On December 1, 2022 Salarius Pharmaceuticals, Inc. (NASDAQ: SLRX), a clinical-stage biopharmaceutical company using targeted protein inhibition and targeted protein degradation to develop therapies for patients with cancer in need of new treatment options, reported interim clinical trial results from the company’s Phase 1/2 trial of its novel oral, reversible, targeted LSD1 inhibitor, seclidemstat, as a treatment for Ewing sarcoma and FET-rearranged sarcomas (Press release, Salarius Pharmaceuticals, DEC 1, 2022, View Source [SID1234624700]).

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These interim results appear to indicate that first- and second-relapse Ewing sarcoma patients treated with seclidemstat in combination with topotecan and cyclophosphamide who achieve disease control may have an increased time to tumor progression (TTP) compared with treatment of topotecan and cyclophosphamide alone, per the Phase 3 rEECur study described below. The Ewing sarcoma and FET-rearranged sarcoma trial is currently on a partial clinical hold as described below.

As of October 31, 2022, 13 first- and second-relapse Ewing sarcoma patients were evaluable for confirmed disease control rate assessment. Of these 13 patients, five patients (38%) achieved confirmed disease control with no tumor progression observed while treated with seclidemstat in combination with topotecan and cyclophosphamide. Treatment duration for the 13 patients ranged from 0.7 months to 13.8 months.

Five first-relapse Ewing sarcoma patients were treated and three (60%) achieved confirmed disease control, including one complete response, one partial response and one stable disease at 12.8 months and continuing. First-relapse patients had a median TTP of 7.4 months and treatment duration ranged from 1.4 months to 13.8 months.

At ASCO (Free ASCO Whitepaper) 2022, Euro Ewing Consortium presented rEECur2 Phase 3 study results in relapsed/refractory Ewing sarcoma patients that showed median event-free survival of 3.5 months in the topotecan/ cyclophosphamide arm (n=73) compared with 5.7 months in the high-dose ifosfamide arm (n=73)2. The rEECur data includes approximately 80% primary refractor or first-relapse. Ewing sarcoma patients after relapse have 5-year overall survival of about 13% and 10-year overall survival of about 9%3.

"Ewing sarcoma is a devastating bone and soft tissue cancer that affects children, adolescents and young adults, and treatment options primarily consist of chemotherapies, radiation and surgical resection," commented Damon Reed M.D., Program Leader, Adolescent Young Adult Program, Chair of Department of Individualized Cancer Management Moffitt Cancer Center and principal investigator in this sarcoma trial. "As an oncologist treating Ewing sarcoma patients, ultimately new treatments that extend a
1 Confirmed disease control rate includes complete remission, partial remission or stable disease confirmed by both cycle 2 and cycle 4 scans. Treatment cycles are 21 days in length, with cycle 2 scans occurring on or about day 42 and cycle 4 scans occurring on or about day 84.

2 rEECur – International Randomized Controlled Trial of Chemotherapy for the Treatment of Recurrent and Primary Refractory Ewing Sarcoma.
3 Risk of recurrence and survival after relapse in patients with Ewing sarcoma, Pediatric Blood & Cancer, Volume 57, Issue 4. First published October 2011.

David Arthur, President and CEO of Salarius, said, "These interim results are encouraging, and I believe they show the potential for seclidemstat to provide a more durable response among Ewing sarcoma patients when used in combination with topotecan and cyclophosphamide. This trial has more than 15 clinical trial sites with more than 20 locations throughout the United States, and when needed Salarius provides travel assistance to patients who want to participate in the sarcoma trial. It is our hope that the potential for seclidemstat to improve outcomes coupled with our support will make it easier for patients to participate when enrollment is restarted."

As previously reported, on October 18, 2022, enrollment of new patients in the Salarius-sponsored seclidemstat sarcoma clinical trial and the MD Anderson investigator-initiated hematologic clinical trial was voluntarily paused due to a suspected unexpected serious adverse reaction (SUSAR) observed in the sarcoma trial; patients currently enrolled in both studies are able to continue treatment after consulting with their physician. The U.S. Food and Drug Administration (FDA) subsequently agreed with Salarius’ approach and placed the sarcoma trial on partial clinical hold; Salarius is working with the FDA to further analyze the available data with the goal of understanding how best to proceed and restart enrollment.

