Gamida Cell Reports First Quarter 2019 Financial Results and Provides Company Update
– Announces USAN selection of omidubicel as nonproprietary name for company’s investigational NAM-expanded hematopoietic stem cells (formerly known as NiCord®) –
– Patient enrollment in Phase 3 study of omidubicelexpected to be completed in second half of 2019; Topline results expected in first half of 2020 –
– Phase 1 clinical study of GDA-201 (formerly known as NAM-NK) continues to progress, with additional data expected in 2H19 –
“Gamida Cell is focused on transforming the treatment landscape for
patients with blood cancers and rare, serious hematologic diseases. We
are pleased that omidubicel has been selected as the nonproprietary name
for NiCord, highlighting our progress toward bringing this important
cell therapy to patients in need of a bone marrow transplant,” stated
Dr. Adams continued, “We are pleased that the multi-center, randomized Phase 3 study of omidubicel is progressing, with patient enrollment expected to be complete by the end of this year and topline data anticipated in the first half of 2020. Positive data from the study would enable the submission of our first biologics license application next year, which would be a significant achievement.”
“Earlier this year, we also reported encouraging data from the Phase 1 clinical study of our natural killer cell product candidate, GDA-201, previously known as NAM-NK. The multiple complete responses observed emboldened us to begin scaling up our manufacturing process to enable the evaluation of a cryopreserved formulation of GDA-201 in a multi-center, multi-dose Phase 1/2 clinical study in patients with non-Hodgkin lymphoma next year,” Dr. Adams concluded.
- Omidubicel selected as nonproprietary name for NiCord: Today
Gamida Cellannounced that the United States Adopted Names (USAN) Councilselected omidubicel as the nonproprietary name for Gamida Cell’s investigational hematopoietic stem cell expanded through the company’s proprietary nicotinamide-based, or NAM, technology. The USAN Councilaims for global standardization and unification of drug nomenclature to ensure that drug information is communicated accurately and unambiguously. Gamida Cell’s lead investigational product has two components: omidubicel (hematopoietic stem cells expanded through the company’s proprietary nicotinamide-based, or NAM, technology) and differentiated immune cells, including T cells. Gamida Cellrefers to the two components collectively as “omidubicel.”Going forward, Gamida Cellwill use the name “omidubicel” in publications and public statements, at conferences and other forums, and in medical and commercial-related materials.
- Reported encouraging data for omidubicel and GDA-201 at TCT Annual
Meeting: In February, data from the omidubicel and GDA-201
clinical programs were reported at the 2019 Transplantation & Cellular
Therapy (TCT) Meetings of
American Society for Blood and Marrow Transplantationand Center for International Blood and Marrow Transplant. Research from the completed Phase 1/2 clinical study of omidubicel demonstrated that recipients who received omidubicel had rapid and robust reconstitution of key immune cells. Successful immune reconstitution is an important factor in the recovery of patients undergoing bone marrow transplant.
Data were also reported from the ongoing Phase 1/2 study of omidubicel in patients with severe aplastic anemia. In the initial cohort of three patients, all successfully underwent a bone marrow transplant consisting of omidubicel plus a haploidentical stem cell graft. The results enable the initiation of a second cohort of patients to be treated with omidubicel as a stand-alone graft.Patient enrollment in the second cohort is expected to begin in the first half of 2019.
Additionally, data reported from the ongoing Phase 1 study of GDA-201 in patients with non-Hodgkin lymphoma (
NHL) and multiple myeloma (MM) demonstrated that GDA-201 was clinically active, with three complete responses observed in patients with NHLand one complete response in a patient with MM. These data, along with safety data showing that GDA-201 was generally well tolerated, support continued clinical development. Gamida Cellis planning to initiate a multi-center, Phase 1/2 clinical study of GDA-201 in patients with NHLin 2020.
- Evolved Board of Directors to reflect company’s progress toward
commercialization: In March,the company announced the
Shawn Cline Tomaselloand Stephen T. Willsto its board of directors. These nominations require approval at the Annual Shareholders Meeting, which will take place in June 2019. Ms. Tomasello has extensive experience in commercializing first-in-class medicines for the treatment of cancer, including Yescarta® (at Kite Pharma, now part of Gilead Sciences) and Imbruvica® (at Pharmacyclics, now part of AbbVie). Mr. Wills has extensive operational, financial and transactional experience over nearly three decades in the life sciences and accounting industries. He has served as chief financial officer of Palatin Technologies, a publicly-traded biotechnology company developing peptide therapeutics, since 1997 and also serves as Palatin’s chief operating officer and executive vice president.
In January, the company appointed
Nurit Benjaminito Gamida Cell’s board of directors and chair of the board’s audit committee. Ms. Benjamini has served as chief financial officer of TabTale Ltd.since 2013. Previously, she held a number of chief financial officer positions, including at Wix.com Ltd., Sigma Designs Israel Ltd.and Compugen Ltd.
