On March 9, 2017 Mirati Therapeutics, Inc. (NASDAQ: MRTX) ("the Company," "we," "our," "us," or "Mirati") reported financial results for the fourth quarter and year ended December 31, 2016 and provided an update on its product development programs (Press release, Mirati, MAR 9, 2017, View Source [SID1234518044]). Schedule your 30 min Free 1stOncology Demo! "We have made significant progress which positions us to report key data from all of our programs in 2017," said Charles M. Baum, M.D., Ph.D., President and CEO of Mirati. "Earlier this year, we presented glesatinib data demonstrating clinical responses in non-small cell lung cancer patients with MET driver alterations. In addition, preliminary data from our Phase 1b trial of sitravatinib has shown clinical benefit in patients with RET mutations. We plan to provide updates for both glesatinib and sitravatinib in the second half of this year.
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We are excited about our immuno-oncology programs which combine sitravatinib or mocetinostat with checkpoint inhibitors. There is strong scientific rationale that sitravatinib or mocetinostat can significantly enhance the efficacy of checkpoint inhibitors. Following our successful public offering in January, we are well positioned to achieve the 2017 milestones in each of our programs with funding into late 2018."
Single Agent Programs
Glesatinib (MGCD265)
The Company is enrolling patients in its registration-enabling Phase 2 non-small cell lung cancer (NSCLC) AMETHYST clinical trial, which is evaluating single agent glesatinib for the treatment of NSCLC patients with MET driver alterations. In January 2017, the Company reported early data that demonstrated promising activity in these molecularly selected patients. In NSCLC patients with MET Exon 14 deletion mutations treated with glesatinib, across both the Phase 1b and Phase 2 trials, tumor reduction was observed in 11 of 13 patients, with confirmed and unconfirmed partial responses in 6 of 13 evaluable patients. Glesatinib also demonstrated clinical benefit in NSCLC patients with MET gene amplification, including tumor reduction in six of eight patients as well as two unconfirmed partial responses out of eight evaluable patients. The Company expects to provide an update on efficacy data from the AMETHYST trial in the second half of 2017.
Sitravatinib (MGCD516)
The Company is enrolling patients in its Phase 1b expansion clinical trial, which is evaluating single agent sitravatinib for the treatment of NSCLC patients with RET, CHR4q12 and CBL genetic alterations. In January 2017, the Company reported that six NSCLC patients with RET fusion mutations had been enrolled and all four evaluable patients showed tumor reductions with one confirmed and one unconfirmed response. The Company expects to provide an update on efficacy data in the third quarter of 2017.
Immuno-oncology Combination Programs
Sitravatinib plus nivolumab
Sitravatinib is a potent inhibitor of the TAM (Tyro, Axl, Mer) and split (KDR, KIT) tyrosine kinase families which have been shown in preclinical studies to enhance anti-tumor immunity and the effects of checkpoint inhibitors. In November 2016, the Company initiated enrollment in a multicenter Phase 2 NSCLC clinical trial evaluating sitravatinib in combination with nivolumab, a PD-1 checkpoint inhibitor approved for the treatment of patients with NSCLC. The trial is enrolling patients who have relapsed after treatment with a checkpoint inhibitor. The Company expects to provide initial results from this combination trial in the second half of 2017.
Mocetinostat (MGCD103) plus durvalumab
The Company is collaborating with MedImmune/Astra Zeneca on a Phase 1b/2 clinical trial combining mocetinostat, an orally administered spectrum-selective Class 1 HDAC inhibitor, and durvalumab, MedImmune’s monoclonal antibody inhibiting PD-L1. Preclinical data have shown that mocetinostat significantly enhances the efficacy of the checkpoint inhibitor through its effects on the tumor microenvironment. The combination trial is exploring the potential of mocetinostat to enhance the effectiveness of durvalumab in NSCLC patients and the Company expects to provide an update in mid 2017.
Preclinical Programs
The Company’s lead preclinical programs to develop inhibitors of LSD1 and KRAS are expected to have meaningful milestones in 2017. The Company’s LSD1 inhibitor is highly-potent and potentially best-in-class with potential for rapid clinical proof-of-concept in small cell lung cancer and/or acute myeloid leukemia. An investigational new drug (IND) submission is planned for this compound in the fourth quarter of 2017. The Company has identified a mutant-selective KRAS inhibitor which is advancing to the candidate selection phase. Prototype inhibitors have demonstrated marked tumor regression in KRAS mutant tumor models and the Company anticipates selecting an IND candidate in the second half of 2017.
