On August 6, 2024 Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) reported financial results for the second quarter of 2024, along with recent product highlights and corporate updates (Press release, Zai Laboratory, AUG 6, 2024, View Source [SID1234645438]).

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"In the second quarter, we achieved impressive commercial growth, maintained financial discipline, and made significant strides across our product portfolio, highlighting our capability to execute on our strategic objectives," said Dr. Samantha Du, Founder, Chairperson, and Chief Executive Officer of Zai Lab. "The success of VYVGART underscores the urgent need for effective and safe treatments for patients living with generalized myasthenia gravis. We will continue to prioritize resources and focus investments on high-value initiatives with the potential to significantly improve human health. Recently, we expanded our global oncology pipeline with a next generation ROR1 ADC program, ZL-6301. Additionally, the progress of our pipeline, including efgartigimod, bemarituzumab, KarXT and our three global clinical-stage assets, keeps us on track to achieve the goals outlined in our five-year strategic plan."

"Our net product revenues grew 45% y-o-y in the second quarter, driven by the successful commercialization of VYVGART," said Josh Smiley, President and Chief Operating Officer of Zai Lab. "In 2024, we will maintain our focus on the strong execution of VYVGART’s launch in generalized myasthenia gravis while also preparing for the anticipated launches of several new products and indications in the near future. Our efforts to build a stronger and more efficient organization, coupled with our innovative pipeline, position us for substantial topline growth and set us on the path to achieve profitability by the end of 2025."

Second-Quarter 2024 Financial Results

•Product revenue, net was $100.1 million in the second quarter of 2024, compared to $68.9 million for the same period in 2023, representing 45% y-o-y growth and 47% y-o-y growth at CER. This increase was primarily driven by increased sales for VYVGART since its launch in September 2023 and China’s National Reimbursement Drug List (NRDL) listing in January 2024, and increased sales for ZEJULA and NUZYRA. Primary drivers of this revenue growth included the following:

–ZEJULA: $45.0 million in the second quarter of 2024, an increase of 5% y-o-y from $43.0 million for the same period in 2023, driven by increased hospital sales in first-line ovarian cancer and increased duration of treatment and supported by the renewal of ZEJULA’s NRDL listing for the maintenance treatment of adult patients with first-line and recurrent ovarian cancer, effective January 1, 2024.

–VYVGART: $23.2 million in the second quarter of 2024, compared to $0.1 million for the same period in 2023, driven by its NRDL listing for the treatment of generalized myasthenia gravis (gMG) effective January 1, 2024 and positive physician and patient reception as well as increased patient access as VYVGART is added to hospital formularies. VYVGART was launched for the treatment of gMG in September 2023.

–NUZYRA: $12.3 million in the second quarter of 2024, an increase of 165% y-o-y compared to $4.6 million for the same period in 2023, driven by the NRDL listings for the IV formulation of NUZYRA for the treatment of adults with community-acquired bacterial pneumonia (CABP) and acute bacterial skin and skin structure infections (ABSSSI) in the first quarter of 2023 and the oral formulation for these indications in the first quarter of 2024.

•Research and Development (R&D) expenses were $61.6 million in the second quarter of 2024, compared to $76.7 million for the same period in 2023. This decrease was primarily due to decreased milestone fees for our license and collaboration agreements, partially offset by increased clinical trial expenses related to newly initiated studies and progress of existing studies.

•Selling, General and Administrative expenses were $79.7 million in the second quarter of 2024, compared to $67.9 million for the same period in 2023. This increase was primarily driven by higher general selling expenses and headcount growth primarily to support VYVGART.

•Net loss was $80.3 million in the second quarter of 2024, or a loss per ordinary share attributable to common stockholders of $0.08 (or loss per American Deposit Share (ADS) of $0.82), compared to a net loss of $120.9 million for the same period in 2023, or a loss per ordinary share of $0.13 (or loss per ADS of $1.25).

•Cash and cash equivalents, short-term investments, and current restricted cash totaled $730.0 million as of June 30, 2024, compared to $750.8 million as of March 31, 2024.