Thomas Klimaas chief commercial officer: In January, the company announced the appointment of Thomas Klimaas chief commercial officer.In this newly created role, Mr. Klima will be responsible for building the team and executing the strategy to potentially bring omidubicel to patients, including oversight of reimbursement and patient services.Klima brings nearly 20 years of global experience in the pharmaceutical industry with expertise in cellular therapy, hematology, oncology and transplantation. During his career, he has played key roles in building commercial organizations and leading multiple successful product launches.
Anticipated 2019-2020 Milestones
Gamida Cell’s anticipated program milestones in 2019-2020 are as follows:
- Initiate Cohort 2 in the Phase 1/2 study evaluating omidubicel as stand-alone graft in severe aplastic anemia in the first half of 2019
- Complete enrollment in Phase 3 study of omidubicel in patients with hematologic malignancies in the second half of 2019
- Report topline data from the Phase 3 study of omidubicel in patients with hematologic malignancies in the first half of 2020
- Complete BLA submission for omidubicel in hematologic malignancies in the second half of 2020, should Phase 3 data be positive
- Complete patient enrollment in the ongoing Phase 1 study in the second half of 2019
- Present additional data at a medical meeting in the second half of 2019
Initiate multi-center, Phase 1/2 clinical study in patients with
First Quarter 2019 Financial Results
March 31, 2019, Gamida Cellhad total cash, cash equivalents and available-for-sale securities of $50.3 million, compared to $60.7 millionas of December 31, 2018.
Research and development expenses in the first quarter of 2019 were
$7.3 million, compared to $5.1 millionin the same period in 2018. The difference was attributable mainly to a $1.2 millionincrease in clinical activities relate to the advancement of omidubicel and GDA-201, $0.5 millionreduction in grants received from the Israeli Innovation Authority(IIA) and an increase of $0.5 millionin compensation and other R&D expenses.
General and administrative expenses were
$3.8 millionfor the first quarter of 2019, compared to $1.7 millionin the same period in 2018. The increase was due mainly to a $1.0 millionincrease in expenses related to hiring and establishing the U.S. headquarters, an increase of $0.5 millionin non-cash stock-based compensation expenses, and $0.6 millionin professional services, rent and other expenses.
Finance expenses, net, were
$4.4 millionfor the three months ended March 31, 2019, compared to $0.7 millionin income in the same period in 2018. The increase was primarily due to noncash expenses resulting from revaluation of warrants and the revaluation of royalty-bearing grant IIA liability.
Net loss for the first quarter of 2019 was
$15.5 million, compared to a net loss of $7.4 millionin the same period in 2018.
2019 Financial Guidance
Conference Call Information
Omidubicel (formerly known as NiCord®), the company’s lead clinical program, is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers).1 Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the
Omidubicel and GDA-201 are investigational therapies, and their
safety and efficacy have not been evaluated by the
Cautionary Note Regarding Forward Looking Statements
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to the patient enrollment in and timing of initiation and progress of and data reported from the clinical trials of Gamida Cell’s product candidates, and Gamida Cell’s expectations regarding its projected operating expenses and cash runway, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Gamida Cell’s clinical trials and variability, and ramifications for the cost thereof; and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section of Gamida Cell’s public filing on Form 20-F, filed with the
|INTERIM CONSOLIDATEDSTATEMENTS OF FINANCIAL POSITION|
|U.S. dollars in thousands|
|March 31,||December 31,|
|Cash and cash equivalents||$||43,749||$||40,272|
|Available-for-sale financial assets||6,507||20,417|
|Prepaid expenses and other current assets||684||1,502|
|Property and equipment, net||2,782||2,311|
LIABILITIES AND EQUITY
|Employees and payroll accruals||2,580||2,888|
|Current maturities of lease liabilities||2,156||-|
|Accrued expenses and other payables||1,739||1,832|
Total current liabilities
|Liabilities presented at fair value||25,031||24,049|
|Employee benefit liabilities, net||276||183|
|Liability to Israel Innovation Authority (IIA)||10,108||9,540|
Total non-current liabilities
|Capital reserve due to actuarial gains||(160)||(77)|
Total shareholders' equity
Total liabilities and shareholders' equity
1 Gamida Cell’s lead development candidate consists of
omidubicel (expanded hematopoietic stem cells) and differentiated immune
cells, including T cells.
2 Horwitz M.E., Wease S., Blackwell B., Valcarcel D. et al. Phase I/II study of stem-cell transplantation using a single cord blood unit expanded ex vivo with nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367-374.
3 ClinicalTrials.gov identifier NCT02730299.
4 ClinicalTrials.gov identifier NCT03173937.
5 ClinicalTrials.gov identifier NCT03019666.
Jaren Irene Madden
Krystle Gibbs (media)