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Verastem Oncology Reports Second Quarter 2018 Financial Results
“During the second quarter of 2018, we’ve been actively preparing for
the commercialization of duvelisib, our first-in-class, oral dual
inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma for
the treatment of patients with relapsed or refractory chronic
lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) or follicular
lymphoma (FL),” said
Second Quarter 2018 and Recent Highlights:
Corporate and Financial
-
Duvelisib NDA accepted by
FDA with priority review – InApril 2018 , Verastem Oncology announced that the U.S. Food and Drug Administration (FDA ) accepted the duvelisib New Drug Application (NDA) for filing with Priority Review, with a target action date ofOctober 5, 2018 . In the accepted NDA, the Company is seeking full approval for duvelisib, its first-in-class investigational oral dual inhibitor of PI3K-delta and PI3K-gamma, for the treatment of relapsed or refractory CLL/SLL and accelerated approval for the treatment of relapsed or refractory FL. The duvelisib NDA is supported by clinical data from the randomized Phase 3 DUO™ study evaluating duvelisib as a monotherapy in patients with relapsed or refractory CLL/SLL, as well as the Phase 2 DYNAMO™ study evaluating patients with iNHL that are double-refractory to both rituximab and chemotherapy or radioimmunotherapy. Both DUO and DYNAMO achieved their primary endpoints. -
Hosted Analyst and Investor Day highlighting commercial
potential of duvelisib – In early
May 2018 ,Verastem Oncology hosted an Analyst and Investor Day inNew York City titled, “Duvelisib: Harnessing the Power of Dual PI3K Inhibition.” Key opinion leaders in the hematologic oncology field including Dr.Lori Kunkel , Former Chief Medical Officer, Pharmacyclics, Dr.Jennifer Brown ,Dana-Farber Cancer Institute , Dr.Ian Flinn ,Sarah Cannon Research Institute , Dr.Steven Horwitz ,Memorial Sloan Kettering Cancer Center as well as Dr.Brian Koffman , Founder & Medical Director of theChronic Lymphocytic Leukemia (CLL) Society , and a CLL patient, joined the Verastem Oncology executive leadership team for an in-depth discussion regarding the unmet need among CLL/SLL and FL patients, where PI3K-delta and PI3K-gamma inhibitors fit into the treatment paradigm, and the growing opportunity for duvelisib in CLL/SLL and FL, and beyond. The Company also provided an overview of its duvelisib commercial strategy and initiatives. The webcast is available within the “Media” section of the Company’s website at www.verastem.com. -
Signed exclusive license agreement with
Yakult Honsha Co., Ltd. (Yakult) to develop and commercialize duvelisib inJapan – InJune 2018 , Verastem Oncology announced its entry into an exclusive license and collaboration agreement with Yakult to develop and commercialize duvelisib for the treatment, prevention or diagnosis of all oncology indications inJapan . The transaction, which carries a total deal value of up to$100.0 million , includes a one-time upfront payment of$10.0 million and up to an additional$90.0 million if certain future pre-specified development, regulatory and commercial milestones are successfully achieved by Yakult. In addition,Verastem Oncology is also eligible to receive double-digit royalties based on future net sales of duvelisib inJapan . Pursuant to the agreement, Yakult has the right to develop and commercialize duvelisib inJapan at its own cost and expense. In addition, Yakult may fund certain global development costs on a pro-rata basis. Verastem Oncology retains all rights to duvelisib outside ofJapan . -
Strengthened the balance sheet through the sale of equity for
net proceeds of approximately
$105 Million – InMay 2018 , Verastem Oncology completed an underwritten registered offering of 8,944,444 shares of its common stock at a price to the public of$4.50 per share. The net proceeds toVerastem from the offering were approximately$38.3 million . InJune 2018 , Verastem Oncology completed a registered offering of 7,166,666 shares of its common stock at a price of$6.00 per share to funds managed byConsonance Capital . The net proceeds to Verastem Oncology from this offering were approximately$42.8 million . The Company also sold 6,314,410 shares of common stock under its at-the-market equity offering program for net proceeds of approximately$23.7 million . -
Joined the Russell 3000® Index – In
June 2018 , the Company joined the broad-market Russell 3000® Index as part of the Russell US Indexes annual reconstitution.