Financing
In January 2017, the Company strengthened its balance sheet by completing a public offering of common stock and pre-funded common stock warrants generating net proceeds of $66.8 million. Following this offering, the Company expects that its available cash, cash equivalents and short-term investments are sufficient to fund operations into late 2018.
Fourth Quarter and Full Year Financial Results
Cash, cash equivalents, and short-term investments were $56.7 million on December 31, 2016, as compared to $122.3 million on December 31, 2015.
Research and development expenses for the fourth quarter of 2016 were $16.0 million, compared to $14.9 million for the same period in 2015. Research and development expenses for the year ended December 31, 2016 were $68.5 million, compared to $49.0 million for the same period in 2015. The increases in research and development expenses for both periods primarily reflect costs to advance the clinical development of the Company’s glesatinib and sitravatinib oncology development programs, an increase in salaries and related expense, which is due to an increase in research and development employees during 2016 compared to the same period of 2015, and an increase in early discovery expenses.
General and administrative expenses for the fourth quarter of 2016 were $3.9 million, compared to $3.6 million for the same period in 2015. General and administrative expenses for the year ended December 31, 2016 were $15.3 million, compared to $15.8 million for the same period in 2015. The comparable level of expenses for both periods reflect a consistent level of general and administrative activities during both periods.
Other income, net, was $0.1 million for both the fourth quarter of 2016 and 2015. Other income, net, for the year ended December 31, 2016 was $0.6 million compared to $0.2 million for the same period in 2015.
Net loss for the fourth quarter of 2016 was $19.7 million, or $0.99 per share basic and diluted, compared to net loss of $18.4 million, or $0.96 per share basic and diluted for the same period in 2015. Net loss for the year ended December 31, 2016 was $83.1 million, or $4.20 per share basic and diluted, compared to net loss of $64.5 million, or $3.82 per share basic and diluted for the same period in 2015.
Epizyme Reports 2016 Financial Results and Provides 2017 Pipeline Goals
On March 9, 2017 Epizyme, Inc. (NASDAQ:EPZM), a clinical-stage biopharmaceutical company creating novel epigenetic therapies, reported financial results for the fourth quarter and full year 2016 (Press release, Epizyme, MAR 9, 2017, View Source [SID1234518042]). In addition, the Company reviewed recent highlights and provided guidance on its clinical and research pipeline in 2017.
“2016 was a year of tremendous progress across all aspects of Epizyme, which set the stage for 2017 to be a transformational year for the company,” said Robert Bazemore, president and chief executive officer of Epizyme. “We have a broad clinical development program with tazemetostat in multiple tumor types and treatment settings. We plan to present data from both our ongoing, global Phase 2 studies in both molecularly defined solid tumors and non-Hodgkin lymphoma in June, and to initiate discussions with regulators to define registration pathways for tazemetostat. We are advancing our ongoing monotherapy and combination studies and plan to announce the next development candidate from our preclinical pipeline this year. As we look ahead, we are on our way to achieving our vision of rewriting cancer treatment through novel epigenetic medicines.”
Recent Achievements
Tazemetostat Combination with Prednisolone Initiated: Epizyme has initiated clinical investigation of tazemetostat in combination with prednisolone in relapsed/refractory patients with DLBCL, based on observed preclinical synergy of the agents. This combination regimen is being conducted as the sixth cohort to the ongoing Phase 2 NHL study. Epizyme also anticipates initiating a new combination study in patients with follicular lymphoma in 2017.
Enrollment Completed in Three of Five NHL Study Cohorts: Epizyme has completed enrollment in three of the five cohorts of its ongoing, global Phase 2 study of tazemetostat in patients with relapsed/refractory non-Hodgkin lymphoma (NHL): the two cohorts enrolling patients with diffuse large B-cell lymphoma (DLBCL) with wild-type EZH2 and a third enrolling patients with follicular lymphoma with wild-type EZH2.
Follicular Lymphoma Enrollment Initiated in U.S.: In January 2017, following a positive written response from the U.S. Food and Drug Administration (FDA) to the company’s written request, Epizyme opened enrollment to patients with follicular lymphoma in the United States as part of the Company’s Phase 2 study in NHL.
