On May 8, 2024 Phanes Therapeutics, Inc. (Phanes), a clinical stage biotech company focused on innovative drug discovery and development in oncology, reported that it has entered into a clinical supply agreement with Roche to study PT217, its first-in-class bispecific antibody targeting DLL3 and CD47, in combination with Roche’s anti-PD-L1 therapy, atezolizumab, in patients with small cell lung cancer (SCLC), large cell neuroendocrine carcinoma of the lung (LCNEC) and extrapulmonary neuroendocrine carcinomas (EP-NECs) (Press release, Phanes Therapeutics, MAY 8, 2024, View Source [SID1234642905]). PT217 was granted orphan drug designation (ODD) for the treatment of SCLC by the FDA in 2022, and recently granted Fast Track designation by the agency for the treatment of patients with extensive-stage small cell lung cancer (ES-SCLC) with disease progression following platinum chemotherapy with or without a checkpoint inhibitor.

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Phanes is currently enrolling patients in a multi-center Phase I clinical trial of PT217. The Phase I clinical trial (NCT05652686), known as the SKYBRIDGE study, is currently evaluating the safety, tolerability, pharmacokinetics and preliminary efficacy of PT217 in patients with advanced or refractory cancers expressing DLL3. The next phase of Phanes’ study is investigating the therapeutic potential of PT217 as a combination therapy in SCLC, LCNEC and EP-NECs. The clinical collaboration with Roche will evaluate PT217 in combination with atezolizumab in these patients.

"Phanes is very excited about partnering with Roche on this novel approach to treat patients with SCLC, LCNEC and EP-NECs," said Rita Laeufle, MD, PhD, Chief Medical Officer (CMO) of Phanes. "DLL3 is highly expressed in SCLC, LCNEC and EP-NECs and an important target for treating these cancers. We believe the mechanisms of PT217 and atezolizumab are complementary and the combination has the potential to improve outcomes for patients. This collaboration marks another milestone for Phanes in fulfilling our vision of developing innovative approaches to treat cancer."

TECENTRIQ is a registered trademark of Roche.

On May 8, 2024 Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company developing a novel class of therapeutic biologics to selectively modulate disease-specific T cells, reported that it will deliver two poster presentations at the 20th PEGS Boston Summit, the Essential Protein and Antibody Engineering Summit, being held May 13-17, 2024 in Boston, MA and virtually (Press release, Cue Biopharma, MAY 8, 2024, View Source [SID1234642904]).

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Presentation Details

Date and Time: Monday, May 13 from 7:00 a.m. – 4:30 p.m. and Tuesday, May 14 from 9:00 a.m. – 12:00 p.m. EDT
Session: Poster Session A
Poster number: A028
Title: Immuno-STATs for Targeted Depletion of B Cells in Autoimmune Diseases
Presenter: Nitin Kumar, Scientist, Biologics Discovery and Innovation, Cue Biopharma

Dr. Kumar will discuss Cue Biopharma’s newly deployed CUE-500 series of bispecific Immuno-STAT biologics, designed to redirect and activate cytotoxic anti-viral memory T cells to deplete pathogenic B cells, which is a therapeutically relevant mechanism for the treatment of several autoimmune diseases.

Date and Time: Tuesday, May 14 from 2:00 p.m. – 4:00 p.m. and Wednesday, May 15 from 9:00 a.m. – 7:30 p.m. EDT
Session: Poster Session B
Poster number: B023
Title: Immuno-STATs (ISTs): A Novel and Unique T-Cell Engager Platform for the Treatment of Immuno-Oncology and Autoimmune Diseases
Presenter: Ahmet Vakkasoglu, Associate Director, Biologics Discovery and Innovation, Cue Biopharma

Dr. Vakkasoglu will present an overview of Cue Biopharma’s Immuno-STAT platform and biologics. This will include a discussion of our lead oncology assets, which have demonstrated clinical anti-tumor activity, as well as an overview of our next generation platforms and molecules for the treatment of cancer, autoimmune and inflammatory diseases.

