On May 9, 2024 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, reported business updates and reported financial results for the first quarter ended March 31, 2024 (Press release, Verastem, MAY 9, 2024, View Source [SID1234643042]).

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"In the first quarter of 2024, we received FDA Orphan Drug Designation for avutometinib and defactinib combination in recurrent low-grade serous ovarian cancer, which recognizes this rare cancer as different and distinct from other forms of ovarian cancer and reinforces the need for new treatment options," said Dan Paterson, president and chief executive officer of Verastem Oncology. "We look forward to starting our planned rolling NDA submission and sharing topline data for avutometinib and defactinib combination in recurrent low-grade serous ovarian cancer. We also plan to announce initial data from the RAMP 205 trial in first-line metastatic pancreatic cancer at ASCO (Free ASCO Whitepaper) and plan to provide updates across our other clinical programs in the second half of 2024."

First Quarter 2024 and Recent Updates

Avutometinib and Defactinib Combination in Low-Grade Serous Ovarian Cancer (LGSOC)

Enrollment and site activations are underway in the U.S., Australia, and the UK, for the international confirmatory Phase 3 RAMP 301 trial evaluating the avutometinib and defactinib combination versus standard of care chemotherapy or hormonal therapy for the treatment of recurrent LGSOC.
Granted Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for avutometinib alone or in combination with defactinib for the treatment of all patients with recurrent LGSOC, in March 2024.
Multiple abstracts were selected for oral and poster presentations at the Society of Gynecologic Oncology (SGO) 2024 Annual Meeting on Women’s Cancer on March 16-18 in San Diego. These presentations included a late-breaking oral presentation on a planned subgroup analysis of Part A of the Phase 2 RAMP 201 trial of avutometinib and defactinib combination of heavily pretreated patients with LGSOC and a plenary oral presentation of preclinical efficacy data of avutometinib in combination with a focal adhesion kinase (FAK) inhibitor in recurrent LGSOC as well as a trials-in-progress poster about the Phase 3 RAMP 301 trial.
Plan to announce updated topline data from the RAMP 201 trial in LGSOC to coincide with the start of our planned rolling New Drug Application (NDA) submission for Accelerated Approval for the avutometinib and defactinib combination in recurrent LGSOC in Q2 2024.
Preparations for a potential U.S. commercial launch in 2025 are ongoing and plans to initiate discussions with European and Japanese regulatory authorities to address patient needs outside the U.S. continue to advance.
Avutometinib in Combination with KRAS G12C Inhibitors in Non-Small Cell Lung Cancer (NSCLC)

Verastem Oncology announced today it has received Fast Track Designation from the FDA for avutometinib in combination with Mirati’s (BMS) G12C inhibitor, KRAZATI (adagrasib) for the treatment of patients with KRAS G12C-mutated metastatic NSCLC who have received at least one prior systemic therapy and have not been previously treated with a KRAS G12C inhibitor, in April 2024.
Verastem Oncology announced today it has received Fast Track Designation from the FDA for the combination of avutometinib plus defactinib with Amgen’s G12C inhibitor, LUMAKRAS (sotorasib) for the treatment of patients with KRAS G12C-mutated metastatic NSCLC who have received at least one prior systemic therapy, in April 2024.
The FDA granted Fast Track Designation for avutometinib in combination with Amgen’s G12C inhibitor, LUMAKRAS (sotorasib), for the treatment of patients with KRAS G12C-mutant metastatic NSCLC who have received at least one prior systemic therapy and have not been previously treated with a KRAS G12C inhibitor, in January 2024.
Data updates from patients with KRAS G12C-mutant NSCLC in the Phase 1/2 RAMP 203 trial evaluating avutometinib plus defactinib and sotorasib are planned for H2 2024.
Data from patients with KRAS G12C-mutant NSCLC in the Phase 1/2 RAMP 204 trial evaluating avutometinib and adagrasib are planned for H2 2024.
Avutometinib and Defactinib Combination in First-Line Metastatic Pancreatic Cancer

