On March 28, 2022 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that it obtained regulatory approval from the Ministry of Health, Labour and Welfare (MHLW) for the anti-HER2 humanized monoclonal antibodies, Perjeta for intravenous infusion 420mg/14 mL [generic name: pertuzumab] (hereafter, Perjeta) and Herceptin for intravenous infusion 60 and 150 [generic name: trastuzumab] (hereafter, Herceptin), for the additional indication of advanced or recurrent HER2-positive colon cancer or rectal cancer not amenable to curative resection that has progressed after cancer chemotherapy (Press release, Chugai, MAR 28, 2022, View Source [SID1234611046]).

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"There are many effective treatments available for colorectal cancer, however, recent genetic studies have revealed that some patients do not respond adequately to standard treatment. We are very pleased that now we have the opportunity to offer the combination therapy of Perjeta and Herceptin to these patients, as a new treatment option based on personalized medicine," said Chugai’s President and CEO, Dr. Osamu Okuda. "HER2-positive colorectal cancer is a rare subtype that accounts for 1~5% of all colorectal cancers. This approval opens the way for personalized medicine for this subtype for the first time in Japan and overseas. I would like to thank everyone who supported us for this approval, especially those who participated in the investigator-initiated study which formed the basis of this trial, including patients, their families and medical professionals. We are committed to providing information on the proper use of this combination therapy so that it may contribute to the treatment of colorectal cancer."

The approval is based on the results of an investigator-initiated phase II clinical trial (TRIUMPH study) conducted in Japan that evaluated the efficacy and safety of the combination of Perjeta and Herceptin in 30 patients with HER2-positive, curatively unresectable advanced or recurrent colorectal cancer who had previously undergone chemotherapy. The primary endpoint was the objective response rate as determined by the investigator’s judgment. Objective response was observed in 29.6% of patients confirmed HER2 expression in tumor tissue and 28.0% of patients diagnosed with HER2-positive and RAS wild-type by liquid biopsy. Major adverse events included infusion-related reactions, diarrhea, decreased appetite, nausea, stomatitis and nasopharyngitis.

HER2 protein overexpression and gene amplification should be determined with the pathological testing kit VENTANA ultraView Pathway HER2 (4B5) provided by Roche Diagnostics K.K. and Pathvision HER-2 DNA Probe Kit provided by Abbott Japan Joint company. Both tests obtained regulatory approval on March 17, 2022 and March 10, 2022, respectively, as companion diagnostics for Perjeta and Herceptin to identify patients with HER2 positive colorectal cancer who may benefit from the combination therapy.

[Approval Information (Perjeta)] * This additional section only

Indications

Advanced or recurrent HER2-positive colon cancer or rectal cancer not amenable to curative resection that has progressed after cancer chemotherapy
Dosage and administration

For cases of advanced or recurrent HER2-positive colon cancer or rectal cancer not amenable to curative resection that has progressed after cancer chemotherapy, the usual adult dosage is a loading dose of 840 mg pertuzumab (genetical recombination) and subsequent doses of 420 mg every 3 weeks, each administered by intravenous infusion over 60 minutes, in combination with trastuzumab (genetical recombination). If the first infusion is well tolerated, subsequent infusions may be administered over a shorter time of at least 30 minutes.
[Approval Information (Herceptin)] * This additional section only

Indications

Advanced or recurrent HER2-positive colon cancer or rectal cancer not amenable to curative resection that has progressed after cancer chemotherapy
Dosage and administration

Use Regimen B for advanced or recurrent HER2-positive colon cancer or rectal cancer not amenable to curative resection that has progressed after cancer chemotherapy, in combination with pertuzumab (genetical recombination).
Regimen B: The usual adult dosage is a loading dose of 8 mg/kg (body weight) trastuzumab (genetical recombination) and subsequent doses of 6 mg/kg every 3 weeks, each administered by intravenous infusion over at least 90 minutes.

If the first infusion is well tolerated, subsequent infusions may be administered over a shorter time of at least 30 minutes.