On May 8, 2024 Merus N.V. (Nasdaq: MRUS) (Merus, the Company, we, or our), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics and Triclonics), reported financial results for the first quarter and provided a business update (Press release, Merus, MAY 8, 2024, View Source [SID1234642903]).

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"At the upcoming 2024 ASCO (Free ASCO Whitepaper) meeting, we are looking forward to presenting multiple datasets including the first clinical data on safety and efficacy of petosemtamab in combination with pembrolizumab in previously untreated head and neck cancer. With petosemtamab, we continue to believe we have the opportunity to significantly improve the lives of patients with both previously treated, as well as newly diagnosed, head and neck cancer and thus, it remains the focus of the company’s resources," said Bill Lundberg, M.D., President, Chief Executive Officer of Merus. "Additionally, we are thrilled that Zeno’s BLA has been accepted for priority review, a tremendous milestone for Merus representing our first Biclonics advancing from discovery to marketing application."

Petosemtamab (MCLA-158: EGFR x LGR5 Biclonics): Solid Tumors
1L head & neck squamous cell carcinoma (HNSCC) in combination with pembrolizumab ongoing, presentation at 2024 ASCO (Free ASCO Whitepaper); previously treated (2L+) HNSCC phase 3 registration trial planned to initiate mid-2024 and dose comparison of petosemtamab monotherapy 1100 vs 1500 mg in 2L+ HNSCC ongoing; planned initiation of 2L colorectal cancer (CRC) cohort in 2024

An abstract entitled: Petosemtamab (MCLA-158) with pembrolizumab as first-line (1L) treatment of recurrent/metastatic (r/m) head and neck squamous cell carcinoma (HNSCC): Phase 2 study was accepted for rapid oral session presentation at 2024 ASCO (Free ASCO Whitepaper). Merus plans to report initial interim efficacy and safety data from this cohort.

Merus continues to evaluate patients with untreated advanced PD-L1+ HNSCC treated with petosemtamab 1500 mg in combination with pembrolizumab. Initial safety data from this single arm cohort may support the initiation of a 1L phase 3 trial with this combination. Among the initial patients dosed in the 1L combination cohort, the safety profile has been observed to be generally favorable.

Merus plans to initiate a phase 3 clinical trial in mid-2024 to evaluate petosemtamab monotherapy in 2L+ HNSCC. In the planned trial, patients will be randomized to petosemtamab monotherapy or investigators’ choice of single agent chemotherapy or cetuximab. Merus believes a randomized registration trial in HNSCC with an overall response rate (ORR) endpoint could potentially support accelerated approval and the overall survival (OS) results from the same study could potentially verify its clinical benefit to support regular approval.

Merus continues to evaluate approximately 40 patients treated with petosemtamab monotherapy at either 1100 or 1500 mg dose levels to confirm a suitable dose for future potential phase 3 trials. Merus plans to share clinical data from this cohort in the second half of 2024.

At the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2023, Merus provided interim data on 49 2L+ HNSCC patients that were treated with petosemtamab at the recommended phase 2 dose of 1500 mg intravenous every two weeks. Merus plans to provide updated efficacy, durability and safety data of this cohort in the second half of 2024.

In 2024, Merus is planning to initiate the evaluation of petosemtamab with standard chemotherapy in 2L CRC.

Zenocutuzumab (Zeno or MCLA-128: HER2 x HER3 Biclonics): NRG1 fusion-positive (NRG1+) lung, pancreatic and other solid tumors
Zeno BLA for treatment of NRG1+ NSCLC and PDAC accepted for priority review by the FDA

The U.S. Food and Drug Administration (FDA) has accepted for priority review a Biologics License Application (BLA) for the bispecific antibody zenocutuzumab (Zeno) in patients with NRG1+ non-small cell lung cancer (NSCLC) and pancreatic (PDAC) cancer. If approved, Zeno will be the first and only targeted therapy for patients with NRG1+ NSCLC and PDAC.