Verastem Oncology reported the acceptance of an abstract that will include initial safety and efficacy results from the RAMP 205 trial of avutometinib and defactinib in combination with current standard of care gemcitabine and nab-paclitaxel in first-line metastatic pancreatic cancer at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
GFH375 (VS-7375): Oral KRAS G12D (ON/OFF) Inhibitor

GenFleet Therapeutics investigational new drug (IND) application for GFH375 (VS-7375) was submitted in China and accepted for review. Expect to begin a Phase 1 trial in China in H2 2024.
Discovery/lead optimization continues for second and third programs with GenFleet collaboration.
Upcoming Presentations

Verastem Oncology reported the acceptance of an abstract for poster presentation at the ASCO (Free ASCO Whitepaper) Annual Meeting being held from May 31 to June 4, 2024, in Chicago, IL.

Title: Avutometinib/defactinib and gemcitabine/nab-paclitaxel combination in first-line metastatic pancreatic ductal adenocarcinoma: Initial safety and efficacy of phase 1b/2 study (RAMP 205).
Abstract Number: 4140
Date/Time: Saturday, June 1, 2024, 1:30 to 4:30 pm CDT
Corporate Updates

Strengthened the executive leadership team with the appointment of John Hayslip, M.D., to chief medical officer in April 2024.
First Quarter 2024 Financial Results

Verastem Oncology ended the first quarter of 2024 with cash, cash equivalents and investments of $110.1 million.

Total operating expenses for the three months ended March 31, 2024 (the "2024 Quarter") were $28.1 million, compared to $19.3 million for the three months ended March 31, 2023 (the "2023 Quarter").

Research & development expenses for the 2024 Quarter were $17.7 million, compared to $12.0 million for the 2023 Quarter. The increase of $5.7 million, or 47.5%, was primarily related to increased contract research organization costs, increased investigator fees and increased personnel costs, including non-cash stock compensation.

Selling, general & administrative expenses for the 2024 Quarter were $10.4 million, compared to $7.3 million for the 2023 Quarter. The increase of $3.1 million, or 42.5%, was primarily related to additional costs in anticipation of a potential launch of avutometinib and defactinib in LGSOC, increased personnel costs, including non-cash stock compensation, and increased consulting and professional fees.

Net loss for the 2024 Quarter was $33.9 million, or $1.26 per share (basic and diluted), compared to $15.7 million, or $0.94 per share (basic and diluted, each as adjusted for the Company’s reverse stock split) for the 2023 Quarter.

For the 2024 Quarter, non-GAAP adjusted net loss was $26.2 million, or $0.98 per share (diluted) compared to non-GAAP adjusted net loss of $17.8 million, or $1.07 per share (diluted, as adjusted for the Company’s reverse stock split), for the 2023 Quarter. Please refer to the GAAP to non-GAAP Reconciliation attached to this press release.

Use of Non-GAAP Financial Measures

To supplement Verastem Oncology’s condensed consolidated financial statements, which are prepared and presented in accordance with generally accepted accounting principles in the United States (GAAP), the Company uses the following non-GAAP financial measures in this press release: non-GAAP adjusted net loss and non-GAAP net loss per share. These non-GAAP financial measures exclude certain amounts or expenses from the corresponding financial measures determined in accordance with GAAP. Management believes this non-GAAP information is useful for investors, taken in conjunction with the Company’s GAAP financial statements, because it provides greater transparency and period-over- period comparability with respect to the Company’s operating performance and can enhance investors’ ability to identify operating trends in the Company’s business. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of the Company’s operating results as reported under GAAP, not in isolation or as a substitute for, or superior to, financial information prepared and presented in accordance with GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these non-GAAP financial measures and the most comparable GAAP financial measures for the three months ended March 31, 2024 and 2023 are included in the tables accompanying this press release after the unaudited condensed consolidated financial statements.

About the Avutometinib and Defactinib Combination

Avutometinib is a RAF/MEK clamp that induces inactive complexes of MEK with ARAF, BRAF and CRAF potentially creating a more complete and durable anti-tumor response through maximal RAS/MAPK pathway inhibition. In contrast to currently available MEK-only inhibitors, avutometinib blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows avutometinib to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of other MEK-only inhibitors. The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation of the investigational combination of avutometinib and defactinib, a selective FAK inhibitor, for the treatment of all patients with recurrent low-grade serous ovarian cancer (LGSOC) regardless of KRAS status after one or more prior lines of therapy, including platinum-based chemotherapy. Avutometinib alone or in combination with defactinib was also granted Orphan Drug Designation by the FDA for the treatment of LGSOC.