Corporate Updates

Below are key corporate updates since our last earnings release:

•Business Development: In July 2024, Zai Lab entered into a strategic partnership and global license agreement with MabCare Therapeutics. Through this collaboration, the Company expanded its global oncology pipeline with a next generation ADC targeting ROR1, ZL-6301. ZL-6301 has the potential to be used in the treatment of solid tumors where ROR1 is commonly expressed and in hematological malignancies where ROR1 is a validated target. ZL-6301 has demonstrated an encouraging pre-clinical profile, and it is currently in the IND-enabling stage. Zai Lab plans to focus on advancing its global development.

•Organizational Update: In June 2024, Zai Lab appointed Dr. Rafael Amado as President, Head of Global Research and Development, expanding his role to encompass R&D efforts across all of our therapeutic areas upon the retirement of Dr. Harald Reinhart at the end of June. This leadership transition allows for continued momentum and a strategic focus on our pipeline.

Recent Pipeline Highlights

Below are key product updates since our last earnings release:

Oncology Pipeline

•Niraparib (PARP): In July 2024, Zai Lab announced that data of a Zai-supported study published in Cell provides new insights with potential to improve treatment of HRD-positive ovarian cancers, including through neoadjuvant monotherapy with niraparib and a combination of niraparib and ZL-1218, an investigational CCR8 antibody.

•Bemarituzumab (FGFR2b):

•In June 2024, Zai Lab and partner Amgen completed enrollment for the global Phase 3 FORTITUDE-101 study of bemarituzumab plus chemotherapy in first-line gastric cancer.

•The enrollment is ongoing for the global Phase 3 FORTITUDE-102 study of bemarituzumab plus chemotherapy and nivolumab in first-line gastric cancer in mainland China, Hong Kong, Macau, and Taiwan (collectively, Greater China).

•AUGTYRO (Repotrectinib) (ROS1/TRK):

•In May 2024, China’s National Medical Products Administration (NMPA) approved the New Drug Application (NDA) for repotrectinib for the treatment of adult patients with locally advanced or metastatic ROS1-positive NSCLC. The approval was based on the global TRIDENT-1 study that evaluated repotrectinib in TKI naïve and TKI-pretreated patients with ROS1-positive NSCLC. We participated in the Greater China portion of this study.

•In June 2024, Zai Lab partner Bristol Myers Squibb (BMS) announced that the U.S. Food and Drug Administration (FDA) had granted accelerated approval of AUGTYRO for the treatment of patients with NTRK-positive locally advanced or metastatic solid tumors.

•ZL-1310 (DLL3 ADC): The enrollment in the United States and Greater China is ongoing for the global Phase 1 study in relapsed and refractory second-line+ small cell lung cancer (SCLC) who have progressed on or after platinum-based chemotherapy.

•ZL-1218 (CCR8): The enrollment in the United States, Europe, and Greater China is ongoing for the global Phase 1 study of ZL-1218 as a single agent and in combination with pembrolizumab in patients with advanced solid tumor malignancies.

Immunology, Neuroscience, and Infectious Disease Pipeline

•Efgartigimod (FcRn):

•In July 2024, the NMPA approved the Biologics License Application (BLA) for efgartigimod alfa injection (subcutaneous injection) (efgartigimod SC) as an add on to standard therapy for the treatment of adult patients with gMG who are anti-acetylcholine receptor (AChR) antibody positive. This approval will provide additional flexibility and optionality for gMG patients in mainland China.

•In May 2024, the NMPA accepted the supplemental Biologics License Application (sBLA) with priority review for efgartigimod SC in chronic inflammatory demyelinating polyneuropathy (CIDP). In June 2024, Zai Lab partner argenx announced that the FDA approved VYVGART Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) for this indication. We participated in the global ADHERE Study, the largest clinical trial to date studying CIDP.

•XACDURO (Sulbactam-Durlobactam or SUL-DUR): In May 2024, the NMPA approved the NDA for XACDURO for the treatment of adult patients with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia (HABP/VABP) caused by susceptible isolates of Acinetobacter baumannii-calcoaceticus complex.

•Xanomeline-Trospium (KarXT) (M1/M4-agonist): In July 2024, Zai Lab joined the global Phase 3 ADEPT-2 study in Alzheimer’s disease with psychosis in Greater China.

•ZL-1102 (IL-17 Humabody): In May 2024, Zai Lab dosed the first patient in a global Phase 2 study evaluating the efficacy and safety of ZL-1102 for the treatment of chronic plaque psoriasis (CPP).