The Company is also conducting ongoing translational work on potential biomarkers outside of NRG1+ cancer which may support development opportunities for Zeno in additional areas of unmet need. Merus presented a pre-clinical poster: Zenocutuzumab, a HER2xHER3 bispecific antibody, is effective in cancer models with high NRG1 expression at the AACR (Free AACR Whitepaper) Annual Meeting 2024.

Merus believes that obtaining a commercialization partnership agreement will be an essential step in bringing Zeno to patients with NRG1+ cancer, if approved.

MCLA-129 (EGFR x c-MET Biclonics): Solid Tumors
Investigation of MCLA-129 continues in the MET ex14 NSCLC expansion cohort in the phase 1/2 trial; MCLA-129 in combination with chemotherapy in 2L+ EGFR mutant (EGFRm) NSCLC planned to start in 2024

An abstract entitled: Efficacy and safety of MCLA-129, an anti-EGFR/c-MET bispecific antibody, in non-small-cell lung cancer (NSCLC) with c-MET exon 14 skipping mutations (METex14) was accepted for poster presentation at 2024 ASCO (Free ASCO Whitepaper).

We plan to start a cohort investigating MCLA-129 in combination with chemotherapy in 2L+ EGFRm NSCLC in 2024. We also remain interested in exploring partnering MCLA-129 to sufficiently resource the development of MCLA-129 and potential benefit it may have for patients.

MCLA-129 is subject to a collaboration and license agreement with Betta Pharmaceuticals Co. Ltd. (Betta), which permits Betta to develop MCLA-129 and potentially commercialize exclusively in China, while Merus retains global rights outside of China. An abstract sponsored by Betta entitled: Efficacy and safety of MCLA-129, an EGFR/c-MET bispecific antibody, in advanced non-small cell lung cancer (NSCLC) was accepted for poster presentation at 2024 ASCO (Free ASCO Whitepaper).

MCLA-145 (CD137 x PD-L1 Biclonics): Solid Tumors

Investigation continues of the phase 1 trial of MCLA-145 in combination with pembrolizumab

An abstract entitled: Phase I study of MCLA-145, a bispecific antibody targeting CD137 and PD-L1, in solid tumors, as monotherapy or in combination with pembrolizumab was accepted for rapid oral session presentation at 2024 ASCO (Free ASCO Whitepaper).

Research
At the 20th Annual PEGS Boston meeting on May 14th, Merus plans to present preclinical validation of the compatibility and favorable pharmaceutical properties of Biclonics conjugated with a range of linkers and payloads to generate antibody-drug conjugates (ADClonicsTM), demonstrating our platform and format holds the potential for improved binding selectivity, internalization and cancer cell killing activity.

Company News
Effective May 7, 2024, Jason Haddock was appointed to the Merus Board of Directors. Most recently, he served as Chief Financial Officer (CFO) at Archer Dx from May to August 2020 until it was acquired by Invitae Corporation. From 2016 to 2019, he served as CFO of Array BioPharma, Inc. and from 2015 to 2016, Mr. Haddock served as CFO and Chief Operating Officer (COO) of BERG. Mr. Haddock spent 15 years (2001-2015) at Bristol-Myers Squibb in a variety of finance, strategic, commercial and business development capacities, including CFO and COO roles for business units in Asia Pacific, Europe and the United States. He currently serves on the board of directors of PYC Therapeutics. Mr. Haddock holds a BS in accounting from Illinois State University and an Executive MBA from Washington University in St. Louis.

Collaborations

Incyte Corporation
Since 2017, Merus has been working with Incyte Corporation (Incyte) under a global collaboration and license agreement focused on the research, discovery and development of bispecific antibodies utilizing Merus’ proprietary Biclonics technology platform. For each program under the collaboration, Merus receives reimbursement for research activities and is eligible to receive potential development, regulatory and commercial milestones and sales royalties for any products, if approved. During the first quarter of 2024, Merus achieved a milestone of $1 million for candidate nomination and expects to receive payment in the second quarter of 2024. This is the fifth program to obtain candidate nomination under the collaboration.