Verastem Oncology is currently conducting clinical trials with avutometinib in RAS/MAPK driven tumors as part of its Raf And Mek Program or RAMP. RAMP 301 (NCT06072781) is an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent LGSOC. RAMP 201 (NCT04625270) is a Phase 2 registration-directed trial of avutometinib in combination with defactinib in patients with recurrent LGSOC and enrollment has been completed in each of the dose optimization and expansion phases and the low-dose evaluation.

Verastem Oncology has established clinical collaborations with Amgen and Mirati to evaluate LUMAKRAS (sotorasib) in combination with avutometinib and defactinib and KRAZATI (adagrasib) in combination with avutometinib in KRAS G12C mutant NSCLC as part of the RAMP 203 (NCT05074810) and RAMP 204 (NCT05375994) trials, respectively. The RAMP 205 (NCT05669482), a Phase 1b/2 clinical trial evaluating avutometinib and defactinib with gemcitabine/nab-paclitaxel in patients with front-line metastatic pancreatic cancer, is supported by the PanCAN Therapeutic Accelerator Award.

About GFH375 (VS-7375)

GFH375 (VS-7375) is a potential best-in-class, potent and selective oral KRAS G12D (ON/OFF) inhibitor, identified as the lead program from the Verastem Oncology discovery and development collaboration with GenFleet Therapeutics. Upon approval of the investigational new drug (IND) application in China (which is currently under review), GenFleet is expected to initiate a Phase 1 trial in China in the second half of 2024. The collaboration includes three discovery programs, the first being the KRAS G12D inhibitor, and will provide Verastem Oncology with exclusive options to obtain licenses to each of the three compounds in the collaboration after successful completion of pre-determined milestones in Phase 1 trials. The licenses would give Verastem Oncology development and commercialization rights outside of the GenFleet territories of mainland China, Hong Kong, Macau, and Taiwan.

On May 9, 2024 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported financial results for its fiscal 2024 second quarter ended March 31, 2024 (Press release, Arrowhead Pharmaceuticals, MAY 9, 2024, View Source [SID1234643041]). The Company is hosting a conference call today, May 9, 2024, at 4:30 p.m. ET to discuss the results.

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Christopher Anzalone, Ph.D., President and CEO at Arrowhead, said: "Arrowhead has achieved significant progress across our broad pipeline of investigational RNAi-based medicines that leverage the proprietary TRiMTM platform and we continued to strengthen our focus on and investment in our late-stage cardiometabolic programs. As we approach completion of the PALISADE Phase 3 study of plozasiran and initiate additional Phase 3 trials of both plozasiran and zodasiran, we will continue to efficiently execute our clinical studies. Simultaneously, we plan to begin the regulatory submission process, refine our commercial strategy, and build the commercial infrastructure to support it."

Webcast and Conference Call and Details

Investors may access a live audio webcast on the Company’s website at View Source A replay of the webcast will be available approximately two hours after the conclusion of the call.

For analysts that wish to participate in the conference call, please register at https://register.vevent.com/register/BIf9305354ec6b44e3b3e946792a393a5e. Once registered, you will receive the dial-in number and a personalized PIN code that will be required to access the call.

Selected Recent Events

Received a $50 million milestone payment from Royalty Pharma plc, which was paid in the third quarter of fiscal 2024, following the completion of enrollment of the Phase 3 OCEAN(a) – Outcomes Trial of olpasiran, being conducted by Amgen. Olpasiran, a small interfering RNA originally developed by Arrowhead using its proprietary Targeted RNAi Molecule (TRiMTM) platform, is designed to lower levels of lipoprotein(a), a genetically determined risk factor for cardiovascular disease.