About Perjeta

Perjeta is a humanized monoclonal antibody that targets human epidermal growth factor receptor type 2 (HER2), which is involved in the growth of tumor cells. Perjeta in combination with Herceptin blocks the HER-signaling system more extensively. The drug was launched in 2013 for "inoperable or recurrent HER2-positive breast cancer." The indication was amended as "HER-2 positive breast cancer," after obtaining regulatory approval for the additional indication of "neoadjuvant and adjuvant therapy in HER2-positive breast cancer" in 2018.

About Herceptin

Herceptin, like Perjeta, is a humanized monoclonal antibody that targets human epidermal growth factor receptor type 2 (HER2), which is involved in the growth of tumor cells. Herceptin was launched in 2001 for "metastatic breast cancer overexpressing HER2." In 2011, it was approved for the treatment of patients with "advanced or recurrent gastric cancer overexpressing HER2 not amenable to curative resection," and in 2021 for "advanced or recurrent HER2-positive salivary gland cancer not amenable to curative resection."

The product names listed above are protected by law.

On March 28, 2022 Immunome, Inc. (Nasdaq: IMNM), a biopharmaceutical company that utilizes its human memory B cell platform to discover and develop first-in-class antibody therapeutics, reported financial results for the fourth quarter ended December 31, 2021 and provided a corporate update (Press release, Immunome, MAR 28, 2022, View Source [SID1234611062]).

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"There remains a clear need for safe and efficacious antibody treatments against COVID-19, especially those less susceptible to mutational drift. We believe our three-antibody cocktail, IMM-BCP-01, has strong potential to address that unmet need. We are pleased the FDA has given us a safe-to-proceed notification and lifted the clinical hold for our Investigational New Drug (IND) application," stated Purnanand Sarma, Ph.D., President and CEO of Immunome.

"Additionally, we continue to progress IMM-ONC-01, our novel, IL-38-targeting innate immune checkpoint inhibitor, towards an IND submission in the second half of 2022," Sarma continued. "Our preclinical work is ongoing, and we have analyzed IL-38 expression across nearly 60 tumor subtypes and we have confirmed a high frequency of expression in difficult to treat cancers, such as head and neck squamous cell carcinoma, gastroesophageal squamous carcinoma, and squamous lung carcinoma."

Fourth Quarter and Subsequent Highlights

·Demonstrated In Vitro Efficacy of IMM-BCP-01 Against SARS-CoV-2 Omicron Variant in Live Virus Testing. In February 2022, Immunome announced that IMM-BCP-01 demonstrated effective neutralization of the Omicron variant of COVID-19 in in vitro testing. The combination of two antibodies in Immunome’s antibody cocktail, IMM20253/IMM20184, demonstrated neutralization of the Omicron variant within 3.5-fold potency compared to a preclinical version of sotrovimab in a head-to-head test using live virus samples. Additionally, IMM20253 exhibited a novel mechanism of action not reported in any other EUA antibodies by promoting a proteolytic cleavage of the portion of the spike protein needed for ACE2 binding.

·IMM-BCP-01 IND Application for the Treatment of COVID-19. In March 2022, the FDA communicated that the clinical study can be initiated for our antibody cocktail for the treatment of SARS-CoV-2 following a brief clinical hold. Immunome is continuing its ongoing clinical preparations ahead of the Phase 1b trial.

This investigational work was funded by the U.S. Department of Defense’s (DOD) Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense (JPEO-CBRND) in collaboration with the Defense Health Agency (DHA) (Contract number: W911QY-20-9-0019).

Financial Highlights

•Cash and cash equivalents: As of December 31, 2021, cash and cash equivalents totaled $49.2M.
•Research and development (R&D) expenses: R&D expenses for the three months ended December 31, 2021 were $4.4M. R&D expenses for the year ended December 31, 2021 were $14.1M.
•General and administrative (G&A) expenses: G&A expenses for the three months ended December 31, 2021 were $3.5M. G&A expenses for the year ended December 31, 2021 were $10.6M.
•Net loss: Net loss attributable to common stockholders was $7.9M, or $.65 per share, for the three months ended December 31, 2021. Net loss attributable to common stockholders was $24.7M, or $2.14 per share, for the year ended December 31, 2021.
•As of December 31, 2021, Immunome has 12,110,373 shares of common stock outstanding.