Eli Lilly and Company
In January 2021, Merus and Eli Lilly and Company (Lilly), announced a research collaboration and exclusive license agreement to develop up to three CD3-engaging T-cell re-directing bispecific antibody therapies utilizing Merus’ Biclonics platform and proprietary CD3 panel along with the scientific and rational drug design expertise of Lilly. The collaboration is progressing well with three programs ongoing at various stages of preclinical development.

Gilead Sciences
In March 2024, Merus and Gilead Sciences announced a collaboration to discover novel antibody based trispecific T-cell engagers using Merus’ patented Triclonics platform. Under the terms of the agreement, Merus will lead early-stage research activities for two programs, with an option to pursue a third. Gilead will have the right to exclusively license programs developed under the collaboration after the completion of select research activities. If Gilead exercises its option to license any such program from the collaboration, Gilead will be responsible for additional research, development and commercialization activities for such program. Merus received an equity investment by Gilead of $25 million in Merus common shares and an upfront payment of $56 million.

On April 9, 2024, Merus received U.S. Patent Number 11,952,424 covering our proprietary Triclonics format, related to a trispecific antibody comprising a common light chain, capable of binding at least three different epitopes or antigens.

Cash Runway, existing cash, cash equivalents and marketable securities expected to fund Merus’ operations into 2027

As of March 31, 2024, Merus had $398.7 million cash, cash equivalents and marketable securities. Based on the Company’s current operating plan, the existing cash, cash equivalents and marketable securities are expected to fund Merus’ operations into 2027.

First Quarter 2024 Financial Results

We ended the first quarter with cash, cash equivalents and marketable securities of $398.7 million compared to $411.7 million at December 31, 2023. The decrease was primarily the result of cash used to fund the operations partially offset by equity investment from Gilead Sciences.

Collaboration revenue for the three months ended March 31, 2024 decreased by $5.6 million as compared to the three months ended March 31, 2023, primarily as a result of lower cost reimbursement revenue.

Research and development expense for the three months ended March 31, 2024 increased by $3.7 million as compared to the three months ended March 31, 2023, primarily as a result of an increase in clinical and manufacturing costs related to our programs.

General and administrative expense for the three months ended March 31, 2024 increased by $0.7 million as compared to the three months ended March 31, 2023, primarily as a result of increases in personnel related costs partially offset by decrease in facility and consulting costs.

Other income (loss), net consists of interest earned and fees paid on our cash and cash equivalents held on account, accretion of investment earnings and net foreign exchange (losses) gains on our foreign denominated cash, cash equivalents and marketable securities. Other gains or losses relate to the issuance and settlement of financial instruments.

On May 8, 2024 Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) reported financial results for the first quarter of 2024, along with recent product highlights and corporate updates (Press release, Zai Laboratory, MAY 8, 2024, View Source [SID1234642895]).

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"Our first quarter results demonstrate strong commercial execution and pipeline progress across our potential first- and best-in-class product portfolio," said Dr. Samantha Du, Founder, Chairperson, and Chief Executive Officer of Zai Lab. "The launch of VYVGART is off to an impressive start with $13.2 million of sales in the first quarter. Looking ahead, we expect to accelerate commercial performance for the remainder of the year and are preparing for three new potential launches in 2024. We are also excited by the progress of our late-stage pipeline and we are on track to achieve the objectives outlined in our five-year strategic plan, including significant revenue growth and profitability by the end of 2025."

"Our net revenues grew 39% y-o-y or 43% y-o-y at CER in the first quarter, driven by strong execution with the launch of VYVGART and uptake of our existing portfolio," said Josh Smiley, President and Chief Operating Officer of Zai Lab. "With VYVGART’s launch in gMG at the end of last year, and multiple new products and indications expected to launch over the near-term, we are now entering a period of robust growth for Zai Lab. Our significant growth, coupled with our focus on driving efficiencies and productivity across the organization, will drive the evolution of Zai Lab into a profitable, high growth business by the end of 2025. Furthermore, we will continue to focus on expanding our global portfolio through our internal discovery activities and strategic business development," Mr. Smiley concluded.