Anticipated Major Milestones in 2024 and the First Half of 2025

Potential Regulatory Submissions to the NMPA

•TTFields: Marketing Authorization Application (MAA) submission in second-line+ NSCLC following progression on or after platinum-based chemotherapy in the fourth quarter of 2024.

•Tisotumab Vedotin (Tissue Factor ADC): BLA submission in recurrent or metastatic cervical cancer following progression on or after chemotherapy.

•Repotrectinib (ROS1/TRK): supplementary NDA (sNDA) submission in NTRK+ solid tumors.

•Xanomeline-Trospium (KarXT) (M1/M4-agonist): NDA submission in schizophrenia.

Expected Clinical Development and Data Readouts

Efgartigimod (FcRn)

•Zai Lab to join the registrational study of efgartigimod SC given by prefilled syringe in Thyroid Eye Disease (TED) in Greater China in the fourth quarter of 2024.

•argenx to provide topline data from the Phase 2/3 ALKIVIA study evaluating efgartigimod across three myositis subsets (immune-mediated necrotizing myopathy (IMNM), anti-synthetase syndrome (ASyS), and dermatomyositis (DM)) in the fourth quarter of 2024. Zai Lab to join the Phase 3 portion of this study in the fourth quarter of 2024.

•Zai Lab to join the global registrational Phase 3 studies in seronegative gMG and ocular MG in early 2025, aiming to expand the label into broader MG populations.

Xanomeline-Trospium (KarXT) (M1/M4-agonist)

•Zai Lab to complete patient enrollment of the China registrational bridging study in schizophrenia, with topline data expected by the end of 2024.

•BMS to report data from the EMERGENT-4 and EMERGENT-5 trials evaluating the long-term safety for treatment of schizophrenia in the second half of 2024.

TTFields

•Novocure to provide a topline data readout from the Phase 3 PANOVA-3 clinical trial in locally advanced pancreatic cancer in the fourth quarter of 2024. We are participating in the study in Greater China.

ZL-1310 (DLL3 ADC)

•Potential dose escalation data from the global Phase 1 study in relapsed and refractory second-line+ SCLC at the end of 2024 or early 2025.

ZL-1218 (CCR8)

•Present the preliminary clinical PK and PD analysis of the global Phase 1 study in solid tumors at 2024 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) in September 2024.

Conference Call and Webcast Information

Zai Lab will host a live conference call and webcast tomorrow, August 7, 2024, at 8:00 a.m. ET (8:00 p.m. HKT). Listeners may access the live webcast by visiting the Company’s website at View Source Participants must register in advance of the conference call.

Details are as follows:

Registration Link: https://register.vevent.com/register/BIaccafead4b094cf191720bf5d03048c6

All participants must use the link provided above to complete the online registration process in advance of the conference call. Dial-in details will be in the confirmation email which the participant will receive upon registering.

A replay will be available shortly after the call and can be accessed by visiting the Company’s website.

On August 6, 2024 Veracyte, Inc. (Nasdaq: VCYT) reported financial results for the second quarter ended June 30, 2024 (Press release, Veracyte, AUG 6, 2024, View Source [SID1234645437]).

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"Our exceptional second quarter results are a testament to the strength and robustness of Decipher and Afirma," said Marc Stapley, Veracyte’s chief executive officer. "With both tests clearly gaining share in their respective markets and plenty of headroom for durable future expansion, our confidence in their long-term prospects continues to grow. Meanwhile, our positive cash generation and class-leading profitability profile are fueling a portfolio of tests that are poised to drive meaningful advances in precision medicine."

Key Business Highlights
•Increased second quarter total revenue by 27%, to $114.4 million, compared to the second quarter of 2023.
•Grew total test volume to 39,023, an increase of 23% compared to the second quarter of 2023.
•Increased second quarter net income to $5.7 million and delivered adjusted EBITDA of $24.0 million, or 21% of revenue.
•Achieved record Decipher Prostate test volume of close to 19,900 tests, driven by recently updated NCCN* prostate cancer guidelines, in which the Decipher Prostate test received the highest-level rating among gene expression tests.
•Delivered record Afirma test volume of approximately 15,700 and received Medicare coverage for Afirma testing of patients with thyroid nodules classified as "Bethesda V," giving more patients annually access to the test.
•Reinforced Veracyte’s commitment to evidence expansion with three published Decipher Prostate test studies, including a real-world, population-based analysis of Decipher data linked to the National Cancer Institute’s SEER database demonstrating the test’s clinical utility and underscoring the power of the Veracyte Diagnostics Platform.
•Generated $26.7 million of cash during the second quarter to end the quarter with $235.9 million of cash and cash equivalents.