Scientific Presentations at Major Medical Meetings
Duvelisib
-
The efficacy of duvelisib monotherapy following disease
progression on ofatumumab monotherapy in patients with
relapsed/refractory CLL or SLL in the DUO™ crossover extension study
– In
June 2018 , at both theAmerican Society of Clinical Oncology 2018 Annual Meeting (ASCO 2018) and theEuropean Hematology Association 2018 Annual Meeting (EHA 2018), Dr.Byrone Kuss ,Flinders Medical Centre , and Dr.Peter Hillman ,St. James University Hospital , respectively, presented additional data from the open-label, DUO crossover extension study where patients with radiologically confirmed progressive disease (PD) following treatment with ofatumumab in DUO were given the option to receive treatment with duvelisib. Among the 89 evaluable patients (median 3 prior therapies; range 2-8), duvelisib as a monotherapy achieved a 73% overall response rate (ORR) per investigators assessment in the extension study (95% confidence interval CI: 64,82); 5% complete response with incomplete marrow recovery (CRi), and 68% partial response (PR). The median progression-free survival (mPFS) for duvelisib in the DUO crossover extension study was 15 months (95% CI: 10,17). Notably, 83% of patients in the duvelisib arm post-crossover had >50% reductions in the size of their target nodal lesions. The safety profile of duvelisib as a monotherapy was manageable and consistent with what was observed in the Phase 3 DUO™ study. These data build upon the previously reported positive DUO results and further support duvelisib as an effective oral monotherapy treatment option for patients with relapsed or refractory CLL/SLL. -
A Phase IB/II study of duvelisib in combination with Fludarabine
(F), Cyclophosphamide (C), and Rituximab (R) (dFCR) for frontline
therapy of younger CLL patients – At EHA 2018,
Dr.
Matthew Davids ,Dana-Farber Cancer Institute , presented data on the 31 patients evaluable for post-dFCR response. The ORR was 94%, with 26% (n=8) of patients achieving a complete response (CR) or CRi, and 68% achieving a PR. The best rate of minimum residual disease (MRD) negativity in the bone marrow (BM) in patients with at least one evaluation was 76% (22 of 29 patients). All patients who achieved CR/CRi at the primary endpoint also had BM-MRD negativity (26%). Among survivors, the median follow-up is 24.5 months (range 6.9-46 months). The two-year progression-free survival and overall survival rates for patients in the study were both 97%. Eight patients have now completed two years of duvelisib maintenance therapy. Based on these results the recommended Phase 2 dose of duvelisib in combination with FCR was 25mg twice daily. The most common all grade non-hematologic adverse events (AEs) were nausea (72%, all Grade 1/2), fatigue (69%, 3% Grade 3), fever (53%, all Grade 1/2), diarrhea (47%, 3% Grade 3), transaminitis (34%, 28% Grade 3/4), anorexia (34%, all Grade 1/2), vomiting (28%, all Grade 1/2), pruritus (16%, 3% Grade 3), arthritis (9%, all Grade 2) and Cytomegalovirus (CMV) reactivation (6%, both Grade 2). The most common all grade hematologic adverse events were thrombocytopenia (65%; 34% Grade 3-4), neutropenia (59%; 50% Grade 3-4), and anemia (38%, 16% Grade 3). Serious AEs included transaminitis (Grade ≥3), febrile neutropenia (n=6, all Grade 3), pneumonia (n=6, including 3 cases of PJP despite planned prophylaxis), and colitis (n=1 Grade 2, n=1 Grade 3). These results suggest that duvelisib in combination with FCR is an effective regimen for the initial therapy of younger, fit CLL patients and results in a high rate of BM-MRD negativity (76%), significantly higher than historical data with FCR. -
The effect of duvelisib, a dual inhibitor of PI3K-δ,γ, on
components of the tumor microenvironment in previously untreated
follicular lymphoma patients – At both
ASCO 2018 and EHA 2018, Dr.Carla Casulo ,University of Rochester ,Wilmot Cancer Center , presented data from blood samples from healthy volunteers and FL patients treated in the CONTEMPO study. Samples collected both pre- and post-duvelisib treatment were analyzed. Ex vivo and in vitro PI3K-delta assays and PI3K-gamma assays, with PI3K-gamma-selective (idelalisib, TGR-1202, IPI-3063) and PI3K-delta-selective (IPI-549) inhibitors were compared. Collectively, the results of this analysis support the thesis that duvelisib disrupts PI3K-delta and PI3K-gamma function in FL patients, inhibiting the tumor microenvironment (TME) cancer-supportive macrophages and T-cells. -
Duvelisib inhibition of chemokines in patients with CLL (DUO™)
and iNHL (DYNAMO™) – At both
ASCO 2018 and EHA 2018, Dr.David Weaver , Verastem Oncology’s Vice President, Translational Medicine, presented data showing that PI3K-delta inhibition directly targets proliferation and survival of malignant leukemia and lymphoma cells, while PI3K-gamma inhibition modulates the TME through key support cells, including tumor-associated macrophages, nurse-like stroma and T-cells, and via soluble factors stimulating tumor growth, survival and migration. Serum samples from patients in the Phase 3 DUO study in relapsed/refractory CLL/SLL and the Phase 2 DYNAMO study in relapsed/refractory indolent non-Hodgkin lymphoma (iNHL) were collected at baseline and at infusion cycle date C2D1 and used for correlative studies of 24 chemokines, cytokines and serum factors. These data support the hypothesis that treatment with duvelisib results in significant reduction of chemokines potentially derived from the tumor cells and TME and that further investigation of the effects of duvelisib on TME pharmacodynamic markers is warranted. -
Presented scientific data supporting immuno-oncology
applications of duvelisib at the 3rd annual Advances in
Immuno-Oncology Congress – InMay 2018 ,Jonathan Pachter , Ph.D., Verastem Oncology’s Chief Scientific Officer, gave an oral presentation highlighting the unique potential of duvelisib, as a dual inhibitor of PI3K-delta and PI3K-gamma, to enhance the efficacy of immune checkpoint and co-stimulatory antibodies in preclinical models of both hematological malignancies and solid tumors. Dr. Pachter also moderated a round table discussion regarding novel checkpoint pathways and emerging strategies for combined modality treatment.