Additional interim trial results include the following:
•Among the 13 Ewing sarcoma patients treated with seclidemstat, topotecan and cyclophosphamide
oThere was a 38% confirmed disease control rate and 1.6 months median TTP which ranged from 0.7 months to 13.8 months
oOne second relapse patient with confirmed stable disease withdrew from the study with no observed progression at 3.5 months due to a non-study related adverse event
oOne second relapse patient with a confirmed partial response withdrew from the study with no observed progression at 3.1 months
•Among the five first-relapse Ewing sarcoma patients treated, three (60%) achieved confirmed disease control
oOne patient achieved a complete response and withdrew from the study at 7.4 months
oOne patient achieved a partial response with 78% target lesion reduction; this patient subsequently elected radiation treatment as consolidation and withdrew from the study at 13.8 months
oOne patient achieved stable disease and continues on treatment after 12.8 months
•Single-agent activity has not been observed in the FET-rearranged sarcoma cohort of the trial
•More than 85 patients have been treated in either the dose-escalation or the dose-expansion portions of the trial with seclidemstat as a standalone therapy or in combination with standard-of-care chemotherapy

Conference call and webcast
Salarius plans to hold an investor call in mid-December to discuss these interim results and other clinical and preclinical data following presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting by MD Anderson Cancer Center and Salarius. Details about the conference call including how to participate will be provided in a separate news release.

About the Phase 1/2 Ewing’s and other FET-rearranged sarcomas trial
The Phase 1/2 trial currently is in its dose-expansion stage, which includes three patient arms. The first arm is planned to enroll up to 30 patients with Ewing’s sarcoma, a rare and deadly pediatric bone cancer, and will investigate seclidemstat in combination with topotecan and cyclophosphamide, a commonly used second- and third-line chemotherapy regimen. Salarius believes data released during ASCO (Free ASCO Whitepaper) 2021 demonstrated synergy in an Ewing’s sarcoma cell line when seclidemstat was used in combination with these agents. Salarius also believes this treatment combination and its use as a second- and third-line therapy has the potential to expand the addressable patient population for seclidemstat and improve outcomes by allowing physicians to introduce seclidemstat earlier in the Ewing’s sarcoma continuum of care.

The trial’s second patient arm is planned to investigate seclidemstat as a single agent in up to 15 patients with myxoid liposarcoma. The third patient arm is planned to investigate seclidemstat as a single agent in up to 15 patients with select sarcomas that share a similar biology to Ewing’s sarcoma, also referred to as FET-rearranged or Ewing’s-related sarcomas. In data released at ASCO (Free ASCO Whitepaper) 2021, a subset of patients with advanced FET-rearranged sarcomas treated with single-agent seclidemstat resulted in stable disease and prolonged time to progression, which Salarius believes suggests disease control, a clinically relevant endpoint for soft tissue sarcomas.

All three patient arms are designed to evaluate safety and efficacy in patients with advanced disease.

Salarius supports patient referrals to their sarcoma trial sites and provides travel assistance for clinical trial patients and families who are evaluating and participating in the sarcoma trial.

Clinical trial sites include Seattle Cancer Care Alliance (SCCA) – which is comprised of the Fred Hutchinson Cancer Research Center, Seattle Children’s Hospital and University of Washington Medical Center; Oregon Health & Sciences University Portland, OR; Johns Hopkins All Children’s Hospital in St. Petersburg, FL; Children’s Hospital of Los Angeles in Los Angeles, CA; Moffitt Cancer Center in Tampa, FL; Dana-Farber Cancer Institute in Boston, MA; MD Anderson Cancer Center in Houston, TX; Nationwide Children’s Hospital in Columbus, OH; Memorial Sloan Kettering Cancer Center in New York NY; the Sarcoma Oncology Center in Santa Monica, CA; Virginia Cancer Specialists, Fairfax, Virginia; Cleveland Clinic, in Cleveland, OH; Washington University, St. Louis, MO and Fox Chase Cancer Center in Philadelphia PA. Information on the Salarius Sarcoma trial SALA-002-EW16 (NCT03600649) can be found at clinicaltrial.gov.