Presented final data from the Phase 2 SHASTA-2 study of investigational plozasiran in patients with severe hypertriglyceridemia in a late-breaking oral presentation at the American College of Cardiology 73rd Annual Scientific Session & Expo and simultaneously published in the journal JAMA Cardiology. Key results included the following:
Treatment with plozasiran led to dose-dependent placebo-adjusted reductions in triglycerides (primary endpoint) of -49% (P < 0.001), -53% (P < 0.001), and -57% (P < 0.001), driven by placebo-adjusted reductions in APOC3 of -68% (P < 0.001), -72% (P < 0.001), and -77% (P < 0.001) at week 24, after receiving two doses of 10 mg, 25 mg, and 50 mg plozasiran, respectively. Mean maximum, non-placebo adjusted reductions from baseline in triglycerides and APOC3 were up to 86% and 90% and typically occurred around week 16 or week 20.
Among patients treated with plozasiran, 90.6% achieved a triglyceride level less than 500 mg/dL, the level associated with increased risk of acute pancreatitis, at week 24. In addition, 48.4% of patients achieved normal triglyceride levels of less than 150 mg/dL at week 24.

Subjects treated with plozasiran also showed improvements in multiple atherogenic lipid and lipoprotein levels, including remnant cholesterol, HDL-cholesterol, and non-HDL cholesterol.
Plozasiran demonstrated a favorable safety profile in the SHASTA-2 study. The adverse event and serious adverse event profile were similar across treatment groups. Observed adverse events generally reflected the comorbidities and underlying conditions of the study population.

Initiated an Expanded Access Program (EAP) to make investigational plozasiran available outside of a clinical trial for patients with familial chylomicronemia syndrome (FCS) who meet certain program eligibility criteria.
The plozasiran EAP is for individuals living with FCS. As with any investigational medicine that has not been approved by regulatory authorities, investigational plozasiran may or may not be effective in treating your diagnosis or condition, and there may be risks associated with its use. If you are a patient or caregiver wishing to know more about this plozasiran EAP for FCS, please discuss this EAP and all treatment options with your treating physician. If you are a treating physician and are seeking information about the plozasiran EAP or would like to request access for a patient, please contact [email protected].

Launched the 2024 Summer Series of R&D webinars to highlight specific therapeutic areas in Arrowhead’s pipeline. Each event will feature presentations by Arrowhead team members and external key opinion leaders, who will discuss the respective disease areas and treatment landscapes. 2024 Summer Series Schedule:
May 23, 2024 – Muscular
June 25, 2024 – Cardiometabolic
July 16, 2024 – Pulmonary
August 15, 2024 – Obesity/Metabolic
September 25, 2024 – Central Nervous System
Dosed the first subjects in a Phase 1/2a clinical trial (NCT06209177) of ARO-CFB, designed to reduce hepatic expression of complement factor B, and is being developed as a potential treatment for diseases associated with activation of the complement pathway.
Dosed the first subjects in a Phase 1/2a clinical trial (NCT06138743) of ARO-DM1, designed to reduce expression of the dystrophia myotonica protein kinase gene in the muscle, and is being developed as a potential treatment for type 1 myotonic dystrophy, the most common adult-onset muscular dystrophy.
Strengthened the balance sheet through an underwritten registered offering of common stock for gross proceeds of approximately $450 million, before deducting underwriting discounts, commissions, and other offering expenses payable by the company.
Selected Fiscal 2024 Second Quarter Financial Results

ARROWHEAD PHARMACEUTICALS, INC.
CONSOLIDATED CONDENSED FINANCIAL INFORMATION
(in thousands, except per share amounts)

OPERATING SUMMARY

Three Months Ended

March 31,

2024

2023

(unaudited)

Revenue

$

–

$

146,267

Operating Expenses:

Research and development

101,122

74,881

General and administrative expenses

25,069

23,221

Total operating expenses

126,191

98,102

Operating (loss) income

(126,191

)

48,165

Total other expense

(805

)

(489

)

(Loss) income before income tax expense and noncontrolling interest

(126,996

)

47,676

Income tax expense

–

Net (loss) income including noncontrolling interest

(126,996

)

47,676

Net (loss) income attributable to noncontrolling interest, net of tax

(1,696

)

(999

)

Net (loss) income attributable to Arrowhead Pharmaceuticals, Inc.