Fast Track Designation Granted for DLBCL with EZH2 Mutations: In December 2016, the U.S. FDA granted Fast Track designation to tazemetostat for the treatment of patients with DLBCL whose tumors carry an EZH2 activating mutation. DLBCL is the most common form of NHL. The Fast Track designation provides expedited processes that may reduce development time and costs associated with bringing a drug to market.
Epithelioid Sarcoma Cohort Expanded in Phase 2 Solid Tumor Study: In late 2016, Epizyme expanded the epithelioid sarcoma cohort of its ongoing Phase 2 trial of tazemetostat in adult patients with certain molecularly defined solid tumors, following review by the Independent Data Monitoring Committee. This expansion was based on encouraging early activity, including confirmed objective responses, and surpassing the pre-specified futility hurdle in the cohort. This cohort will enroll up to 60 patients, or double the initial cohort size of 30 patients.
Synovial Sarcoma Cohort Completed Enrollment in Phase 2 Solid Tumor Study: The synovial sarcoma cohort of the Phase 2 trial in patients with molecularly defined solid tumors was fully enrolled in November 2016. This arm surpassed its pre-specified futility hurdle; however, the Company concluded that the activity was insufficient to continue further investigation of tazemetostat as a monotherapy. Epizyme is focusing its efforts on the remaining four cohorts of INI1-negative tumors.
Key 2017 Milestones and Goals
Interim Phase 2 Data Presentations Anticipated in the Second Quarter: Epizyme plans to report efficacy, safety and biomarker data from all five arms of the ongoing Phase 2 study in NHL, and from the study cohorts that have reached their futility analysis in the ongoing Phase 2 study in molecularly defined solid tumors. Pending abstract approval, the Company plans to present interim solid tumor data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Pending abstract submission and approval, the Company anticipates presenting interim data from all five arms of the ongoing Phase 2 NHL trial at The International Conference on Malignant Lymphoma (ICML).
Preparing for Regulatory Engagement beginning Mid-Year: Epizyme is preparing for regulatory engagement around its Phase 2 studies, beginning with the United States Food and Drug Administration (FDA) in mid-2017 on its molecularly defined solid tumor program. The Company is also preparing for FDA engagement on its NHL program in the second half of the year. These interactions are intended to determine registration paths for tazemetostat in each disease area.
Combination Studies Progressing: The combination studies of tazemetostat in front-line DLBCL with R-CHOP and in relapsed/refractory DLBCL with atezolizumab (Tecentriq) are continuing to enroll patients globally. Epizyme expects the optimal dose of tazemetostat in these combinations will be established this year, and for enrollment to continue to evaluate the safety and efficacy profile of both combinations.
Mesothelioma Study Progressing: Epizyme is continuing to enroll patients in its global Phase 2 study of tazemetostat in patients with mesothelioma characterized by BAP1 loss-of-function. The Company anticipates completing enrollment in this trial in 2017.
Next Development Candidate to be Named: Epizyme is progressing a pipeline of next-generation small molecules against novel epigenetic targets, and plans to name the next epigenetic development candidate from its pipeline in 2017. This is part of the Company’s goal of bringing three new product candidates into the clinic by 2020.
Fourth Quarter and Full-Year 2016 Financial Results
Cash Position: Cash, cash equivalents and marketable securities were $242.2 million as of December 31, 2016, as compared to $208.3 million as of December 31, 2015.
Revenue: Collaboration revenue was $0.5 million for the fourth quarter of 2016 and $8.0 million for the full year ended December 31, 2016, compared to $0.6 million for the fourth quarter of 2015 and $2.6 million for the full year ended December 31, 2015. The year-over-year increase was driven predominantly by the achievement of a $6.0 million milestone under the Company’s agreement with GSK during 2016.
R&D Expenses: Research and development (R&D) expenses were $28.4 million for the fourth quarter of 2016 and $91.5 million for the full year ended December 31, 2016, compared to $16.8 million for the fourth quarter of 2015 and $111.2 million for the full year ended December 31, 2015. R&D spending on discovery research programs and tazemetostat clinical development increased in both the quarter ended December 31, 2016 and, excluding the impact of the $40.0 million upfront payment to Eisai in the first quarter of 2015 to acquire worldwide rights, excluding Japan, to tazemetostat, in the year ended December 31, 2016.
G&A Expenses: General and administrative (G&A) expenses were $7.6 million for the fourth quarter of 2016 and $28.4 million for the full year ended December 31, 2016, compared to $6.0 million for the fourth quarter of 2015 and $23.9 million for the full year ended December 31, 2015. The increase was primarily due to higher compensation-related expenses associated with expansion of the senior leadership team in the first half of 2016 to support the Company’s operational growth, as well as increased investment in business development activities and tazemetostat-related pre-commercial activities.