On December 1, 2022 Roivant Sciences (Nasdaq: ROIV) and Pfizer Inc. (NYSE: PFE) reported formation of a new Vant to develop and commercialize PF-06480605 (now RVT-3101) (Press release, Roivant Sciences, DEC 1, 2022, View Source [SID1234624699]). RVT-3101 is a fully human monoclonal antibody targeting TL1A, which is currently in Phase 2b development in ulcerative colitis (UC). The Vant has the exclusive option to collaborate with Pfizer on a next-generation TL1A directed antibody which recently entered Phase 1.

RVT-3101 is a potential first-in-class agent that targets both inflammatory and fibrotic pathways by inhibiting TL1A, which has been shown to modulate the location and severity of inflammation and fibrosis by stimulating TH1 and TH17 pathways, in addition to activating fibroblasts. As such, RVT-3101 has the potential to provide greater efficacy by hitting multiple inflammatory pathways as well as fibrotic pathways.

RVT-3101 has been evaluated in an earlier Phase 2 study (TUSCANY) in 50 patients, and is being evaluated in a large global Phase 2b study (TUSCANY-2) in 245 adult participants with moderate to severe ulcerative colitis. The induction portion of TUSCANY-2 is complete, and the maintenance portion remains ongoing.

"Our internally discovered antibody against TL1A, could potentially be the first agent to bring biomarker-selected precision medicine to people living with inflammatory bowel disease," said Mikael Dolsten, Pfizer’s Chief Scientific Officer, President, Worldwide Research, Development and Medical. "We are very excited about the preliminary data from the TUSCANY-2 study and for this new Vant to drive the advancement of this asset. At the same time, it enables Pfizer to bring additional innovative breakthrough medicines and vaccines to patients in need more quickly, allowing us to serve more people."

"We believe in RVT-3101’s potential to transform the inflammatory bowel disease landscape, which has long been in need of new, innovative therapies with greater efficacy," said Mayukh Sukhatme, President and Chief Investment Officer of Roivant. "We are excited about this collaboration with Pfizer on a potential first-in-class program, which we intend to pursue in both ulcerative colitis and in additional inflammatory and fibrotic diseases. TUSCANY-2 builds on the earlier TUSCANY data with many firsts for the class – TUSCANY-2 is the first study with subcutaneous efficacy data and the first dose-ranging study – and is among the largest Phase 2b studies ever conducted in the indication. We are encouraged by the preliminary data and look forward to presenting our data and to seeing other results from the class."

Inflammatory bowel diseases are chronic inflammatory diseases of the gastrointestinal tract. It is estimated that up to 2 million US adults suffer from inflammatory bowel diseases, which include ulcerative colitis and Crohn’s disease. There is significant unmet treatment need for patients with inflammatory bowel disease. High rates of treatment failure mean that approximately 50% of patients are cycling off a given therapy within 6 to 12 months and are unlikely to achieve sustained remission. The commercial markets for these diseases for advanced therapies is nearly $15 billion per year in the US alone and growing. Available treatments have very low remission rates, and thus there is a large need for an efficacious and safe therapy.

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Terms of Collaboration

A new Vant or Roivant subsidiary has been created to develop and fund these programs. The Vant will be fully responsible for funding global development of RVT-3101 in UC and in additional inflammatory and fibrotic diseases and holds commercial rights in the US and Japan. Pfizer owns a 25% equity position in the Vant and maintains commercial rights outside of the US and Japan as well as representation on the company’s Board of Directors.

In addition, the Vant has the exclusive option to collaborate with Pfizer on a next-generation TL1A directed antibody which recently entered Phase 1. The Vant will have the right to enter into an agreement for global development with a 50/50 cost share as well as co-commercialization rights with Pfizer prior to Phase 2 (expected in 2025).