Defactinib
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Presented preliminary Phase 1 results from combination trial
with defactinib, pembrolizumab and gemcitabine in advanced cancer –
At
ASCO 2018, Dr.Andrea Wang-Gillam presented a poster describing results from the ongoing Phase 1 study evaluating defactinib in combination with pembrolizumab and gemcitabine in patients with advanced cancer, including pancreatic cancer. The combination treatment appears to be well tolerated, the recommended Phase 2 dose was established, and the expansion phase of the study is now ongoing. Encouraging signs of clinical activity were observed in three pancreatic ductal adenocarcinoma (PDAC) patients treated beyond 250 days, including one patient with confirmed PR and two patients with stable disease. Meaningful reductions (57-96%) in the pancreatic cancer marker CA19-9 were also observed in all three patients. In addition, analysis of paired biopsies showed that the combination treatment induced desirable biomarker changes including increased proliferating CD8+ T-cells and reduced immunosuppressive Tregs and macrophages. -
Presented scientific data supporting immuno-oncology
applications of defactinib at the 3rd Annual Advances in
Immuno-Oncology Congress – During Dr. Pachter’s oral presentation, he also provided an update on the scientific rationale and clinical progress of Verastem Oncology’s lead focal adhesion kinase (FAK) inhibitor, defactinib, in combination with PD-1 and PD-L1 inhibitors in solid tumors.
All posters and presentations are available within the “Media” section of the Company’s website at www.verastem.com.
Second Quarter 2018 Financial Results
Net loss for the three months ended
Research and development expense for the 2018 Quarter was
General and administrative expense for the 2018 Quarter was
As of
The number of outstanding common shares as of
Financial Guidance
Based on the Company’s current operating plans, assuming a favorable regulatory decision and estimated revenue, it expects to have sufficient cash and cash equivalents to fund operations into 2020.
About Duvelisib
Duvelisib is a first-in-class investigational oral, dual inhibitor of
phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, two enzymes known
to help support the growth and survival of malignant B-cells and
T-cells. PI3K signaling may lead to the proliferation of malignant B-
and T-cells and is thought to play a role in the formation and
maintenance of the supportive tumor microenvironment.1,2,3 Duvelisib
was evaluated in late- and mid-stage extension trials, including DUO™, a
randomized, Phase 3 monotherapy study in patients with relapsed or
refractory chronic lymphocytic leukemia/small lymphocytic lymphoma
(CLL/SLL),4 and DYNAMO™, a single-arm, Phase 2 monotherapy
study in patients with refractory indolent non-Hodgkin lymphoma (iNHL).5 Both
DUO and DYNAMO achieved their primary endpoints. Verastem Oncology’s New
Drug Application (NDA) requesting the full approval of duvelisib for the
treatment of patients with relapsed or refractory CLL/SLL, and
accelerated approval for the treatment of patients with relapsed or
refractory follicular lymphoma (FL) was accepted for filing by the U.S.
