On May 7, 2024 Rigel Pharmaceuticals, Inc. reported financial results for the first quarter ended March 31, 2024, including sales of TAVALISSE (fostamatinib disodium hexahydrate) tablets for the treatment of adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment and sales of 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, MAY 7, 2024, View Source [SID1234642784]).

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"Results for the first quarter of 2024 continued to demonstrate strong commercial demand with the highest number of TAVALISSE and REZLIDHIA bottles sold in a quarter since launch. We are also excited about the recent acquisition of GAVRETO and are on track to include this product in our commercial portfolio in July of this year," said Raul Rodriguez, Rigel’s president and CEO. "At the same time, we are progressing the development of olutasidenib with our strategic collaborators, MD Anderson and CONNECT, and driving forward our other pipeline programs."

Business Update

· In the first quarter of 2024, a total of 2,193 TAVALISSE bottles were sold in the U.S. driven by 2,483 bottles shipped to patients and clinics, the highest number in a quarter since launch. Bottles remaining in distribution channels decreased by 290 bottles during the quarter.

· In the first quarter of 2024, a total of 390 REZLIDHIA bottles were sold in the U.S., significantly accelerating sales growth over last year. This growth was driven by increased demand, with 326 bottles shipped to patients and clinics.

· In April 2024, Rigel announced a peer-reviewed publication in Leukemia & Lymphoma on data from an analysis of the Phase 2 study evaluating REZLIDHIA in patients with mIDH1 AML who were R/R to prior venetoclax-based regimens. The findings from these analyses suggest that REZLIDHIA may provide an effective treatment for patients with recurrent AML following venetoclax combination therapy. REZLIDHIA induced durable remissions consistent with those observed in the pivotal trial and had a favorable tolerability profile.

· In March 2024, Rigel appointed Lisa Rojkjaer, M.D. as Executive Vice President and Chief Medical Officer. Dr. Rojkjaer is an industry veteran with over 20 years of clinical development, regulatory, and medical affairs experience with a focus on hematology and oncology. She is a board-certified hematologist with an international clinical practice background.

· In February 2024, Rigel announced the acquisition of the U.S. rights to GAVRETO (pralsetinib). GAVRETO is a once daily, small molecule, oral, kinase inhibitor of wild-type RET (rearranged during transfection) and oncogenic RET fusions. GAVRETO is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) and advanced or metastatic thyroid cancer. The acquisition of this product further expands Rigel’s portfolio and leverages Rigel’s existing infrastructure in both the institutional and community settings. Rigel expects to complete the transition of the asset and start recognizing product sales in July 2024.

· In January 2024, Rigel and CONNECT announced a strategic development collaboration to evaluate REZLIDHIA (olutasidenib) in combination with temozolomide in patients with high-grade glioma (HGG) harboring an IDH1 mutation. Under the collaboration, CONNECT will include olutasidenib in CONNECT’s TarGeT-D, a molecularly guided Phase 2 umbrella clinical trial for HGG. In the Rigel-sponsored arm, adolescents and young adult patients (≤39 years old) with newly diagnosed IDH1-mutation positive HGG will receive maintenance therapy with olutasidenib in combination with temozolomide for the first year after radiotherapy, followed by olutasidenib monotherapy for the second year. Rigel will provide CONNECT funding up to $3 million and study material over the four-year collaboration.

· Rigel continues to advance its Phase 1b clinical trial evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of R2891, a novel and selective IRAK1/4 inhibitor, in patients with relapsed/refractory lower-risk myelodysplastic syndrome (LR-MDS). Enrollment in the third cohort of the trial has been completed and the company is planning to include two additional cohorts with twice daily dosing regimens. Preliminary data are expected by the end of 2024.

Financial Update

For the first quarter of 2024, total revenues were $29.5 million, consisting of $21.1 million in TAVALISSE net product sales, $4.9 million in REZLIDHIA net product sales, and $3.5 million in contract revenue from collaborations. Although TAVALISSE bottles shipped to patients and clinics reached the highest quarterly number of bottles since launch, net product sales were $21.1 million compared to $22.3 million in the same period of 2023, primarily due to a decrease in the number of bottles remaining in distribution channels. REZLIDHIA net product sales were $4.9 million compared to $1.5 million in the same period of 2023. Contract revenue from collaborations consisted of $2.3 million from Kissei Pharmaceutical Co., Ltd. related to delivery of drug supplies, $1.1 million from Grifols S.A. related to earned royalties, and $0.1 million from Medison Pharma Trading AG related to delivery of drug supplies and earned royalties.