First-Quarter 2024 Financial Results

•Product revenue was $87.1 million in the first quarter of 2024, compared to $62.8 million for the same period in 2023, representing 39% y-o-y growth and 43% y-o-y growth at CER. This increase was primarily driven by increased sales volumes, including from the launch of VYVGART last September and decreased sales rebates to distributors resulting from price reductions in connection with listings on China’s National Reimbursement Drug List (NRDL) for certain products. This revenue growth included the following:

–ZEJULA: $45.5 million in the first quarter of 2024, an increase of 7% y-o-y from $42.7 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: $13.2 million in the first quarter of 2024, compared to nil for the same period in 2023, driven by 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 generalized myasthenia gravis (gMG) in September 2023 and was subsequently included for first-time NRDL listing effective, January 1, 2024.

–OPTUNE (Tumor Treating Fields): $12.5 million in the first quarter of 2024, a decrease of 6% y-o-y from $13.3 million for the same period in 2023. Although revenue declined y-o-y for OPTUNE, it increased 49% versus the fourth quarter of 2023, with continued recovery of patient volume expected throughout 2024.

–QINLOCK: $6.1 million in the first quarter of 2024, an increase of 367% y-o-y from $1.3 million for the same period in 2023, driven by its inclusion in the NRDL in the first quarter of 2023 for the fourth-line treatment of advanced gastrointestinal stromal tumors (GIST).

–NUZYRA: $9.9 million in the first quarter of 2024, an increase of 81% y-o-y from $5.5 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 $54.6 million in the first quarter of 2024, compared to $48.5 million for the same period in 2023. This increase was primarily due to increased clinical trial expenses related to newly initiated studies and progress of existing studies, partially offset by a decrease in milestone fees for our licensed products.

•Selling, General and Administrative expenses were $69.2 million in the first quarter of 2024, compared to $62.5 million for the same period in 2023. This increase was primarily driven by higher general selling expenses and headcount growth associated with the VYVGART launch.

•Net loss was $53.5 million in the first quarter of 2024, or a loss per ordinary share attributable to common stockholders of $0.05 (or loss per American Deposit Share (ADS) of $0.55), compared to a net loss of $49.1 million for the same period in 2023, or a loss per ordinary share of $0.05 (or loss per ADS of $0.51).

•Cash and cash equivalents, short-term investments, and current restricted cash totaled $750.8 million as of March 31, 2024, compared to $806.5 million as of December 31, 2023.

Corporate Update

•In April 2024, Andrew Zhu joined Zai Lab as our Chief Commercial Officer in Greater China2. Mr. Zhu’s rich experience in building innovative business models and resource integration will help us further enhance our commercial operations and drive sales and profit growth across Greater China. He joins us from Simcere Zaiming, where he most recently served as Chief Operating Officer responsible for the commercial and pharmaceutical business. He previously served in various operational, sales, and marketing leadership roles at leading global biopharmaceutical companies, including AstraZeneca, Roche, Sanofi, and Bristol Myers Squibb (BMS).

Recent Pipeline Highlights

Below are key product updates since our last earnings release:

Oncology Pipeline

•Tumor Treating Fields:
–In March 2024, Zai Lab partner Novocure announced positive topline results from the Phase 3 METIS clinical trial for brain metastases from non-small cell lung cancer (NSCLC). The primary endpoint was met with Tumor Treating Fields therapy and supportive care demonstrating a significant improvement in time to intracranial progression versus supportive care alone (21.9 months median versus 11.3 months, respectively). These results will be presented as a late-breaking abstract at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago on June 3. We are participating in the Greater China portion of the METIS trial.

2

•Bemarituzumab (FGFR2b):
–We are enrolling patients in Greater China for the global Phase 3 FORTITUDE-101 and FORTITUDE-102 studies:
◦FORTITUDE-101 is a Phase 3 study of bemarituzumab plus chemotherapy in first-line gastric cancer.
◦FORTITUDE-102 is a Phase 1b/3 study of bemarituzumab plus chemotherapy and nivolumab in first-line gastric cancer.