Net Loss: Net loss was $35.0 million for the fourth quarter of 2016 and $110.2 million for the full year ended December 31, 2016, compared to $22.2 million for the fourth quarter of 2015 and $132.4 million for the full year ended December 31, 2015.
2017 Guidance
Epizyme believes that its cash, cash equivalents and marketable securities of $242.2 million as of December 31, 2016 will be sufficient to fund the Company’s planned operations into at least the third quarter of 2018.
Cascadian Therapeutics Reports Fourth Quarter and Full Year 2016 Financial Results
On March 9, 2017 Cascadian Therapeutics, Inc. (NASDAQ:CASC) reported financial results for the fourth quarter and full year ended December 31, 2016 (Press release, Cascadian Therapeutics, MAR 9, 2017, View Source [SID1234518040]). Schedule your 30 min Free 1stOncology Demo! "In 2016, we focused our efforts on the development of tucatinib for late-stage HER2-positive metastatic breast cancer for patients with and without brain metastases and amended our ongoing HER2CLIMB study by increasing the sample size so that, if successful, the trial could serve as a single pivotal trial to support registration," said Scott Myers, President and CEO of Cascadian Therapeutics. "For 2017, we have prioritized our resources on expanding HER2CLIMB globally and exploring the potential utility of tucatinib in additional HER2-positive expressing cancers. With a global development plan underway, clarity on a U.S. regulatory pathway and a solid financial position, we have set the foundation to execute our strategy."
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Fourth Quarter and Recent Highlights
Tucatinib — targeted HER2 inhibitor
In December 2016, researchers presented updated data from the Company’s ongoing Phase 1b Triplet combination study (tucatinib with capecitabine and trastuzumab) at the 2016 San Antonio Breast Cancer Symposium. This Triplet combination continued to be well tolerated and showed an updated median progression-free survival of 7.8 months, an overall response rate of 61 percent and a median duration of response of 10 months. Patients treated with the Triplet combination previously received a median of 3 HER2-targeted agents, such as trastuzumab, pertuzumab, lapatinib and T-DM1.
In December 2016, the Company announced that, following a meeting with the U.S. Food and Drug Administration (FDA) and discussions with the Company’s external Steering Committee, the Company amended the HER2CLIMB trial of tucatinib by increasing the sample size so that, if successful, the trial could serve as a single pivotal study to support a new drug application.
In October 2016, the Company announced presentation of data from the ongoing Phase 1b Triplet combination study (tucatinib with capecitabine and trastuzumab) at the European Society for Medical Oncology 2016 Congress that showed clinical activity in HER2-positive metastatic lesions to the skin.
CASC-578 — a novel Chk1 cell cycle inhibitor
In December 2016, the Company completed a non-human pharmacology study to evaluate the impact of CASC-578 on multiple cardiovascular endpoints. The results indicate CASC-578 has an acceptable profile at the doses tested and warrants further study of the drug in both single agent and combination settings.
Corporate
In January 2017, the Company strengthened its balance sheet through an underwritten public offering, resulting in net proceeds of approximately $88 million.
Fourth Quarter and Full Year 2016 Financial Results
Cash, cash equivalents and investments totaled $62.8 million as of December 31, 2016, compared to $56.4 million at December 31, 2015, an increase of $6.4 million, or 11.3 percent. The increase was primarily due to the result of net proceeds of $43.3 million from the Company’s June 2016 financing offset by cash used to fund operations of $36.9 million.
Net loss attributable to common stockholders for the three months ended December 31, 2016 was $10.5 million, or $0.47 per share, compared to a net loss attributable to common stockholders of $9.1 million, or $0.58 per share, for the same period in 2015. The $1.4 million increase in net loss attributable to common stockholders for the quarter was primarily due to an increase in research and development expense associated with the development of the Company’s product candidates and an increase in general and administrative expense, which included expenses related to headcount and the Company’s adoption of the Retention Plan in early January 2016.