For the first quarter of 2024, total costs and expenses were $36.5 million compared to $38.8 million for the same period of 2023. The decrease in costs and expenses was partly due to decreased research and development costs due to the timing of clinical trial activities related to the IRAK 1/4 inhibitor program, as well as the timing of trial completion activities related to two Phase 3 clinical trials of fostamatinib in patients with COVID-19 and wAIHA. In addition, the decrease was due to lower consulting and third-party services as well as lower facility-related costs. These decreases were partially offset by higher stock-based compensation expenses, mainly from performance-based awards.

For the first quarter of 2024, Rigel reported a net loss of $8.2 million, or $0.05 per basic and diluted share, compared to a net loss of $13.5 million, or $0.08 per basic and diluted share, for the same period of 2023.

As of March 31, 2024, Rigel had cash, cash equivalents and short-term investments of $49.6 million, compared to $56.9 million as of December 31, 2023. In April 2024, Rigel entered into an amendment to the Credit Agreement with MidCap Financial Trust. As part of the amendment, Rigel extended the maturity date and interest only period by one year.

Conference Call and Webcast with Slides Today at 4:30pm Eastern Time

Rigel will hold a live conference call and webcast today at 4:30pm Eastern Time (1:30pm Pacific Time).

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 ITP

In patients with ITP (immune thrombocytopenia), the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs), and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

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 there will be about 20,800 new cases in the United States, most in adults, in 2024.2

Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.3 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.4 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 NSCLC

It is estimated that over 230,000 adults in the U.S. will be diagnosed with lung cancer in 2024. Lung cancer is the leading cause of cancer death in the U.S, with NSCLC being the most common type accounting for 80-85% of all lung cancer diagnoses.5 RET fusions are implicated in approximately 1-2% of patients with NSCLC.6

About TAVALISSE

Indication

TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Important Safety Information

Warnings and Precautions

· Hypertension can occur with TAVALISSE treatment. Patients with pre-existing hypertension may be more susceptible to the hypertensive effects. Monitor blood pressure every 2 weeks until stable, then monthly, and adjust or initiate antihypertensive therapy for blood pressure control maintenance during therapy. If increased blood pressure persists, TAVALISSE interruption, reduction, or discontinuation may be required.

· Elevated liver function tests (LFTs), mainly ALT and AST, can occur with TAVALISSE. Monitor LFTs monthly during treatment. If ALT or AST increase to ≥3 x upper limit of normal, manage hepatotoxicity using TAVALISSE interruption, reduction, or discontinuation.

· Diarrhea occurred in 31% of patients and severe diarrhea occurred in 1% of patients treated with TAVALISSE. Monitor patients for the development of diarrhea and manage using supportive care measures early after the onset of symptoms. If diarrhea becomes severe (≥Grade 3), interrupt, reduce dose or discontinue TAVALISSE.

· Neutropenia occurred in 6% of patients treated with TAVALISSE; febrile neutropenia occurred in 1% of patients. Monitor the ANC monthly and for infection during treatment. Manage toxicity with TAVALISSE interruption, reduction, or discontinuation.

· TAVALISSE can cause fetal harm when administered to pregnant women. Advise pregnant women the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 1 month after the last dose. Verify pregnancy status prior to initiating TAVALISSE. It is unknown if TAVALISSE or its metabolite is present in human milk. Because of the potential for serious adverse reactions in a breastfed child, advise a lactating woman not to breastfeed during TAVALISSE treatment and for at least 1 month after the last dose.

Drug Interactions

· Concomitant use of TAVALISSE with strong CYP3A4 inhibitors increases exposure to the major active metabolite of TAVALISSE (R406), which may increase the risk of adverse reactions. Monitor for toxicities that may require a reduction in TAVALISSE dose.

· It is not recommended to use TAVALISSE with strong CYP3A4 inducers, as concomitant use reduces exposure to R406.

· Concomitant use of TAVALISSE may increase concentrations of some CYP3A4 substrate drugs and may require a dose reduction of the CYP3A4 substrate drug.

· Concomitant use of TAVALISSE may increase concentrations of BCRP substrate drugs (eg, rosuvastatin) and P-Glycoprotein (P-gp) substrate drugs (eg, digoxin), which may require a dose reduction of the BCRP and P-gp substrate drug.

Adverse Reactions

· Serious adverse drug reactions in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis, which occurred in 1% of TAVALISSE patients. In addition, severe adverse reactions occurred including dyspnea and hypertension (both 2%), neutropenia, arthralgia, chest pain, diarrhea, dizziness, nephrolithiasis, pain in extremity, toothache, syncope, and hypoxia (all 1%).

· Common adverse reactions (≥5% and more common than placebo) from FIT-1 and FIT-2 included: diarrhea, hypertension, nausea, dizziness, ALT and AST increased, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia.

Please see www.TAVALISSEUSPI.com for Full Prescribing Information.

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

TAVALISSE is a registered trademark of Rigel Pharmaceuticals, Inc.