$

(125,300

)

$

48,675

Net (loss) income per share attributable to Arrowhead Pharmaceuticals, Inc. – Diluted

$

(1.02

)

$

0.45

Weighted-average shares used in calculating – Diluted

123,285

108,143

FINANCIAL POSITION SUMMARY

March 31, 2024

September 30, 2023

(unaudited)

Cash, cash equivalents and restricted cash

$

127,704

$

110,891

Investments

395,410

292,735

Total cash resources (cash and investments)

523,114

403,626

Other assets

432,036

361,926

Total Assets

$

955,150

$

765,552

Current deferred revenue

$

–

$

866

Other liabilities

459,745

477,524

Total Liabilities

$

459,745

$

478,390

Total Arrowhead Pharmaceuticals, Inc. Stockholders’ Equity

$

483,794

$

271,343

Noncontrolling Interest

11,611

15,819

Total Noncontrolling Interest and Stockholders’ Equity

$

495,405

$

287,162

Total Liabilities, Noncontrolling Interest and Stockholders’ Equity

$

955,150

$

765,552

Shares Outstanding

124,133

107,312

On May 9, 2024 Kite, a Gilead Company (NASDAQ: GILD), and Arcellx, Inc. (NASDAQ: ACLX) reported several key operational updates on their partnered anitocabtagene autoleucel (anito-cel) multiple myeloma program (Press release, Gilead Sciences, MAY 9, 2024, View Source [SID1234643040]). Anito-cel is the first BCMA CAR T to be investigated in multiple myeloma that utilizes Arcellx’s novel and compact D-Domain binder.

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The companies shared the design of a global, Phase 3 randomized controlled clinical trial, iMMagine-3, which Kite expects to start in the second half of this year. The trial will compare the efficacy and safety of anito-cel randomized against the standard of care (SOC) in patients with relapsed and/or refractory multiple myeloma (rrMM) who have received one to three prior lines of therapy, including an immunomodulatory drug (lMiD) and an anti-CD38 monoclonal antibody.

Kite’s facility in Frederick, Maryland will manufacture anito-cel for this trial. This follows the completion of the technical transfer from a third-party contract manufacturing organization to Kite, as well as the transfer of the Investigational New Drug (IND) application for anito-cel, which has been cleared by the U.S. Food and Drug Administration.

"We are pleased to start the Phase 3 pivotal trial, iMMagine-3, in the second half of this year given the tremendous unmet need that remains in patients with relapsed and/or refractory multiple myeloma," said Cindy Perettie, Executive Vice President, Kite. "As we prepare for this pivotal program, we look forward to leveraging our manufacturing expertise to further position anito-cel as a potential best-in-class cell therapy. We know manufacturing quality, reliability and speed are critically important as every day matters for these patients."

"Our global iMMagine-3 trial will evaluate anito-cel as a second through fourth line treatment in patients with multiple myeloma who were previously exposed to both an immunomodulatory drug and an anti-CD38 monoclonal antibody," said Rami Elghandour, Arcellx’s Chairman and Chief Executive Officer. "The iMMagine-3 study allows us to maximize the impact of anito-cel as it captures what will become the largest second line patient population based on the current treatment paradigm, as anti-CD38 therapies move to front line treatment. This population represents an emerging significant unmet clinical need allowing us to provide access to a unique patient population. In addition, the completion of the technical transfer to Kite allowed us to accelerate our development program and launch iMMagine-3 globally, which will enable broader and earlier patient access to anito-cel."