•Tisotumab Vedotin (Tissue Factor ADC):
–In April 2024, Zai Lab partner Pfizer Inc. and Genmab A/S announced that the U.S. Food and Drug Administration (FDA) approved the supplemental Biologics License Application (sBLA) granting full approval for tisotumab vedotin (or TIVDAK) for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy. We are participating in the global Phase 3 innovaTV 301 trial and extension study in Greater China.

•Adagrasib (KRASG12C):
–We are evaluating the clinical data of the global Phase 3 KRYSTAL-12 study evaluating adagrasib in previously treated patients with KRASG12C-mutated NSCLC as we decide on next steps in the development of this product across indications.

•ZL-1310 (DLL3 ADC):
–In March 2024, Zai Lab presented findings from preclinical studies highlighting the therapeutic potential of ZL-1310 at the European Lung Cancer Congress (ELCC) 2024.
–We are enrolling patients in the United States and Greater China in the global Phase 1 study in relapsed and refractory second-line+ small cell lung cancer (SCLC) who have progressed after platinum-based treatment.

•ZL-1218 (CCR8):
–We are enrolling patients in the United States, Europe, and Greater China in the global Phase 1 study of ZL-1218 as a single agent and in combination with pembrolizumab in patients with advanced solid tumor malignancies.

Autoimmune Disorders, Infectious Disease, and Neuroscience Pipeline

•Efgartigimod (FcRn):
–In April 2024, Zai Lab submitted an sBLA for efgartigimod SC for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) to the National Medical Products Administration (NMPA).

•Xanomeline-Trospium (or KarXT) (M1/M4-agonist):
–In April 2024, Zai Lab partner BMS presented new interim long-term data from the Phase 3 EMERGENT program at the Annual Congress of the Schizophrenia International Research Society (SIRS).
◦In the new interim analysis of long-term efficacy data from the Phase 3 EMERGENT-4 open-label extension trial, KarXT was associated with significant improvement in symptoms of schizophrenia across all efficacy measures at 52 weeks.
◦In the new pooled interim long-term safety and metabolic outcomes from the Phase 3 EMERGENT-4 and EMERGENT-5 trials, KarXT demonstrated a favorable long-term metabolic profile where most patients experienced stability or improvements on metabolic parameters over 52 weeks of treatment.
–We are enrolling patients in a registrational bridging study in mainland China.

Anticipated Major Milestones in 2024

Oncology

Repotrectinib
•Potential NMPA approval of our NDA for the treatment of adult patients with locally advanced or metastatic ROS1-positive NSCLC.

Tumor Treating Fields
•Zai Lab to submit a Marketing Authorization Application (MAA) to the NMPA in second-line+ NSCLC, following progression on or after platinum-based therapy.
•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.

3

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.

Neuroscience, Autoimmune Disorders, and Infectious Diseases (NSAiID)

Efgartigimod (FcRn)
•Potential NMPA approval of the BLA for efgartigimod SC for gMG.
•We plan to join in the registrational study of efgartigimod SC in Thyroid Eye Disease (TED) in Greater China in the second half of 2024.

Sulbactam-Durlobactam (SUL-DUR)
•Potential NMPA approval of our NDA for infections caused by susceptible isolates of Acinetobacter baumannii-calcoaceticus complex (ABC).

Xanomeline-Trospium (KarXT) (M1/M4-agonist)
•Zai Lab to complete patient enrollment in the China bridging study in schizophrenia.
•Zai Lab to join the global Phase 3 ADEPT-2 and ADEPT-3 studies in Alzheimer’s disease with psychosis in Greater China in mid-year.
•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.

ZL-1102 (IL-17 Humabody)
•Zai Lab to initiate a global Phase 2 study in mild-to-moderate chronic plaque psoriasis in the second quarter of 2024.