Food and Drug Administration (
About Defactinib
Defactinib is an investigational inhibitor of focal adhesion kinase
(FAK), a non-receptor tyrosine kinase that mediates oncogenic signaling
in response to cellular adhesion and growth factors.7 Based
on the multi-faceted roles of FAK, defactinib is used to treat cancer
through modulation of the tumor microenvironment and enhancement of
anti-tumor immunity.8,9 Defactinib is currently being
evaluated in three separate clinical collaborations in combination with
immunotherapeutic agents for the treatment of several different cancer
types including pancreatic cancer, ovarian cancer, non-small cell lung
cancer (NSCLC), and mesothelioma. These studies are combination clinical
trials with pembrolizumab and avelumab from Merck & Co. and Pfizer/
About Verastem Oncology
Forward-looking statements notice:
This press release includes forward-looking statements about
References |
1 Winkler D.G., Faia K.L., DiNitto J.P. et al. PI3K-delta and PI3K-gamma inhibition by IPI-145 abrogates immune responses and suppresses activity in autoimmune and inflammatory disease models. Chem Biol 2013; 20:1-11. |
2 Reif K et al. Cutting Edge: Differential Roles for Phosphoinositide 3 kinases, p110-gamma and p110-delta, in lymphocyte chemotaxis and homing. J Immunol 2004:173:2236-2240. |
3 Schmid M et al. Receptor Tyrosine Kinases and TLR/IL1Rs Unexpectedly activate myeloid cell PI3K, a single convergent point promoting tumor inflammation and progression. Cancer Cell 2011;19:715-727. |
4www.clinicaltrials.gov, NCT02004522 |
5www.clinicaltrials.gov, NCT01882803 |
6www.clinicaltrials.gov, NCT02783625, NCT02158091 |
7 Schaller M.D. and Parsons J.T. Focal adhesion kinase: an integrin-linked protein tyrosine kinase. Trends Cell Biol. 1993 3: 258-62. |
8 Jiang H et al. Targeting focal adhesion kinase renders pancreatic cancers responsive to checkpoint immunotherapy. Nat Med 2016: Aug 22(8) 851-60. |
9 Sulzmaier F.J. et al. FAK in cancer: mechanistic findings and clinical applications. Nature Rev Cancer. 2014 14: 598-610. |
10www.clinicaltrials.gov, NCT02546531 |
11www.clinicaltrials.gov, NCT02943317 |
12www.clinicaltrials.gov, NCT02758587 |
Verastem, Inc. Condensed Consolidated Balance Sheets (in thousands) |
|||||||||
June 30, | December 31, | ||||||||
2018 | 2017 | ||||||||
(unaudited) | |||||||||
Cash, cash equivalents and investments | $ | 168,692 | $ | 86,672 | |||||
Prepaid expenses and other current assets | 1,745 | 1,115 | |||||||
Property and equipment, net | 1,270 | 861 | |||||||
Other assets | 1,211 | 1,143 | |||||||
Total assets | $ | 172,918 | $ | 89,791 | |||||
Accounts payable, accrued expenses and other current liabilities | $ | 22,132 | $ | 17,128 | |||||
Long-term debt | 23,520 | 14,828 | |||||||
Other liabilities | 399 | 151 | |||||||
Stockholders’ equity | 126,867 | 57,684 | |||||||
Total liabilities and stockholders’ equity | $ | 172,918 | $ | 89,791 | |||||
Verastem, Inc. Unaudited Condensed Consolidated Statements of Operations (in thousands, except per share amounts) |
||||||||||||||||
Three months ended June 30, | Six months ended June 30, | |||||||||||||||
2018 | 2017 | 2018 | 2017 | |||||||||||||
Revenue: | ||||||||||||||||
License revenue | $ | 10,000 | $ | — | $ | 10,000 | $ | — | ||||||||
Total revenue | 10,000 | — | 10,000 | — | ||||||||||||
Operating expenses: | ||||||||||||||||
Research and development | 12,381 | 9,042 | 23,315 | 17,427 | ||||||||||||
General and administrative | 15,813 | 4,425 | 25,640 | 9,188 | ||||||||||||
Total operating expenses | 28,194 | 13,467 | 48,955 | 26,615 | ||||||||||||
Loss from operations | (18,194 | ) | (13,467 | ) | (38,955 | ) | (26,615 | ) | ||||||||
Interest income | 343 | 140 | 534 | 295 | ||||||||||||
Interest expense | (516 | ) | (109 | ) | (996 | ) | (121 | ) | ||||||||
Net loss | $ | (18,367 | ) | $ | (13,436 | ) | $ | (39,417 | ) | $ | (26,441 | ) | ||||
Net loss per share—basic and diluted | $ | (0.30 | ) | $ | (0.36 | ) | $ | (0.70 | ) | $ | (0.71 | ) | ||||
Weighted-average number of common shares used in net loss per share-basic and diluted | 61,256 | 36,992 | 56,074 | 36,992 |
View source version on businesswire.com: https://www.businesswire.com/news/home/20180808005717/en/
Source:
Verastem Oncology
Marianne M. Lambertson
Vice
President, Corporate Communications
Investor Relations/Public
Relations
+1 781-292-4273
mlambertson@verastem.com