On March 28, 2022 Molecular Templates, Inc. (Nasdaq: MTEM, "Molecular Templates," or "MTEM"), a clinical-stage biopharmaceutical company focused on the discovery and development of proprietary targeted biologic therapeutics, engineered toxin bodies (ETBs), reported financial results for the fourth quarter of 2021 (Press release, Molecular Templates, MAR 28, 2022, View Source [SID1234611108]).

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"2022 is off to a very promising start, following a number of important developments across our pipeline of ETBs in 2021," said Eric Poma, Ph.D., Chief Executive and Chief Scientific Officer of Molecular Templates. "We continue to see differentiated pharmacodynamic effects and evidence of antigen seeding with MT-6402 with additional data expected throughout 2022. We continue dose finding for the MT-5111 and MT-0169 programs with clinical data expected this year. We plan to file an IND in 2H22 for our CTLA-4 program and are moving forward with our earlier stage pipeline of ETBs in preclinical development targeting TIGIT, TROP-2, and BCMA."

Company Highlights and Upcoming Milestones

Corporate

MTEM expects to provide periodic updates on MT-6402, MT-5111, and MT-0169 throughout 2022.
MTEM has had six abstracts accepted for presentation at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Meeting 2022, taking place from April 8-13, 2022.
Gabriela Gruia, M.D. appointed to the Board of Directors.
Megan Filoon promoted to General Counsel.
MT-6402 (PD-L1 ETB with Antigen Seeding Technology)

MTEM continues to enroll patients in the Phase 1 study of MT-6402 which began in July 2021. MT-6402 is the first of MTEM’s 3rd generation ETBs to enter the clinic. It was designed to induce potent anti-tumor effects via PD-L1 targeting through multiple mechanisms that may overcome the limitations of approved checkpoint inhibitors.
The Phase 1 study is a multi-center, open-label, dose escalation and dose expansion trial in the United States. Patients with confirmed PD-L1 expressing tumors or confirmed PD-L1 expression in the tumor microenvironment are eligible for enrollment.
The 16 mcg/kg cohort (cohort 1) was completed with no DLTs observed in the six patients treated. One patient in cohort 1 with non-small cell lung cancer (NSCLC) had evaluable-only multiple sites of bone disease that appeared to have resolved on bone scan with only one remaining site which showed decreased uptake. This patient remained on MT-6402 up to cycle 8 when increased uptake was noted on bone scan and treatment was discontinued.
Six patients have been treated in the 24 mcg/kg cohort (cohort 2). One DLT, a grade 3 dermatitis of two days duration, occurred six days after the first dose in cohort 2 in one patient. The patient was treated with systemic steroids and treatment with MT-6402 was held until cycle 2, dose 1, at which time the patient was re-challenged at the same dose without development of recurrent dermatitis.
Following a review of the safety data from cohort 2 (24 mcg/kg), patient enrollment in cohort 3 initiated at a dose of 32 mcg/kg.
MTEM continues to observe pharmacodynamic (PD) effects including monocyte depletion and T cell activation in the 24 mcg/kg cohort. The extent and timing of these PD effects appear dose-related with patients in the 24 mcg/kg generally showing a more rapid and profound PD effect.
Six patients remain on study with five patients awaiting their first efficacy assessment. Dose escalation continues as planned and following determination of the maximum tolerated dose (MTD), expansion cohorts are planned to evaluate MT-6402 as a monotherapy in tumor-specific and PD-L1 positive basket tumor cohorts.
In 2021, MT-6402 was granted Fast Track Designation by the U.S. Food and Drug Administration for the treatment of patients with advanced NSCLC expressing PD-L1.
MT-5111 (HER2 ETB)