On December 1, 2022 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, reported that it has completed the previously announced acquisition of Renovacor, Inc. (NYSE: RCOR), a biotechnology company focused on delivering innovative precision therapies to improve the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases (Press release, Rocket Pharmaceuticals, DEC 1, 2022, View Source [SID1234624698]). Under the terms of the merger agreement entered into on September 19, 2022, Renovacor shareholders received, for each share of Renovacor common stock, 0.1763 shares of Rocket common stock.

The acquisition provides Rocket with Renovacor’s most advanced program, REN-001, an AAV-based gene therapy targeting BAG3-associated dilated cardiomyopathy (DCM), a severe form of heart failure. BAG3-DCM represents a significant unmet medical need in a patient population with rapidly progressive cardiac dysfunction in whom no treatments targeting the underlying mechanism of disease exist. Additionally, Rocket gains access to world-class scientific collaborators, a robust intellectual property portfolio and personnel with expertise in BAG3-DCM. These assets and capabilities altogether further extend Rocket’s leadership position in AAV-based cardiac gene therapy and help advance the Company’s goal of pursuing gene therapy cures for patients living with rare and devastating diseases.

Prior to market opening today, December 1, 2022, Renovacor shares ceased to trade on NYSE. Rocket’s common stock will continue to trade on Nasdaq under the ticker symbol RCKT.

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On December 1, 2022 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) reported that the U.S. Food and Drug Administration (FDA) has approved REZLIDHIA (olutasidenib) capsules for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test (Press release, Rigel, DEC 1, 2022, View Source [SID1234624697]). REZLIDHIA is an oral, small molecule, inhibitor of mutated IDH1 designed to bind to and inhibit mIDH1 to reduce 2-hydroxyglutarate levels and restore normal cellular differentiation of myeloid cells.

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"REZLIDHIA is a novel, non-intensive monotherapy treatment in the relapsed/refractory AML setting demonstrating a CR+CRh rate of 35% in patients with over 90% of those responders in complete remission. The 25.9 months median duration of CR+CRh is a clinically meaningful improvement for AML patients and appears to be longer than currently available treatment options," said Jorge E. Cortes, M.D., Director, Georgia Cancer Center, Cecil F. Whitaker Jr., GRA Eminent Scholar Chair in Cancer, and Phase 2 trial investigator. "Given the limited treatment options for adult patients with mIDH1 R/R AML, who typically have a poor prognosis, REZLIDHIA may provide an effective, new treatment option with a well characterized safety profile."

The FDA approval was supported by data from the open-label Phase 2 registrational study evaluating REZLIDHIA monotherapy at a dose of 150 mg twice daily in 153 mIDH1 R/R AML patients. The efficacy-evaluable population was 147 patients who initiated REZLIDHIA at least six months prior to the interim analysis cutoff date of June 18, 2021, and who had a centrally confirmed IDH1 mutation. The primary endpoint was a composite of a complete remission (CR) plus a complete remission with partial hematological recovery (CRh). CRh is defined as less than 5% blasts in the bone marrow, no evidence of disease, and partial recovery of peripheral blood counts (platelets >50,000/microliter and absolute neutrophil count >500/microliter).

Results from the trial demonstrated a 35% (51/147) CR+CRh rate in mIDH1 R/R AML patients, with a median duration of response of 25.9 months. The median time to CR or CRh was 1.9 months. Of the patients who achieved the primary endpoint of CR+CRh, 92% (47/51) were CR with a median duration of response of 28.1 months. REZLIDHIA was well tolerated in the study with an adverse event profile largely characteristic of symptoms or conditions experienced by patients with AML undergoing treatment. Differentiation syndrome was observed in 16% of patients and was manageable in most cases with dose interruption and corticosteroids. Hepatotoxicity, presenting as increases in liver function parameters, occurred in 23% of patients and most cases were manageable with dose modifications.

"We are delighted by the approval of REZLIDHIA based on the strength of data supporting the efficacy and safety of the product," said Raul Rodriguez, Rigel’s president and CEO. "REZLIDHIA provides a new and important, oral therapy option for patients who typically have a poor clinical outcome. Additionally, this approval greatly strengthens and expands Rigel’s commercial hematology-oncology portfolio. I would like to extend our sincerest thanks to all the patients, their families and caregivers, the doctors, the FDA, and our team members who have all contributed to the approval of REZLIDHIA."

