open at multiple sites and we are committed to enrolling the study as quickly as possible."

INTRIGUE is an international, multi-center study conducted in 122 active sites across 22 countries, where 453 patients in the all patient intent-to-treat population (AP-ITT) with second-line GIST were randomized to receive ripretinib (n=226) or sunitinib (n=227).

In the AP-ITT population, QINLOCK demonstrated similar efficacy with a median progression-free survival (PFS) of 8.0 months versus 8.3 months for sunitinib (HR 1.05, nominal p=0.72). There were fewer patients with Grade 3-4 drug-related treatment emergent adverse events (TEAE) with QINLOCK (26.5%) compared with sunitinib (55.2%). Based on the primary results from the INTRIGUE study, QINLOCK was included in the National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology (version 1.2023) as the preferred second-line regimen for patients with advanced GIST who are intolerant to sunitinib.

A prespecified exploratory objective in INTRIGUE was to evaluate anti-tumor efficacy of QINLOCK according to baseline KIT primary and secondary mutation status. Baseline peripheral whole blood was analyzed by Guardant360, a 74-gene ctDNA next-generation sequencing liquid biopsy assay in patients for whom evaluable samples were available (n=362) out of whom 280 patients had detectable ctDNA. In patients with a detectable KIT exon 11 primary mutation (n=157), 52 patients also had mutations in KIT exon 17/18 only and 41 had mutations in KIT exon 13/14 only.

Patients with mutations in KIT exon 11 and 17/18 only had improved progression-free survival (PFS), objective response rate (ORR), and overall survival (OS) with QINLOCK versus sunitinib while patients with mutations in KIT exon 11 and 13/14 only had improved PFS, ORR, and OS with sunitinib compared to QINLOCK.

Summary of INTRIGUE Efficacy Results of ctDNA Analysis for Patients with Mutations in KIT Exon 11 and 17/18 Only

Ripretinib

(n=27)

Sunitinib

(n=25)

Hazard
Ratio/Response
Difference
(95% CI)

Median Progression-Free Survival (1)

14.2 months

1.5 months

0.22 (0.11, 0.44), nominal p value <0.0001

Objective Response Rate (1)

44.4%

0%

44.4% (23.0%, 62.7%)

nominal p value = 0.0001

Overall Survival (2)

Not Estimable

17.5 months

0.34 (0.15, 0.76), nominal p value = 0.0061

Notes: (1) Data cutoff as of September 1, 2021; (2) Data cutoff as of September 1, 2022.

The subgroup safety profile was consistent with the primary analysis in the AP-ITT population and demonstrated a more favorable safety profile for QINLOCK compared with sunitinib with fewer patients experiencing Grade 3-4 drug-related TEAEs (KIT exon 11 and 17/18 only: 33.3% for QINLOCK versus 50.0% for sunitinib).

About the INSIGHT Study

The INSIGHT Phase 3 clinical study is a randomized, global, multicenter, open-label study to evaluate the efficacy and safety of QINLOCK compared to sunitinib in patients with GIST previously treated with imatinib with mutations in KIT exon 11 and 17/18 only (excluding patients with mutations in KIT exons 9, 13, or 14). In the study, 54 patients will be randomized 2:1 to either QINLOCK 150 mg once daily or sunitinib 50 mg once daily for four weeks followed by two weeks without sunitinib. The primary endpoint is PFS as determined by independent radiologic review using modified RECIST 1.1 criteria. Secondary endpoints include ORR as determined by independent radiologic review using modified RECIST 1.1 criteria and OS.

About the INTRIGUE Study

The INTRIGUE Phase 3 clinical study is a randomized, global, multicenter, open-label study to evaluate the efficacy and safety of QINLOCK compared to sunitinib in patients with GIST previously treated with imatinib. In the study, 453 patients were randomized 1:1 to either QINLOCK 150 mg once daily or sunitinib 50 mg once daily for four weeks followed by two weeks without sunitinib. As previously reported, the study did not achieve the primary efficacy endpoint of PFS as determined by independent radiologic review using modified RECIST 1.1 criteria. The statistical analysis plan included a hierarchical testing sequence that included testing patients with a KIT exon 11 primary mutation and then in the all patient intent-to-treat (AP-ITT) population. In patients with a KIT exon 11 primary mutation (n=327), QINLOCK demonstrated a median PFS of 8.3 months compared to 7.0 months for the sunitinib arm (HR 0.88, p=0.360). Although not formally tested due to the rules of the hierarchical testing sequence, in the AP-ITT population QINLOCK demonstrated a median PFS of 8.0 months compared to 8.3 months for the sunitinib arm (HR 1.05, nominal p=0.72). QINLOCK was generally well tolerated. Fewer patients in the QINLOCK arm experienced Grade 3-4 treatment-emergent adverse events compared to sunitinib (41.3% vs. 65.6%). Similarly, there were fewer patients with Grade 3-4 drug-related TEAEs with ripretinib (26.5%) compared with sunitinib (55.2%).