Conference Call and Webcast Information

Zai Lab will host a live conference call and webcast tomorrow, May 9, 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/BIb8622a7cf98e46cd9bc9198a5f105c36

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 May 8, 2024 Syndax Pharmaceuticals (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the quarter ended March 31, 2024, and provided a business update (Press release, Syndax, MAY 8, 2024, View Source [SID1234642894]).

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"With the potential near-term approvals of revumenib and axatilimab in the third quarter as well as pivotal data from the mNPM1 cohort of the AUGMENT-101 trial in the fourth quarter, the Company is on track to have a historic year punctuated by major value-creating milestones," said Michael A. Metzger, Chief Executive Officer. "Syndax is unparalleled as a SMID cap oncology company with the potential launch of two first- and best-in-class agents into multi-billion-dollar markets with the opportunity for expansion beyond their initial indications. We remain keenly focused on laying the foundation and building an experienced team of experts to ensure our successful transition into a commercial organization."

Recent Pipeline Progress and Anticipated Milestones

Revumenib

In March 2024, the Company announced that the FDA had granted Priority Review for the New Drug Application (NDA) filing for revumenib, a potent, selective small molecule menin inhibitor, for the treatment of adult and pediatric relapsed or refractory (R/R) KMT2A-rearranged (KMT2Ar) acute leukemia. The NDA filing is being reviewed under the FDA’s Real-Time Oncology Review Program (RTOR) and has been assigned a Prescription Drug User Fee Act (PDUFA) target action date of September 26, 2024. RTOR allows for a more efficient review and close engagement between the sponsor and the FDA throughout the submission process, which historically has led to earlier approvals.
In March 2024, the Company also announced the completion of enrollment in the AUGMENT-101 pivotal trial cohort of patients with R/R mutant nucleophosmin (mNPM1) acute myeloid leukemia (AML). Topline data is expected in the fourth quarter of 2024 and could support a supplemental NDA (sNDA) filing for revumenib in R/R mNPM1 AML in the first half of 2025.
Positive results from a subset of the pivotal AUGMENT-101 trial in pediatric patients with R/R KMT2Ar AML and acute lymphoid leukemia (ALL) treated with revumenib were featured in a plenary session at the American Society of Pediatric Hematology/Oncology (ASPHO) Annual Meeting in April 2024.
Multiple Phase 1 combination trials of revumenib in mNPM1 and KMT2Ar acute leukemias are ongoing across the treatment landscape. The trials are expanding to validate recommended Phase 2 doses, with additional data expected in the second half of 2024. These trials include:
BEAT AML: Evaluating the combination of revumenib with venetoclax and Azacytidine in front-line AML patients. This trial is being conducted as part of the Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial.
SAVE: Evaluating the all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in R/R AML or mixed phenotype acute leukemias. The trial is being conducted by investigators from the MD Anderson Cancer Center.
AUGMENT-102: Evaluating the combination of revumenib with fludarabine and cytarabine in patients with R/R acute leukemias.
A Phase 1 trial of revumenib in combination with 7+3 chemotherapy followed by maintenance treatment in newly diagnosed patients with mNPM1 or KMT2Ar acute leukemias was initiated during the quarter.
The Company plans to initiate a pivotal trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar acute leukemia patients unfit to receive intensive chemotherapy by year-end 2024.
Enrollment is ongoing in a Phase 1 proof-of-concept clinical trial of revumenib in patients with unresectable metastatic microsatellite stable colorectal cancer. The Company expects to provide an update on the trial in the second quarter of 2024.
Axatilimab

In February, the Company announced that the FDA had accepted the Biologics License Application (BLA) filing for axatilimab, an anti-CSF-1R antibody, in patients with chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy. The application was granted Priority Review and assigned a PDUFA action date of August 28, 2024.
Enrollment is ongoing in a 26-week randomized, double-blinded, placebo-controlled Phase 2 trial of axatilimab on top of standard of care in patients with idiopathic pulmonary fibrosis (IPF).
Our partner Incyte plans to initiate two combination trials with axatilimab in chronic GVHD in 2024, including a Phase 2 combination trial with ruxolitinib and a Phase 3 combination trial with steroids.
Corporate Updates

In March 2024, the Company announced the appointment of Steven Closter as Chief Commercial Officer. Mr. Closter brings to Syndax more than 30 years of commercial experience in the biopharmaceutical industry.
First Quarter 2024 Financial Results

As of March 31, 2024, Syndax had cash, cash equivalents, and short and long-term investments of $522.0 million and 85.3 million common shares and prefunded warrants outstanding.