In August 2022, Rigel and Forma Therapeutics, Inc. announced they entered an exclusive, worldwide license agreement to develop, manufacture and commercialize REZLIDHIA. Under the terms of the agreement, Rigel will be responsible for the launch and commercialization of REZLIDHIA in the U.S., and intends to work with potential partners to further develop and commercialize the product outside the U.S.

Conference Call and Webcast Today at 6:30PM Eastern Time
Rigel will hold a live conference call and webcast today at 6:30 p.m. Eastern Time (3:30 p.m. Pacific Time) to discuss the FDA approval of REZLIDHIA.

Participants can access the live conference call by dialing 877-407-3088 (domestic) or 201-389-0927 (international). The conference call will also be webcast live and can be accessed from the Investor Relations section of the company’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

About AML
Acute myeloid leukemia (AML) is a rapidly progressing cancer of the blood and bone marrow that affects myeloid cells, which normally develop into various types of mature blood cells. AML occurs primarily in adults and accounts for about 1 percent of all adult cancers. The American Cancer Society estimates that in the United States alone, there will be about 20,050 new cases, most in adults, in 2022.1

Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.2 Refractory AML, which affects between 10 and 40 percent of newly diagnosed patients, occurs when a patient fails to achieve remission even after intensive treatment.3 Quality of life declines for patients with each successive line of treatment for AML, and well-tolerated treatments in relapsed or refractory disease remain an unmet need.

About REZLIDHIA
INDICATION
REZLIDHIA is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.

IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME

Differentiation syndrome, which can be fatal, can occur with REZLIDHIA treatment.
Symptoms may include dyspnea, pulmonary infiltrates/pleuropericardial effusion, kidney
injury, hypotension, fever, and weight gain. If differentiation syndrome is suspected,
withhold REZLIDHIA and initiate treatment with corticosteroids and hemodynamic
monitoring until symptom resolution.

WARNINGS AND PRECAUTIONS
Differentiation Syndrome
REZLIDHIA can cause differentiation syndrome. In the clinical trial of REZLIDHIA in patients with relapsed or refractory AML, differentiation syndrome occurred in 16% of patients, with grade 3 or 4 differentiation syndrome occurring in 8% of patients treated, and fatalities in 1% of patients. Differentiation syndrome is associated with rapid proliferation and differentiation of myeloid cells and may be life-threatening or fatal. Symptoms of differentiation syndrome in patients treated with REZLIDHIA included leukocytosis, dyspnea, pulmonary infiltrates/pleuropericardial effusion, kidney injury, fever, edema, pyrexia, and weight gain. Of the 25 patients who experienced differentiation syndrome, 19 (76%) recovered after treatment or after dose interruption of REZLIDHIA. Differentiation syndrome occurred as early as 1 day and up to 18 months after REZLIDHIA initiation and has been observed with or without concomitant leukocytosis.

If differentiation syndrome is suspected, temporarily withhold REZLIDHIA and initiate systemic corticosteroids (e.g., dexamethasone 10 mg IV every 12 hours) for a minimum of 3 days and until resolution of signs and symptoms. If concomitant leukocytosis is observed, initiate treatment with hydroxyurea, as clinically indicated. Taper corticosteroids and hydroxyurea after resolution of symptoms. Differentiation syndrome may recur with premature discontinuation of corticosteroids and/or hydroxyurea treatment. Institute supportive measures and hemodynamic monitoring until improvement; withhold dose of REZLIDHIA and consider dose reduction based on recurrence.

Hepatotoxicity
REZLIDHIA can cause hepatotoxicity, presenting as increased alanine aminotransferase (ALT), increased aspartate aminotransferase (AST), increased blood alkaline phosphatase, and/or elevated bilirubin. Of 153 patients with relapsed or refractory AML who received REZLIDHIA, hepatotoxicity occurred in 23% of patients; 13% experienced grade 3 or 4 hepatotoxicity. One patient treated with REZLIDHIA in combination with azacitidine in the clinical trial, a combination for which REZLIDHIA is not indicated, died from complications of drug-induced liver injury. The median time to onset of hepatotoxicity in patients with relapsed or refractory AML treated with REZLIDHIA was 1.2 months (range: 1 day to 17.5 months) after REZLIDHIA initiation, and the median time to resolution was 12 days (range: 1 day to 17 months). The most common hepatotoxicities were elevations of ALT, AST, blood alkaline phosphatase, and blood bilirubin.