The Phase 1 study of MT-5111 in HER2-positive cancers is ongoing with multiple sites open for enrollment.
The HER2-positive breast cancer expansion cohort initiated in November 2021 at a dose of 10 mcg/kg.
As of January 2022, 30 patients had been treated with MT-5111 across eight dose escalation cohorts ranging from 0.5 mcg/kg to 13 mcg/kg without any DLTs.
There have been no signs of capillary leak syndrome (CLS) or significant cardiotoxicity observed to date with MT-5111.
Enrollment has initiated for the next cohort at 17 mcg/kg. Dose escalation will continue to determine the MTD, while the breast cancer expansion cohort collects efficacy and safety data.
MT-0169 (CD38 ETB)

In August 2021, MTEM assumed full rights to MT-0169 from its former co-development partner, Takeda, including full control of MT-0169 clinical development, per the terms of the terminated collaboration agreement with Takeda. Upon approval of a revised protocol, MTEM will continue to conduct the ongoing Phase 1 study for MT-0169 in relapsed/refractory multiple myeloma and non-Hodgkin’s lymphoma and plans to open new sites for the Phase I study.
A more rapid and complete elimination of CD38+ NK cells (a known PD marker for CD38-targeting therapeutics) was observed in the first five patients than had been predicted from in vitro and in vivo models, suggesting that the starting dose of 50 mcg/kg is higher than required.
A revised protocol was submitted to explore a lower dose of MT-0169 to reduce the risk of adverse events observed at the initial dose and to enable patients to continue MT-0169 therapy for a longer duration that may drive tumor benefit. Importantly, the robust and rapid NK cell depletion that was observed at the starting dose is expected to be observed at lower doses.
Research

MTEM continues to advance its pipeline of next-generation ETBs targeting CTLA-4, TIGIT, TROP2, BCMA, SLAMF-7, and CD45.
IND filing of an ETB in the CTLA-4 program is expected in 2H22.
Lead selection for TIGIT, TROP-2, and BCMA is ongoing.
MTEM plans to present preclinical data on its ETB candidates at AACR (Free AACR Whitepaper) in April 2022 and expects to present further preclinical data throughout the year at medical and scientific conferences.
Financial Results for the Fourth Quarter of 2021

The net loss attributable to common shareholders for the fourth quarter of 2021 was $10.2 million, or $0.18 per basic and diluted share. This compares with a net loss attributable to common shareholders of $28.4 million, or $0.57 per basic and diluted share, for the same period in 2020.

Revenues for the fourth quarter of 2021 were $18.0 million, compared to $3.5 million for the same period in 2020. Revenues for the fourth quarter of 2021 were comprised of revenues from collaborative research and development agreements with Vertex and Bristol Myers Squibb. Total research and development expenses for the fourth quarter of 2021 were $19.3 million, compared with $22.3 million for the same period in 2020. Total general and administrative expenses for the fourth quarter of 2021 were $7.9 million, compared with $7.1 million for the same period in 2020.

As of December 31, 2021, MTEM’s cash and investments totaled $152.0 million. MTEM’s current cash and investments are expected to fund operations into the fourth quarter of 2023.

On March 28, 2022 Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported financial results for the full-year ended December 31, 2021 and recent corporate highlights (Press release, Checkpoint Therapeutics, MAR 28, 2022, View Source [SID1234611047]).

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James F. Oliviero, President and Chief Executive Officer of Checkpoint, said, "The past year represented a truly transformational period for Checkpoint Therapeutics, with the foundation laid for multiple significant potentially value enhancing catalysts in 2022. Following the positive topline results from our ongoing registrational trial of cosibelimab in metastatic cutaneous squamous cell carcinoma announced earlier this year, we look forward to a planned Biologics License Application submission for cosibelimab later in 2022." Mr. Oliviero continued, "We remain focused on expeditiously advancing our pipeline of product candidates with the goal of expanding patient access globally to potentially life-saving novel oncology therapies through a disruptive pricing strategy."