* National Comprehensive Cancer Network. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way.

A reconciliation of GAAP to non-GAAP financial measures has been provided in the tables included in this press release. An explanation of these measures is also included below under the heading "Note Regarding Use of Non-GAAP Financial Measures."

Second Quarter 2024 Financial Results

Total revenue for the second quarter of 2024 was $114.4 million, an increase of 27% compared to $90.3 million reported in the second quarter of 2023. Testing revenue was $107.0 million, an increase of 31% compared to $81.7

million in the second quarter of 2023, driven by the strong performance of our Decipher Prostate and Afirma tests. Product revenue was $3.9 million, a decrease of 3% compared to $4.0 million in the second quarter of 2023. Biopharmaceutical and other revenue was $3.6 million, a decrease of 22% compared to $4.6 million in the second quarter of 2023.

Total gross margin for the second quarter of 2024 was 68%, compared to 62% in the second quarter of 2023. Non-GAAP gross margin, which excludes the amortization of acquired intangible assets, stock-based compensation, other acquisition related expenses, and certain other adjustments was 71%, compared to 68% in the second quarter of 2023.

Operating expenses were $73.3 million for the second quarter of 2024. Non-GAAP operating expenses, which excludes amortization of acquired intangible assets, stock-based compensation, other acquisition related expenses, and other restructuring costs, grew 14% to $59.0 million compared to $51.7 million in the second quarter of 2023.

Net income for the second quarter of 2024 was $5.7 million, an improvement of 168% compared to the second quarter of 2023. Basic and diluted net earnings per common share was $0.07, an improvement of $0.19 compared to the second quarter of 2023. Non-GAAP diluted net earnings per common share was $0.30, an improvement of $0.18 compared to the second quarter of 2023. Net cash provided by operating activities in the first six months of 2024 was $20.6 million, an improvement of $6.1 million compared to the same period in 2023.

Adjusted EBITDA for the second quarter of 2024 was $24.0 million, an improvement of 118% compared to the second quarter of 2023, representing 21% of revenue compared to 12% of revenue respectively.

A reconciliation of GAAP to non-GAAP financial measures has been provided in the tables included in this press release. An explanation of these measures is also included below under the heading "Note Regarding Use of Non-GAAP Financial Measures."

2024 Financial Outlook

The company is raising full-year 2024 total revenue guidance to $432 million to $438 million, representing year-over-year growth of 20% to 21% and testing revenue growth of approximately 25%. This guidance range represents an increase compared to prior guidance of $402 million to $410 million. In addition, the company now expects cash, cash equivalents and short-term investments at the end of the year to be $260 million to $270 million compared to prior guidance of $236 million to $240 million.

Conference Call and Webcast Details

Veracyte will host a conference call and webcast today at 4:30 p.m. Eastern Time to discuss the company’s financial results and provide a general business update. The conference call will be webcast live from the company’s website and will be available via the following link: View Source The webcast should be accessed 10 minutes prior to the conference call start time. A replay of the webcast will be available for one year following the conclusion of the live broadcast and will be accessible on the company’s website at View Source

The conference call dial-in can be accessed by registering at the following link: https://register.vevent.com/register/BIbb7f6b70b4ce4820b6391059390d6139

On August 6, 2024 Sensei Biotherapeutics, Inc. (Nasdaq: SNSE), a clinical stage immuno-oncology company focused on the discovery and development of next-generation therapeutics for cancer patients, reported financial results for the second quarter ended June 30, 2024, and provided corporate updates (Press release, Sensei Biotherapeutics, AUG 6, 2024, View Source [SID1234645436]).