Monitor patients frequently for clinical symptoms of hepatic dysfunction such as fatigue, anorexia, right upper abdominal discomfort, dark urine, or jaundice. Obtain baseline liver function tests prior to initiation of REZLIDHIA, at least once weekly for the first two months, once every other week for the third month, once in the fourth month, and once every other month for the duration of therapy. If hepatic dysfunction occurs, withhold, reduce, or permanently discontinue REZLIDHIA based on recurrence/severity.

ADVERSE REACTIONS
The most common (≥20%) adverse reactions, including laboratory abnormalities, were aspartate aminotransferase increased, alanine aminotransferase increased, potassium decreased, sodium decreased, alkaline phosphatase increased, nausea, creatinine increased, fatigue/malaise, arthralgia, constipation, lymphocytes increased, bilirubin increased, leukocytosis, uric acid increased, dyspnea, pyrexia, rash, lipase increased, mucositis, diarrhea and transaminitis.

DRUG INTERACTIONS

Avoid concomitant use of REZLIDHIA with strong or moderate CYP3A inducers.
Avoid concomitant use of REZLIDHIA with sensitive CYP3A substrates unless otherwise instructed in the substrates prescribing information. If concomitant use is unavoidable, monitor patients for loss of therapeutic effect of these drugs.
LACTATION
Advise women not to breastfeed during treatment with REZLIDHIA and for 2 weeks after the last dose.

GERIATRIC USE
No overall differences in effectiveness were observed between patients 65 years and older and younger patients. Compared to patients younger than 65 years of age, an increase in incidence of hepatotoxicity and hypertension was observed in patients ≥65 years of age.

HEPATIC IMPAIRMENT
In patients with mild or moderate hepatic impairment, closely monitor for increased probability of differentiation syndrome.

Click here for Full Prescribing Information, including Boxed WARNING.

To report side effects of prescription drugs to the FDA, visit www.fda.gov/medwatch or call 1-800-FDA-1088 (800-332-1088).

REZLIDHIA is a registered trademark of Rigel Pharmaceuticals, Inc.

On December 1, 2022 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that new and updated data across its oncology pipeline will be presented at the European Society for Medical Oncology Immuno-Oncology (ESMO IO) Congress 2022 from December 7 to 9 in Geneva, Switzerland (Press release, Regeneron, DEC 1, 2022, View Source [SID1234624696]). Presentation highlights include first clinical results and new exploratory analyses from trials investigating LAG-3 inhibitor fianlimab and/or PD-1 inhibitor Libtayo (cemiplimab) in non-small cell lung cancer (NSCLC), melanoma and cervical cancer.

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"Our ESMO (Free ESMO Whitepaper) IO presentations reflect our continued progress toward developing a differentiated oncology pipeline with the potential to treat a variety of cancers with unique combinations," said Israel Lowy, M.D., Ph.D., Senior Vice President, Translational and Clinical Sciences, Oncology at Regeneron. "Libtayo has already proven to be an effective immunotherapy across multiple tumor types and is poised to serve as a foundational therapy for our investigational combination approaches. Fianlimab combined with Libtayo has generated early-but-promising clinical activity in advanced melanoma and non-small cell lung cancer, demonstrating the potential of Regeneron’s oncology pipeline to potentially advance the standard-of-care in various cancers."

Among the research published by ESMO (Free ESMO Whitepaper) IO today were initial results for an investigational combination of fianlimab and Libtayo in patients with unresectable stage IIIB-C or IV NSCLC. The results are from two expansion cohorts of a Phase 1 trial – one with anti-PD-1/PD-L1-naïve patients (naïve cohort) and the other with anti-PD-1/PD-L1-experienced patients (experienced cohort). Patients received fianlimab 1600 milligrams and Libtayo 350 milligrams intravenously every 3 weeks for 12 months, with a median follow up of 9 months and 5 months for the naïve and experienced cohorts, respectively.