Curis Reports Inducement Grants Under NASDAQ Listing Rule 5635(c)(4)

On January 5, 2024 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported that on January 2, 2024, the independent Compensation Committee of the Board of Directors of Curis approved the grant of inducement stock options to purchase a total of 5,800 shares of Curis common stock to a new employee, with a grant date of January 2, 2024 (the "Q1 2024 Inducement Grant") (Press release, Curis, JAN 5, 2024, View Source [SID1234639006]).

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The Q1 2024 Inducement Grant has an exercise price per share equal to the closing price of the Company’s common stock on January 2, 2024. The stock option has a 10 year term and vests over four years, with 25% of the original number of shares underlying the award vesting on the first anniversary of the employee’s date of hire and an additional 6.25% of the original number of shares underlying the award vesting on each successive three-month period thereafter, subject to the employee’s continued service with the Company through the respective vesting dates. The stock option was granted as an inducement equity award outside of the Company’s Fourth Amended and Restated 2010 Stock Incentive Plan and was made as an inducement material to the employee’s acceptance of employment with the Company.

Bexion Pharmaceuticals, Inc. to Present at Biotech Showcase 2024

On January 5, 2024 Bexion Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing a new generation of biologic therapy to treat solid tumor cancers and chemotherapy-induced peripheral neuropathy (CIPN), reported that the Company will present at Biotech Showcase 2024 during the J.P. Morgan 42nd Annual Healthcare Conference week (Press release, Bexion, JAN 5, 2024, View Source [SID1234639005]). Biotech Showcase 2024 will be held in-person, from January 8-10, 2024, in San Francisco, CA.

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Presentation Details:
Date: Monday, January 8, 2024
Time: 3:00 pm PT
Location: Hilton San Francisco – Union Square
Track: Franciscan A (Ballroom Level)

Scott Shively, CEO and President of Bexion Pharmaceuticals, and Joyce LaViscount, Chief Financial Officer of Bexion Pharmaceuticals, will attend the conference. Additionally, the Bexion management team will host one-on-one meetings with potential investors in San Francisco from January 8-11, 2024. If you would like to schedule a meeting, please reach out to the Company’s Investor Contact below.

Corporate Overview

On January 5, 2024 AnaptysBio presented its corporate presentation (Presentation, AnaptysBio, JAN 5, 2024, View Source [SID1234639004]).

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ADC Therapeutics Provides Business Updates

On January 4, 2024 ADC Therapeutics reported business updates (Press release, ADC Therapeutics, JAN 4, 2024, View Source [SID1234642026]).

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"During 2023, we took a number of decisive actions to help position the Company for success in 2024 and beyond. We prioritized our pipeline, strengthened our organization and implemented a disciplined capital allocation model to generate cost efficiencies," said Ameet Mallik, Chief Executive Officer of ADC Therapeutics. "We believe we are starting to see signs of the commercial turnaround. We are also encouraged to see positive initial signals in the LOTIS-7 trial of ZYNLONTA in combination with bispecifics as well as early signs of antitumor activity in the Phase 1b trial of ADCT-601. We now expect our cash runway to extend into the fourth quarter of 2025 and believe we are on a path to unlock the substantial value in the Company."