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"Collectively, the clinical data presented on SNS-101 over the past six months demonstrated early signs of clinical activity in a patient population generally resistant to immunotherapy, a well-tolerated safety profile, and the avoidance of a pharmacokinetic sink that hindered first-generation approaches to targeting VISTA," said John Celebi, President and Chief Executive Officer. "We believe these data validate that a pH-selective approach can overcome the previous hurdles associated with targeting VISTA, and we look forward to advancing patient enrollment in dose expansion cohorts to inform our Phase 2 trial design. With cash runway into the fourth quarter of 2025, we believe we are well positioned to substantially progress SNS-101 as we seek to create value for Sensei’s stockholders by developing innovative new treatment options for patients."

Clinical Highlights and Milestones

SNS-101

SNS-101 is a conditionally active antibody harnessing the acidic tumor microenvironment to selectively target the immune checkpoint VISTA (V-domain Ig suppressor of T cell activation). VISTA is implicated in numerous cancer indications and its expression correlates with low survival rates.

Sensei is conducting a multi-center Phase 1/2 clinical trial to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of SNS-101 as both a monotherapy and in combination with Regeneron’s PD-1 inhibitor Libtayo (cemiplimab) in patients with advanced solid tumors.

In May 2024, Sensei presented promising clinical data from the dose escalation portion of its Phase 1/2 trial of SNS-101 at the 2024 American Society of Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. As of the April 30, 2024 data cutoff, SNS-101 demonstrated preliminary evidence of promising clinical activity in multiple tumor types, a potentially best-in-class pharmacokinetic (PK) profile and was well tolerated alone and in combination with cemiplimab, with no dose-limiting toxicities observed.
Patient enrollment is advancing in the dose expansion portion of the Phase 1/2 study. The Company is on track to report initial data from the dose expansion cohorts and hold an end-of-Phase 1 meeting with the FDA by the end of 2024.
In April 2024, Sensei published a peer-reviewed research paper in Nature Communications describing the mechanism of action of SNS-101 selectively targeting the active form of VISTA within the tumor microenvironment.
Corporate Updates

Sensei announces the appointment of Josiah Craver effective as of July 22, 2024, as Senior Vice President of Finance. Mr. Craver brings extensive experience in finance and accounting. Prior to joining Sensei in July 2024, he was the SVP of finance and corporate controller at KALA BIO and held senior finance positions at Solid Biosciences, including VP of finance and corporate controller after starting his career at PricewaterhouseCoopers in the health industries audit practice primarily serving life science and biotech companies of all sizes. Mr. Craver holds a M.S. in Accountancy and is a Certified Public Accountant.
Second Quarter 2024 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $52.3 million as of June 30, 2024. Sensei expects its current cash balance to fund operations into the fourth quarter of 2025.

Research and Development (R&D) Expenses: R&D expenses were $4.6 million for the quarter ended June 30, 2024, compared to $4.8 million for the quarter ended June 30, 2023. The decrease in R&D expenses was primarily attributable to lower outside research fees and lower costs related to preclinical research, primarily offset by higher expenses associated with clinical trials.

General and Administrative (G&A) Expenses: G&A expenses were $3.2 million for the quarter ended June 30, 2024, compared to $5.4 million for the quarter ended June 30, 2023. The decrease in G&A expense was primarily attributable to decreased cost for external professional services.

Net Loss: Net loss was $7.1 million for the quarter ended June 30, 2024, compared to $9.4million for the quarter ended June 30, 2023.

On August 6, 2024 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) for SLS009, a novel, and highly selective CDK9 inhibitor, for the treatment of relapsed/refractory (r/r) peripheral T-cell lymphomas (PTCL) (Press release, Sellas Life Sciences, AUG 6, 2024, View Source [SID1234645435]).

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"We are pleased to announce the EMA’s granting of ODD for SLS009, highlighting another important milestone following recent FDA’s Orphan Drug and Fast Track Designations for SLS009 in PTCL," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "PTCL is an aggressive form of lymphoma with limited treatment options, underscoring the urgent need for new and effective therapies. We are delighted that the potential of SLS009 has been recognized by both regulatory agencies and across multiple indications including acute myeloid leukemia (AML), pediatric AML, and pediatric acute lymphoblastic leukemia (ALL). This additional orphan drug designation also highlights our strong internal regulatory expertise, and we look forward to advancing the SLS009 development and our overall clinical programs to deliver its potential benefits to cancer patients."