Efficacy results demonstrated an investigator-assessed objective response rate (ORR) of 27% (4 of 15 patients; all partial responses [PR]) in the naïve cohort and 7% in the experienced cohort (1 of 15 patients with a PR). Additionally, exploratory analyses of the naïve cohort found the ORR was 50% (3 patients) among those who had not received any prior systemic therapy and 100% (3 patients) among those with tumors that had ≥50% PD-L1 expression. Median duration of response was not reached in the naïve cohort and was 5 months in the experienced cohort (95% confidence interval: not evaluable to not evaluable).

In terms of safety for the naïve and experienced cohorts, rates of ≥grade 3 adverse events (AE) were respectively 33% (5 patients) and 40% (6 patients), while rates of serious AEs were respectively 20% (3 patients) and 13% (2 patients). One patient in the naïve cohort and no patients in the experienced cohort discontinued treatment due to an AE; there were no treatment-related deaths reported in either cohort. Updated efficacy and safety data will be presented during a poster session (abstract #127P).

Other notable presentations at ESMO (Free ESMO Whitepaper) IO include:

An exploratory analysis of two expansion cohorts from a Phase 1 trial investigating fianlimab in combination with Libtayo in patients with advanced melanoma, which offer new insights on the use of this treatment combination in patients with poor prognostic features at baseline.
A post-hoc exploratory analysis of Regeneron’s Phase 3 EMPOWER trials in advanced NSCLC and advanced cervical cancer, focusing on efficacy outcomes in Libtayo-treated patients with liver metastases compared to those treated with chemotherapy alone.
An oral presentation on EMPOWER-Lung 3 Part 1, a Phase 3 trial which assessed the efficacy and safety of Libtayo plus ipilimumab and platinum-based doublet chemotherapy, compared to Libtayo plus chemotherapy, and chemotherapy alone, in patients with advanced NSCLC and <50% PD-L1 expression.
Regeneron data at ESMO (Free ESMO Whitepaper) IO

Medicine

Abstract title

Abstract

Presentation date/time

(all CET)

Lung cancer

Libtayo,
fianlimab

Phase 1 study of fianlimab: A human lymphocyte activation gene-3 (LAG-3) monoclonal antibody, in combination with cemiplimab in advanced NSCLC

#127P

December 8

12:30-13:15 pm

Libtayo

Cemiplimab (cemi) + platinum doublet chemotherapy (chemo) + ipilimumab (ipi) for first-line treatment of advanced non-small cell lung cancer (NSCLC): EMPOWER-Lung 3 part 1

#122MO

December 8

9:35-9:40 am

Libtayo

An observational study to assess the effectiveness and safety of cemiplimab in patients with advanced non-small cell lung cancer (NSCLC) in routine clinical practice within Europe (CEMI-LUNG)

#119TiP

December 8

12:30-13:15 pm

Skin cancer

Libtayo,
fianlimab

Phase 1 study of fianlimab, a human lymphocyte activation gene-3 (LAG-3) monoclonal antibody, in combination with cemiplimab in advanced melanoma (mel): subgroup analysis

#150P

December 8

12:30-13:15 pm

Ovarian cancer

Libtayo,
ubamatamab

First-in-human (FIH) Phase 1/2 study of ubamatamab, a MUC16xCD3 bispecific antibody, administered alone or in combination with cemiplimab in patients with recurrent ovarian cancer (OC)

#197TiP

December 8

12:30-13:15 pm

Advanced solid tumors

Libtayo

Liver metastases (mets) and treatment effect of cemiplimab-based therapy: An analysis from three Phase 3 trials (EMPOWER-Lung 1, EMPOWER-Lung 3 part 2, and EMPOWER-Cervical 1)

#168P

December 8

12:30-13:15 pm

The potential uses of Libtayo, fianlimab, ubamatamab and REGN6569 described above are investigational, and their safety and efficacy in these uses have not been fully evaluated by any regulatory authority. Fianlimab, ubamatamab and REGN6569 are not currently approved for use in any indication.