About iMMagine-3 Global Phase 3 Randomized Controlled Clinical Trial

iMMagine-3 is a phase 3, randomized controlled trial designed to compare the efficacy and safety of anitocabtagene autoleucel (anito-cel) with SOC in patients with relapsed and/or refractory multiple myeloma (rrMM) who have received one to three prior lines of therapy, including an immunomodulatory drug (lMiD) and an anti-CD38 monoclonal antibody.

iMMagine-3 will enroll approximately 450 adult patients. Prior to randomization, investigator’s choice of SOC regimens include: pomalidomide, bortezomib, and dexamethasone (PVd); daratumumab, pomalidomide, and dexamethasone (DPd); carfilzomib, daratumumab and dexamethasone (KDd); or carfilzomib and dexamethasone (Kd). Patients in the anito-cel arm will undergo leukapheresis and optional bridging therapy (with the SOC regimen selected by the investigator prior to randomization) followed by lymphodepleting chemotherapy (fludarabine 30 mg/m2/d and cyclophosphamide 300 mg/m2/d for 3 days) and one infusion of anito-cel (115×106 CAR+ T cells) on Day 1.

The primary endpoint is progression free survival (PFS) per blinded independent review according to the 2016 IMWG uniform response criteria for MM with the hypothesis that anito-cel will prolong PFS compared to SOC. Key secondary endpoints include complete response rate (CR/sCR), minimal residual disease negativity, overall survival, and safety.

The iMMagine-3 trial is expected to initiate in the second half of 2024 at ~130 study sites across North America, Europe, and rest of world.

On May 9, 2024 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a clinical stage biotechnology company whose proprietary INTASYL siRNA gene silencing technology is designed to make immune cells more effective in killing tumor cells, reported its Q1 2024 financial results and provided a business update (Press release, Phio Pharmaceuticals, MAY 9, 2024, View Source [SID1234643036]).

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Recent Corporate Updates

· Phio’s INTASYL compound PH-762 is currently being investigated in an open-label Phase 1b clinical study (NCT 06014086) to evaluate the safety and tolerability of intratumoral PH-762 in cutaneous squamous cell (cSCC), melanoma, or Merkel cell carcinoma. Two patients have already completed treatment. Four sites across the US are now engaged in the Phase 1b study. The sites are:
o George Washington University in Washington DC
o Banner MD Anderson in Arizona
o Centricity Research in Ohio
o Integrity Research in Florida.
· Presented new data on the immunotherapeutic activity of INTASYL at:
o 10th Annual Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Conference (ITOC10): this preclinical data demonstrates the potential of the INTASYL compound PH-905 targeting Cbl-b to improve the function of natural killer (NK) cells.
o 27th Annual American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper): this preclinical data demonstrates that intratumoral injection of PH-762 significantly inhibits tumor growth in murine cells and may generate memory-specific T cells.
· The INTASYL siRNA platform is the focus of a National Spotlight on PBS Viewpoint, a national program hosted by Dennis Quaid, and on Fox Business Network; both are airing through May.
· Patent granted by USPTO for two of its INTASYL Compounds, RXI-185 and RXI-231 that treat age-related skin disorders including photo-aging and dermal hyperpigmentation targeting down-regulation of the Matrix metalloproteinase-1(MMP-1) and Tyrosinase (TYR) proteins.

Financial Results

Cash Position

At March 31, 2024, we had cash of $6.5 million as compared with $8.5 million at December 31, 2023.

Research and Development Expenses

Research and development expenses were $1.1 million for the three months ended March 31, 2024 as compared with $2.1 million for the three months ended March 31, 2023, a decrease of 46%. The decrease was primarily driven by the Company’s cost rationalization measures in transitioning from a discovery research company to a product development company resulting in decreased costs for the wind-down of preclinical studies, salary-related costs and lab supplies. Additional decreases in research and development expenses were due to clinical consulting fees incurred in connection with the Company’s IND filing and manufacturing fees for PH-762 in the prior year period.

General and Administrative Expenses

General and administrative expenses were $1.1 million for the three months ended March 31, 2024 as compared with $1.5 million for the three months ended March 31, 2023, a decrease of 28%. The decrease was primarily due to decreases in consulting expenses and legal expenses as compared to the prior year period.

Net Loss

Net loss was $2.2 million for the three months ended March 31, 2024 as compared with $3.6 million for the three months ended March 31, 2023. The decrease in net loss was primarily due to the changes in research and development expenses, as described above.