In the completed dose-escalation portion of the Phase 1 trial in relapsed/refractory hematological malignancies, SLS009 demonstrated favorable safety/tolerability and promising clinical efficacy. Complete or partial responses were observed in AML and lymphoma patients, with a 36.4% response rate achieved specifically in the PTCL patient group, including one patient who remained in continuous treatment for over 56 weeks. The current standard of care for r/r PTCL, belinostat, showed in its pivotal Phase 2 study a 25.8% response rate in a similar patient population to that in the SLS009 Phase 1 clinical trial.

Orphan Designation is granted to therapies aimed at the treatment, prevention, or diagnosis of life-threatening or chronically debilitating diseases that affect no more than five in 10,000 persons in the European Union (EU) and for which no satisfactory therapy is available. The treatment must also provide significant benefit to those affected by the condition.

On August 6, 2024 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, reported financial results for the second quarter ended June 30, 2024, including sales of TAVALISSE (fostamatinib disodium hexahydrate) for the treatment of chronic immune thrombocytopenia (ITP); REZLIDHIA (olutasidenib) for the treatment of relapsed or refractory (R/R) mutated isocitrate dehydrogenase-1 (mIDH1) acute myeloid leukemia (AML); and GAVRETO (pralsetinib) for the treatment of metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) and advanced or metastatic thyroid cancer, and recent business progress (Press release, Rigel, AUG 6, 2024, View Source [SID1234645434]).

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"In the second quarter, we advanced key strategic initiatives including the successful transfer of GAVRETO to our commercial portfolio, enabling us to bring this important treatment option to current and newly prescribed patients without interruption," said Raul Rodriguez, Rigel’s president and CEO. "The addition of GAVRETO, combined with record revenues from TAVALISSE and REZLIDHIA, has allowed us to approach net income break even. We look to maintain our financial discipline as we expand our commercial reach and advance our development programs."

Second Quarter 2024 Business Update
Commercial Update

Commercial strength continues with record TAVALISSE and REZLIDHIA bottles shipped to patients and clinics and total bottles sold.
In June 2024, Rigel announced the completion of the transfer of the New Drug Application (NDA) for the U.S. rights to GAVRETO. GAVRETO became commercially available from Rigel in the U.S. beginning June 27, 2024, ahead of the company’s July 1 target for commercial availability.
The following table summarizes total bottles shipped for the second quarter:

TAVALISSE

REZLIDHIA

GAVRETO*

Bottles shipped to patients and clinics

2,672

424

–

Change in bottles remaining in distribution channel

50

(23)

228

Total bottles shipped

2,722

401

228

*GAVRETO bottle count represents 60-count bottle equivalent

Clinical and Development Update

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 fourth dose level (250 mg twice daily) of the trial is underway. Preliminary data are expected by the end of 2024.
In early August, The University of Texas MD Anderson Cancer Center, with Rigel’s support, opened enrollment for a Phase 1b/2 trial of decitabine and venetoclax in combination with olutasidenib in patients with IDH1-mutated AML (NCT06445959). This is the first trial in Rigel’s multi-year strategic development collaboration with MD Anderson. The Phase 1b part of the trial seeks to determine the safety and tolerability and recommended Phase 2 dose of decitabine and venetoclax in combination with olutasidenib. The primary objective of the Phase 2 part of the trial is to determine the complete remission rate in both newly diagnosed and relapsed/refractory patients.
In late July, City of Hope National Medical Center opened enrollment for a pilot trial of olutasidenib as maintenance therapy following allogeneic hematopoietic cell transplantation (HCT). The primary objective of the trial is to evaluate the safety and tolerability of olutasidenib as post-HCT maintenance therapy in patients with mIDH1 AML, myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML).
Rigel presented the final long-term efficacy data from the registrational Phase 2 trial of REZLIDHIA in heavily pretreated patients with R/R mIDH1 AML, including those receiving prior venetoclax, in an oral presentation and 4 posters at the EHA (Free EHA Whitepaper)2024 Hybrid Congress. In addition, the company presented 3 posters at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting, which included safety and efficacy of olutasidenib treatment in elderly patients with R/R mIDH1 AML and an overview of the Phase 1b trial of R289 in patients with LR-MDS.
Dr. Jorge E. Cortes, Director, Georgia Cancer Center, Cecil F. Whitaker Jr., GRA Eminent Scholar Chair in Cancer, and Phase 2 trial investigator, was published in the Expert Review of Hematology in May outlining the drug profile and summarizing key safety and efficacy data for olutasidenib, including in patients previously treated with venetoclax or ivosidenib.
Second Quarter 2024 and Year-To-Date Financial Update
For the second quarter ended June 30, 2024, total revenues were $36.8 million, consisting of $26.4 million in TAVALISSE net product sales, $5.2 million in REZLIDHIA net product sales, $1.9 million in GAVRETO net product sales, and $3.4 million in contract revenue from collaborations. TAVALISSE net product sales grew 24% compared to $21.3 million in the same period of 2023. REZLIDHIA net product sales grew 102% compared to $2.6 million in the same period of 2023. GAVRETO became commercially available from Rigel on June 27, 2024. Contract revenue from collaborations consisted of $2.2 million from Kissei Pharmaceutical Co., Ltd. (Kissei) related to delivery of drug supplies, $1.1 million from Grifols S.A. (Grifols) related to earned royalties, and $0.1 million from Medison Pharma Trading AG (Medison) related to delivery of drug supplies and earned royalties.