Recent Highlights and Developments

ZYNLONTA (loncastuximab tesirine-lpyl)

ZYNLONTA net sales for the fourth quarter of 2023 are expected to be approximately $16.5 million.
The Phase 1 LOTIS-7 trial of ZYNLONTA in combination with bispecifics glofitamab or mosunetuzumab for the treatment of patients with diffuse large B-cell lymphoma (DLBCL), follicular lymphoma (FL) and marginal zone lymphoma (MZL) is actively enrolling patients. The dose-limiting toxicity (DLT) period has been cleared for the first dosing level of ZYNLONTA 90 µg/kg in both arms, and there have been no discontinuations due to adverse events (AEs). To date, each of the first five patients eligible for assessment in this dosing level has shown a response (partial response or complete response) at first scan.​
An oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2023 Annual Meeting from the University of Miami investigator-initiated trial exploring ZYNLONTA in combination with rituximab in high-risk relapsed or refractory FL indicated a best overall response rate of 96.3% and a complete response rate of 85.2%​. After a median follow-up of 9.7 months, the median progression-free survival (PFS) was not reached, and the 12-month PFS was 92.3%​. The majority of AEs were grade 1. Grade 3 AEs included neutropenia (n=2; 6.2%), and one case each (3.1%) of hyperglycemia, increased ALT, fatigue, dyspnea and skin infection. Neutropenia was the only grade 4 AE (n=1; 3.1%).
Pipeline

ADCT-601 (targeting AXL): In the Phase 1b trial, the maximum-tolerated dose has been reached, and the study is currently in dose optimization. There have been early signs of antitumor activity in both monotherapy and in combination. The dose-optimization/ expansion phase is comprised of a monotherapy arm including patients with sarcoma, pancreatic cancer and AXL-expressing non-small cell lung cancer (NSCLC) and a combination arm with gemcitabine in patients with sarcoma and pancreatic cancer.
ADCT-901 (targeting KAAG1): The Company has decided to discontinue this program due to limited signs of efficacy in the dose escalation phase and to reallocate capital to prioritized programs.
ADCT-602 (targeting CD22): Dose escalation and expansion in the Phase 1 trial in collaboration with MD Anderson Cancer Center for patients with relapsed or refractory acute lymphoblastic leukemia is progressing, and additional clinical trial sites are being added to accelerate enrollment.
Early-stage pipeline: The Company is advancing a portfolio of investigational ADCs including those targeting Claudin-6, NaPi2b and PSMA. These candidates utilize exatecan with a novel hydrophilic linker as a highly potent and differentiated payload.
Balance Sheet
The Company ended the fourth quarter of 2023 with cash and cash equivalents of ~$278.5 million.

Guidance
The Company expects the following based on its current business plan:

Decrease in total operating expenses expected in full year 2023 and 2024 as compared to 2022
Cash runway expected into 4Q 20252 (previously: mid-2025)
Expected Milestones in 2024

ZYNLONTA

Achieve commercial brand profitability in 2024
LOTIS-5: Complete enrollment in 2024
LOTIS-7: Additional safety and efficacy data from the dose-escalation and dose-expansion portions of the Phase 1 study in 2024
Pipeline

ADCT-601 (targeting AXL)

Additional data updates from the Phase 1 study in patients with sarcoma, pancreatic cancer and NSCLC in 2024
ADCT-602 (targeting CD22)

Additional data from Phase 1 study in 2024
Please refer to the Company’s Form 8-K and accompanying presentation filed with the Securities and Exchange Commission today for additional information.

About ZYNLONTA (loncastuximab tesirine-lpyl)

ZYNLONTA is a CD19-directed antibody drug conjugate (ADC). Once bound to a CD19-expressing cell, ZYNLONTA is internalized by the cell, where enzymes release a pyrrolobenzodiazepine (PBD) payload. The potent payload binds to DNA minor groove with little distortion, remaining less visible to DNA repair mechanisms. This ultimately results in cell cycle arrest and tumor cell death.

The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have approved ZYNLONTA (loncastuximab tesirine-lpyl) for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (NOS), DLBCL arising from low-grade lymphoma and also high-grade B-cell lymphoma. The trial included a broad spectrum of heavily pre-treated patients (median three prior lines of therapy) with difficult-to-treat disease, including patients who did not respond to first-line therapy, patients refractory to all prior lines of therapy, patients with double/triple hit genetics and patients who had stem cell transplant and CAR-T therapy prior to their treatment with ZYNLONTA. This indication is approved by the FDA under accelerated approval and in the European Union under conditional approval based on overall response rate and continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Please see full prescribing information including important safety information about ZYNLONTA at www.ZYNLONTA.com.

ZYNLONTA is also being evaluated as a therapeutic option in combination studies in other B-cell malignancies and earlier lines of therapy.