Net loss attributable to common stockholders for the year ended December 31, 2016 was $60.3 million, or $3.13 per share, compared to a net loss attributable to common stockholders of $32.6 million, or $2.02 per share, for the same period in 2015. The increase in net loss attributable to common stockholders for the year ended December 31, 2016 was primarily due to the intangible asset impairment charge of $19.7 million, which was the result of the mutual termination of the STC.UNM agreement. In addition, the increases in research and development expenses of $4.0 million, due to greater activity related to the development of the Company’s product candidates, and increases in general and administrative expenses of $8.3 million, primarily related to the retirement and separation of the former chief executive officer and other headcount-related expenses, contributed to the year-over-year increase in net loss. The Company also recognized a non-cash $2.6 million deemed dividend due to the beneficial conversion feature on the Series D convertible preferred stock. The increase in the net loss attributable to common stockholders was partially offset by a $6.9 million tax benefit during the year ended December 31, 2016.
2017 Financial Outlook
Cascadian Therapeutics believes the following financial guidance to be correct as of the date provided and is providing the guidance as a convenience to investors and assumes no obligation to update it.
Cascadian Therapeutics expects operating expenses in 2017 to be slightly higher than in 2016, which included a one-time expense associated with the intangible asset impairment. The 2017 increase is due to an increase in activities related to the ongoing HER2CLIMB pivotal trial. Cash used in operations is expected to be approximately $50.0 million to $54.0 million.
Agenus Reports Fourth Quarter and Full Year 2016 Results
On March 9, 2017 Agenus Inc. (NASDAQ: AGEN), an immuno-oncology (I-O) company with a pipeline of immune checkpoint antibodies and cancer vaccines, reported a corporate update and reported financial results for the fourth quarter and year ended December 31, 2016 (Filing, Q4/Annual, Agenus, 2016, MAR 9, 2017, View Source [SID1234518039]). Schedule your 30 min Free 1stOncology Demo! "Actions we took last year put us on a path to register our lead antibodies that target CTLA-4 and PD-1 in the next four years. We also advanced programs directed at novel targets, such as 4-1BB and TIGIT, and upgraded our Berkeley manufacturing facility," said Garo H. Armen, Ph.D., Chairman and CEO of Agenus. "As we enter 2017, we have strengthened our balance sheet with our recently announced Incyte transaction resulting in a cash infusion of $80 million and a reduction of our cash burn rate for 2017 and beyond."
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Incyte Collaboration
Earlier this year, Agenus amended its collaboration with Incyte, resulting in $80 million of cash to Agenus: $60 million from an equity investment at $6/share plus $20 million in accelerated clinical development milestones for the GITR and OX40 programs. In addition, these programs were converted from co-funded development and profit-share arrangements to royalty-bearing programs at a 15% royalty rate, with Agenus eligible for up to $510 million in future milestones.
UCB Collaboration
Agenus entered into a research collaboration with UCB to advance the development of multi-specific therapeutic antibodies. The collaboration presents a unique opportunity to discover novel therapeutics. This approach has the potential to expedite the development of Agenus’ portfolio of discovery programs focused on the next generation of I-O targets.
2017 Anticipated Milestones:
Start Phase 1 dose-escalation trial for anti-PD-1 antagonist AGEN2034 in H1.
Start Phase 1b combination trial with AGEN1884 (CTLA-4) and AGEN2034 (PD-1) in H2.
Start Phase 1 trial for AutoSynVax in H1.
Start cervical cancer trial for PD-1 monotherapy in H2.
Readouts of AutoSynVax immunogenicity in H2.
Execute additional strategic transactions.
2016 Select Highlights:
Research & Development
Started Phase 1 dose escalation trial of AGEN 1884, Agenus’ proprietary anti-CTLA-4 antibody.
Started Phase 1 trial for INCAGN1876, anti-GITR antibody in partnership with Incyte.
Started Phase 1 trial for INCAGN1949, anti-OX40 antibody in partnership with Incyte.
Advanced collaboration with Merck with the selection of a lead product candidate.
GlaxoSmithKline filed for regulatory approval of Shingrix vaccine containing Agenus’ QS-21 Stimulon.
Leadership
Appointed Ulf Wiinberg to Board of Directors, bringing three decades of leadership experience in the global biopharmaceutical industry to our governance team.
Appointed Dr. Jean-Marie Cuillerot as Vice President and Global Head of Clinical Development, who has since assumed the role of a Chief Medical Officer. Dr. Cuillerot has been integral to the development of Yervoy and Avelumab during his tenure at BMS and Merck Serono.