2021 and Recent Corporate Highlights:

In January 2022, Checkpoint announced positive topline results from the ongoing registration-enabling clinical trial evaluating the safety and efficacy of its anti-PD-L1 antibody, cosibelimab, administered as a fixed dose of 800 mg every two weeks in patients with metastatic cutaneous squamous cell carcinoma ("cSCC"). The study met its primary endpoint, with cosibelimab demonstrating a confirmed objective response rate of 47.4% (95% CI: 36.0, 59.1) based on independent central review of 78 patients enrolled in the metastatic cSCC cohort using Response Evaluation Criteria in Solid Tumors version 1.1 criteria. Checkpoint intends to submit a Biologics License Application for cosibelimab in late 2022, followed by a Marketing Authorization Application submission in Europe and other territories worldwide. With a potentially favorable safety profile versus anti-PD-1 therapy and a plan to commercialize at a substantially lower price, Checkpoint believes cosibelimab has the potential to be a market disruptive product in the $30 billion and growing PD-(L)1 class.
In December 2021, Checkpoint announced the initiation of the CONTERNO study, a global, randomized Phase 3 trial of cosibelimab in combination with pemetrexed and platinum chemotherapy for the first-line treatment of patients with non-squamous non-small cell lung cancer.
During the second quarter of 2021, Checkpoint had productive interactions with the FDA regarding its ongoing development program for olafertinib (formerly CK-101), a third-generation epidermal growth factor receptor inhibitor being evaluated by its partner in an ongoing double-blind, randomized Phase 3 study in China.
In March 2021, Checkpoint announced the formation of a Scientific Advisory Board comprised of clinical and scientific thought leaders in oncology. Members include Wayne A. Marasco, M.D., Ph.D., F. Stephen Hodi, Jr., M.D., Bruce E. Johnson, M.D., Roy S. Herbst, M.D., Ph.D., David Miller, M.D., Ph.D., and Emily Ruiz, M.D., M.P.H.
Financial Results:

Cash Position: As of December 31, 2021, Checkpoint’s cash and cash equivalents totaled $54.7 million, compared to $40.8 million at December 31, 2020, an increase of $13.9 million.
R&D Expenses: Research and development expenses for the year ended December 31, 2021, were $48.5 million, compared to $16.4 million for the year ended December 31, 2020, an increase of $32.1 million. Research and development expenses for the year ended December 31, 2021 included $7.3 million of non-cash stock expenses, compared to $5.2 million in non-cash stock expenses for the year ended December 31, 2020.
G&A Expenses: General and administrative expenses for the year ended December 31, 2021 were $8.5 million, compared to $7.9 million for the year ended December 31, 2020, an increase of $0.6 million. General and administrative expenses for the year ended December 31, 2021 included $3.5 million of non-cash stock expenses, compared to $3.1 million in non-cash stock expenses for the year ended December 31, 2020.
Net Loss: Net loss attributable to common stockholders for the year ended December 31, 2021 was $56.7 million, or $0.75 per share, compared to a net loss of $23.1 million, or $0.41 per share, for the year ended December 31, 2020.

On March 28, 2022 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, reported financial results for the three months and full year ended December 31, 2021, and highlighted recent progress (Press release, Verastem, MAR 28, 2022, View Source [SID1234611063]).

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"We anticipate a catalyst-driven year and are well-positioned to maximize the potential of VS-6766 as a backbone therapy across RAS pathway-driven solid tumors in order to bring new solutions to patients in areas of high unmet need. Our recent debt facility adds flexibility to our financial strength and is expected to support the continued development and potential commercial launches of VS-6766 and defactinib, including building on our breakthrough therapy designation in low-grade serous ovarian cancer," said Brian Stuglik, Chief Executive Officer of Verastem Oncology. "We plan to report results from the selection phase of RAMP 201, our lead program in patients with low-grade serous ovarian cancer, regardless of KRAS status, during the second quarter. And, as part of our non-small cell lung cancer program, we are targeting KRAS mutant patient subpopulations across four clinical trials and we are anticipating initial readouts from three NSCLC studies, including RAMP 202 with defactinib, the investigator-sponsored study with everolimus, and RAMP 203 with LUMAKRASTM (sotorasib) this year."

Fourth Quarter 2021 and Recent Highlights

Verastem’s lead drug candidate VS-6766 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 pathway inhibition.