On May 9, 2024 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company focused on delivering innovative immunology therapeutics, reported the execution of an amended agreement with Sagard Healthcare for additional Anaptys Jemperli (dostarlimab) royalties (Press release, AnaptysBio, MAY 9, 2024, View Source [SID1234643035]). Anaptys intends to utilize the proceeds of the transaction to continue the broad development of its immune cell modulators (ICMs), including its best-in-class checkpoint agonists, in heterogeneous, systemic autoimmune and inflammatory diseases.

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"We believe this amended agreement with Sagard further validates the commercial potential of Anaptys-discovered Jemperli and brings significant non-dilutive funding to Anaptys, further strengthening our balance sheet," said Daniel Faga, president and chief executive officer of Anaptys. "This additional capital will further enable investment across our industry leading ICM portfolio where we expect to end 2024 with four programs in clinical development to potentially bring transformational medicines to patients."

"We are thrilled to amend our partnership with Anaptys on this royalty transaction which reflects our growing confidence in Jemperli plus chemotherapy as a treatment for patients with certain types of endometrial cancer, as well as the potential of Jemperli as a monotherapy and in combination with other therapeutics to treat a broad range of solid tumor cancers. Sagard’s investment is aligned with our goal of accelerating biopharmaceutical innovation by providing our partners with flexible sources of financing," said Ali Alagheband, partner at Sagard.

Upon closing of this transaction, which is anticipated by the end of next week, Sagard will pay Anaptys $50 million upfront in exchange for royalties payable to Anaptys under its GSK collaboration on all annual global net sales of Jemperli. The amended agreement now includes the royalty tiers of 12% to 25% for annual global net sales above $1 billion.

Unchanged from the prior agreement, Sagard receives royalties of 8% for annual global net sales below $1 billion and may also receive up to a total of $105 million in potential cash milestones, of which $15 million are subject to certain future Jemperli regulatory filing and approval milestones and up to $90 million are subject to certain commercial sales milestones due prior to Jemperli achieving the $1 billion in annual global net sales threshold.

A $75 million milestone due upon Jemperli achieving the $1 billion in annual global net sales threshold remains not subject to this agreement. Also, royalties and milestones due to Anaptys upon further development and commercialization of the Anaptys-discovered anti-TIM-3 antagonist (cobolimab, (GSK4069889)) antibody under the GSK collaboration remain not subject to this agreement.

The capped aggregate Jemperli royalties and milestones to be received by Sagard under this amended agreement have been revised. Now, once Sagard receives an aggregate of either $600 million if received by March 31, 2031, or $675 million if received at any time thereafter, the agreement will expire, resulting in Anaptys regaining all subsequent Jemperli royalties and milestones under the GSK collaboration.

Goodwin Procter LLP is acting as counsel to Anaptys, and Foley Hoag LLP is acting as counsel to Sagard, in this monetization transaction.

About Jemperli

Jemperli is a programmed death receptor-1 (PD-1)-blocking antibody that binds to the PD-1 receptor and blocks its interaction with the PD-1 ligands PD-L1 and PD-L2.

In the U.S., Jemperli is indicated in combination with carboplatin and paclitaxel, followed by Jemperli as a single agent for the treatment of adult patients with primary advanced or recurrent endometrial cancer that is dMMR, as determined by a U.S. Food and Drug Administration (FDA)-approved test, or MSI-H, and as a single agent for adult patients with dMMR recurrent or advanced endometrial cancer, as determined by a U.S. FDA-approved test, that has progressed on or following a prior platinum-containing regimen in any setting and are not candidates for curative surgery or radiation. The sBLA supporting this indication in combination with carboplatin and paclitaxel for dMMR/MSI-H primary advanced or recurrent endometrial cancer received Breakthrough Therapy designation and Priority Review from the U.S. FDA. Jemperli is also indicated in the U.S. for patients with dMMR recurrent or advanced solid tumors, as determined by a U.S. FDA-approved test, that have progressed on or following prior treatment and who have no satisfactory alternative treatment options. The latter indication is approved in the U.S. under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication in solid tumors may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Jemperli was discovered by Anaptys and licensed to TESARO, Inc., under a collaboration and exclusive license agreement signed in March 2014. Under this agreement, GSK is responsible for the ongoing research, development, commercialization and manufacturing of Jemperli and cobolimab.