Fourth Quarter 2016 Financial Results
Cash, cash equivalents and short-term investments were $76.4 million as of December 31, 2016. Subsequent to the end of the year, Agenus received $80 million in cash as part of the amended partnership and stock purchase agreement with Incyte. The increased cash combined with substantially reduced clinical development expense obligations under the prior Incyte agreement, will significantly reduce our cash burn and extend our cash runway through the second quarter of 2018.
For the fourth quarter, Agenus reported a net loss of $26.1 million, or $0.30 per share, compared with a net loss for the fourth quarter of 2015 of $15.6 million, or $0.18 per share. The company’s cash burn for the quarter was approximately $19.0 million compared to approximately $27.9 million during the third quarter.
The increased net loss for the quarter ended December 31, 2016, compared to the same period in 2015, was due primarily to the expansion and growth of the research activities at the Company partially offset by non-cash income for the quarter ended December 31, 2016 of $9.4 million, due to the fair value adjustment of the contingent purchase price considerations compared to $623,000 for the same period in 2015. In addition, during the quarter ended December 31, 2015 we recorded a $5.4 million income tax benefit recognized as a result of our 2015 acquisitions.
For the year ended December 31, 2016, the Company incurred a net loss of $127 million, or $1.46 per share, compared with a net loss of $88 million, or $1.13 per share, in the same period in 2015.
The increase in net loss for the year ended December 31, 2016, compared to the net loss for the same period in 2015, was primarily due to the Company’s growth and to the advancement of our programs, and increased interest expense on our long-term debt partially offset by the decreased non-cash expense for fair value adjustments to our contingent obligations.
Servier and Pfizer announce FDA clearance of IND application for UCART19 in Adult Relapsed/Refractory Acute Lymphoblastic Leukemia
On March 9, 2017 Servier, together with Pfizer Inc. (NYSE:PFE) and Cellectis (Alternext: ALCLS; Nasdaq: CLLS), reported that the U.S. Food and Drug Administration (FDA) has granted Servier with an Investigational New Drug (IND) clearance to proceed in the U.S. with the clinical development of UCART19, an allogeneic, gene-edited cellular therapy candidate to treat relapsed/refractory acute lymphoblastic leukemia (Press release, Cellectis, MAR 9, 2017, View Source [SID1234518037]). Schedule your 30 min Free 1stOncology Demo! Servier is sponsoring the CALM Phase 1 study on UCART19. In 2015, Servier acquired exclusive rights from Cellectis for UCART19, which is being co-developed by Servier and Pfizer.
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The CALM study was initiated in the UK in August 2016. CALM is an open label, dose-escalation study designed to evaluate safety, tolerability and antileukemic activity of UCART19 in patients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia (B-ALL).
The allogeneic UCART19 candidate and CALM protocol were reviewed at the National Institutes of Health’s Recombinant DNA Advisory Committee (RAC) meeting on December 14, 2016. Servier submitted an IND application on February 1, 2017, with Pfizer’s support. With this IND clearance, the CALM study will be expanded to include several centers in the U.S., including the MD Anderson Cancer Center in Houston (Texas).
"We are very pleased that Servier’s first IND approval has been granted for such an innovative approach as allogeneic CAR T therapy", said Dr Patrick Thérasse, Director of Clinical Development Oncology at Servier. "B-ALL is a devastating disease and this study is key to gaining greater insight into the efficacy and safety profile of this new immune-oncology approach in patients with B-ALL."
"Pfizer is excited by the potential of this investigational CAR T approach to treating ALL and other B-Cell malignancies," said Barbara Sasu, Vice President, CAR T Research at Pfizer. "We are looking forward to having the opportunity to investigate this approach in the U.S."
About UCART19
UCART19 is an allogeneic CAR T-cell product candidate being developed for treatment of CD19-expressing hematological malignancies, gene edited with TALEN. UCART19 is initially being developed in acute lymphoblastic leukemia (ALL) and is currently in Phase I. The current approach with UCART19 is based on the preliminary positive results from clinical trials using autologous products based on the CAR technology. UCART19 has the potential to overcome the limitation of the current autologous approach by providing an allogeneic, frozen, "off-the-shelf" T cell based medicinal product.
In November 2015, Servier acquired the exclusive rights to UCART19 from Cellectis. Following further agreements, Servier and Pfizer began collaborating on a joint clinical development program for this cancer immunotherapy. Pfizer has been granted exclusive rights by Servier to develop and commercialize UCART19 in the United States, while Servier retains exclusive rights for all other countries.