Low Grade Serous Ovarian Cancer (LGSOC)

Planned enrollment is complete in the selection phase (Part A; n=64) of the registration-directed Phase 2 RAMP 201 study investigating VS-6766 alone or in combination with defactinib for the treatment of recurrent LGSOC. Verastem remains on track to report topline results from Part A during the second quarter of 2022, following discussions with regulatory authorities.
Enrollment has commenced in the expansion phase (Part B) of RAMP 201, with both treatment arms (VS-6766 alone and in combination with defactinib) currently advancing in all patients. Verastem expects to complete enrollment in Part B during the second half of 2022.
KRAS Mutant Non-Small Cell Lung Cancer (NSCLC)

Planned enrollment is now complete in the selection phase (Part A; n=32) of the registration-directed RAMP 202 study investigating VS-6766 alone and in combination with defactinib in patients with KRAS G12V mutant NSCLC. The Company expects to report topline results from Part A and initiate Part B during the second half of 2022, following discussions with regulatory authorities.
Based on preclinical rationale, Verastem has added BRAF mutant cohorts to the RAMP 202 study in order to efficiently evaluate VS-6766 with defactinib in BRAF-mutant NSCLC. In Part A of the study, the Company expects to enroll two cohorts comprised of 15 patients each to evaluate the combination in patients with V600E or non-V600E BRAF mutations, respectively. These cohorts are open and enrolling.
The Company entered into two clinical agreements to study VS-6766 in combination with KRAS G12C inhibitors in patients with KRAS G12C-mutant NSCLC, including patients who have progressed on a KRAS G12C inhibitor. Initial results from the ongoing Phase 1/2 RAMP 203 study evaluating VS-6766 in combination with Amgen’s LUMAKRASTM (sotorasib) are expected to be reported during the second half of 2022. Initiation of the Phase 1/2 RAMP 204 study evaluating VS-6766 in combination with Mirati’s adagrasib is expected during the second quarter of 2022.
Corporate Updates

Secured debt facility with Oxford Finance LLC for up to $150 Million. Under the terms of the credit facility with Oxford Finance LLC, Verastem drew an initial $25 million term loan at closing. The Company has the ability to access up to an additional $125 million in a series of tranches, $75 million of which are based on certain pre-determined milestones and $50 million at the lender’s discretion.
With the credit facility and expected milestones related to the sale of COPIKTRA (duvelisb) to Secura Bio Inc. (Secura) in 2020, the Company expects to have a cash runway through 2025 to support the continued development and potential commercial launches of VS-6766 and defactinib.
In the first quarter of 2022, Secura Bio Inc.’s (Secura) sublicensee, CSPC Pharmaceutical Group Limited, obtained drug registration approval for duvelisib granted by the National Medical Products Administration of the People’s Republic of China for the treatment of adult patients with relapsed or refractory follicular lymphoma after at least two prior systematic therapies. This entitles Verastem to a $2.5 million milestone payment from Secura, pursuant to the sale of COPIKTRA (duvelisb) to Secura in 2020.
Appointed Michelle Robertson to join the Verastem Board of Directors. Ms. Robertson is the Chief Financial Officer at Editas Medicine and brings more than 25 years of finance and commercial operations leadership to the Board. Timothy Barberich will be retiring from the Board as of March 31, 2022. Verastem appreciates his significant contributions since his appointment in March, 2014.
Appointed Channing Der, PhD, Sarah Graham Kenan Distinguished Professor at the University of North Carolina at Chapel Hill to the Company’s Scientific Advisory Board.
Fourth Quarter 2021 Financial Results

Total revenue for the three months ending December 31, 2021 (2021 Quarter) was $0.5 million, compared to $0.5 million for the three months ended December 31, 2020 (2020 Quarter).

Total research and development (R&D) and selling, general and administrative (SG&A) expenses for the 2021 Quarter were $17.1 million, compared to $17.3 million for the 2020 Quarter.

R&D expenses for the 2021 Quarter were $11.4 million, compared to $10.2 million for the 2020 Quarter. The increase of $1.2 million, or 11.8%, primarily resulted from increase in contract research organization costs and investigator fees.

SG&A expenses for the 2021 Quarter were $5.7 million, compared to $7.1 million for the 2020 Quarter. The decrease of $1.4 million, or 19.7%, primarily resulted from lower employee related expenses and consulting and professional fees.