Total costs and expenses were $36.4 million compared to $32.2 million for the same period of 2023. The increase in costs and expenses was partly due to higher cost of product sales, driven primarily by higher amortization of intangibles and royalties, increased personnel-related costs, and increased research and development costs due to the progress of clinical activities, including R289, the company’s IRAK 1/4 inhibitor program.

Rigel reported a net loss of $1.0 million, or $0.06 per basic and diluted share, compared to a net loss of $6.6 million, or $0.38 per basic and diluted share, for the same period of 2023. The basic and diluted share and per share amounts have been restated to reflect the 1-for-10 reverse stock split effected on June 27, 2024 on a retroactive basis for all periods presented.

For the six months ended June 30, 2024, total revenues were $66.4 million, consisting of $47.5 million in TAVALISSE net product sales, $10.0 million in REZLIDHIA net product sales, $1.9 million in GAVRETO net product sales, and $6.9 million in contract revenue from collaborations. TAVALISSE net product sales grew 9% compared to $43.6 million in the same period of 2023. REZLIDHIA net product sales grew 150% compared to $4.0 million in the same period of 2023. As mentioned above, GAVRETO became commercially available from Rigel on June 27, 2024. Contract revenue from collaborations consisted of $4.5 million from Kissei related to delivery of drug supplies, $2.2 million from Grifols related to earned royalties, and $0.2 million from Medison related to delivery of drug supplies and earned royalties.

Total costs and expenses were $72.9 million compared to $70.9 million for the same period of 2023. The increase in costs and expenses was partly due to higher cost of product sales, driven primarily by higher amortization of intangibles and royalties, increased personnel-related costs and higher stock-based compensation expenses mainly from performance awards. These increases were partially offset by decreased research and development costs due to the timing of clinical trial activities related to R289, the company’s IRAK 1/4 inhibitor program, as well as reduced trial activities related to the completed Phase 3 clinical trials of fostamatinib in patients with COVID-19 and warm antibody hemolytic anemia (wAIHA).

Rigel reported a net loss of $9.3 million, or $0.53 per basic and diluted share, compared to a net loss of $20.1 million, or $1.16 per basic and diluted share, for the same period of 2023. As discussed above, the share and per share amounts have been restated to reflect the 1-for-10 reverse stock split on a retroactive basis for all periods presented.

Cash, cash equivalents and short-term investments as of June 30, 2024 was $49.1 million, compared to $49.6 million as of March 31, 2024, and $56.9 million as of December 31, 2023.

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
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.

Please click here for Important Safety Information and Full Prescribing Information for TAVALISSE.

About REZLIDHIA
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.

Please click here for Important Safety Information and Full Prescribing Information, including Boxed WARNING, for REZLIDHIA.

About GAVRETO
GAVRETO is indicated for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test and adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).*

*Thyroid indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

Please click here for Important Safety Information and Full Prescribing Information for GAVRETO.

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, REZLIDHIA and GAVRETO are registered trademarks of Rigel Pharmaceuticals, Inc.