First quarter 2024 research and development expenses increased to $56.5 million from $34.1 million for the comparable prior year period. The increase in research and development expenses was primarily due to increased clinical development and manufacturing costs, increased employee-related expenses and professional fees, and development milestone expense recognized in the current period.

First quarter 2024 selling, general and administrative expenses increased to $23.0 million from $12.0 million for the comparable prior year period. The increase in selling, general and administrative expenses was primarily due to increased employee-related expenses and professional fees as well as increased commercialization activities for revumenib and axatilimab.

For the three months ended March 31, 2024, Syndax reported a net loss attributable to common stockholders of $72.4 million, or $0.85 per share, compared to a net loss attributable to common stockholders of $41.1 million, or $0.59 per share, for the comparable prior year period.

Financial Guidance

For the second quarter of 2024, the Company expects research and development expenses to be $50 to $55 million and total operating expenses to be $80 to $85 million. For the full year of 2024, the Company continues to expect research and development expenses to be $240 to $260 million and total operating expenses to be $355 to $375 million, which includes an estimated $43 million in non-cash stock compensation expense.

The Company believes that it has sufficient cash runway to fund its research, clinical development and commercial operations through 2026.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 8:00 a.m. ET today, Wednesday, May 8, 2024.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website. Alternatively, the conference call may be accessed through the following:

Conference ID: Syndax1Q24
Domestic Dial-in Number: 800-590-8290
International Dial-in Number: 240-690-8800
Live webcast: https://www.veracast.com/webcasts/syndax/events/SNDX1Q24.cfm

For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company’s website at www.syndax.com approximately 24 hours after the conference call and will be available for 90 days following the call.

About Revumenib

Revumenib is a potent, selective, small molecule inhibitor of the menin-KMT2A binding interaction that is being developed for the treatment of KMT2A-rearranged (KMT2Ar), also known as mixed lineage leukemia rearranged or MLLr, acute leukemias including ALL and AML, and mutant nucleophosmin (mNPM1) AML. Positive topline results from the Phase 2 AUGMENT-101 trial in R/R KMT2Ar acute leukemia showing the trial met its primary endpoint were presented at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, and data from the Phase 1 portion of AUGMENT-101 in acute leukemia was published in Nature. Revumenib was granted Orphan Drug Designation by the FDA and European Commission for the treatment of patients with AML and Fast Track designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation. Revumenib was granted Breakthrough Therapy Designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement.

About Axatilimab

Axatilimab is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor has been shown to reduce the number of disease-mediating macrophages along with their monocyte precursors, which has been shown to play a key role in the fibrotic disease process underlying diseases such as chronic GVHD and IPF. Positive topline results from the Phase 2 AGAVE-201 trial showing the trial met its primary endpoint were recently presented at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, and Phase 1/2 data of axatilimab in chronic GVHD were published in the Journal of Clinical Oncology. Axatilimab was granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with chronic GVHD and IPF. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab. Syndax has exercised its option under the collaboration agreement to co-commercialize axatilimab in the U.S. and will provide 30% of the commercial effort. Axatilimab is being developed under an exclusive worldwide license from UCB entered into between Syndax and UCB in 2016.

About the Real-Time Oncology Review Program (RTOR)

RTOR provides a more efficient review process for oncology drugs to ensure that safe and effective treatments are available to patients as early as possible, while improving review quality and engaging in early iterative communication with the applicant. Specifically, it allows for close engagement between the sponsor and the FDA throughout the submission process and it enables the FDA to review individual sections of modules of a drug application rather than requiring the submission of complete modules or a complete application prior to initiating review. Additional information about RTOR can be found at: View Source