Net loss for the 2021 Quarter was $16.5 million, or $0.09 per share (basic and diluted), compared to net loss of $19.9 million, or $0.12 per share (basic and diluted), for the 2020 Quarter.

For the 2021 Quarter, non-GAAP adjusted net loss was $14.9 million, or $0.08 per share (diluted), compared to non-GAAP adjusted net loss of $14.8 million, or $0.09 per share (diluted), for the 2020 Quarter. Please refer to the GAAP to Non-GAAP Reconciliation attached to this press release.

Full-Year 2021 Financial Results

Verastem Oncology ended the fourth quarter of 2021 with cash, cash equivalents and investments of $100.3 million. On a pro forma basis, inclusive of the funding received from the $25 million drawdown of new debt facility, cash, cash equivalents and investments were $125.3 million as of December 31, 2021.

Total revenue for the year ended December 31, 2021 (2021 Period) was $2.1 million, compared to $88.5 million for the year ended December 31, 2020 (2020 Period). Revenue for the 2021 Period was comprised of (i) $1.4 million of revenue recognized for milestones and royalties as part of the sale of COPIKTRA to Secura Bio, Inc. (Secura), and (ii) $0.6 million of transition services revenue for certain support functions provided to Secura pursuant to the Secura transition services agreement which was entered into in connection with the sale of COPIKTRA to Secura. Revenue for the 2020 Period revenue was comprised of (i) $70.0 million recognized for the upfront payment made as part of the sale of COPIKTRA to Secura, (ii) $15.2 million of net product revenue, (iii) $2.9 million of license and collaboration revenue, and (iv) $0.4 million of transition services revenue for certain support functions provided to Secura.

Total R&D and SG&A expenses for the 2021 Period were $63.5 million, compared to $104.1 million for the 2020 Period.

R&D expense for the 2021 Period was $39.3 million, compared to $41.4 million for the 2020 Period. The decrease of $2.1 million, or 5.1%, was primarily related to the upfront non-refundable payment of $3.0 million to Chugai Pharmaceutical Co., Ltd for the VS-6766 license in the 2020 Period, and a decrease in other operating costs. This decrease was partially offset by an increase in investigator fees and an increase in personnel related costs.

SG&A expense for the 2021 Period was $24.1 million, compared to $62.8 million for the 2020 Period. The decrease of $38.7 million, or 61.6%, was primarily resulted from the Company’s shift in strategic direction and the sale of COPIKTRA to Secura, which led to lower employee-related expenses and consulting and professional fees.

Net loss for the 2021 Period was $71.2 million, or $0.41 per share (basic and diluted), compared to $67.7 million, or $0.44 per share (basic and diluted), for the 2020 Period.

For the 2021 Period, non-GAAP adjusted net loss was $54.1 million, or $0.31 per share (diluted), compared to non-GAAP adjusted net loss of $37.8 million, or $0.25 per share (diluted), for the 2020 Period. 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) income and non-GAAP net (loss) income 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 and year ended December 31, 2021 and 2020 are included in the tables accompanying this press release after the unaudited condensed consolidated financial statements.

About VS-6766

VS-6766 (formerly known as CH5126766 and RO5126766) 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 pathway inhibition. VS-6766 is currently in late-stage development.

In contrast to other MEK inhibitors, VS-6766 blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows VS-6766 to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of other inhibitors. The U.S. Food and Drug Administration granted Breakthrough Therapy designation for the combination of Verastem Oncology’s investigational RAF/MEK inhibitor VS-6766, with defactinib, its 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.1

Verastem Oncology is conducting Phase 2 registration-directed trials of VS-6766 alone and with defactinib in patients with recurrent LGSOC and in patients with recurrent KRAS G12V-mutant NSCLC as part of its RAMP (Raf And Mek Program) clinical trials, RAMP 201 and RAMP 202, respectively. Verastem Oncology has also established clinical collaborations with Amgen and Mirati to evaluate LUMAKRAS (sotorasib) and adagrasib in combination with VS-6766 in KRAS G12C-mutant NSCLC as part of the RAMP 203 and RAMP 204 trials, respectively.