Gamida Cell Reports Fourth Quarter and Full Year 2018 Financial Results and Provides Company Update
– Successfully Completed IPO Raising
– Patient Enrollment in Phase 3 Study of NiCord® on Track for Completion in Second Half of 2019; Topline Results Expected in First Half of 2020 –
– Recently Presented NAM-NK and NiCord Data at 2019 TCT Annual Meeting Demonstrate Clinical Progress and Potential of Proprietary NAM Expansion Technology –
“We are off to a strong start this year and are making meaningful
progress across every dimension of our company. The data presented last
week at the TCT Annual Meeting demonstrates the potential for NiCord to
offer a new cell therapy option for patients in need of a bone marrow
Dr. Adams continued, “We are committed to leveraging the transformative potential of our proprietary nicotinamide-, or NAM-based, cell expansion technology, to deliver a multi-product pipeline. Last week, we presented encouraging data from our ongoing Phase 1 study of NAM-NK in patients with non-Hodgkin lymphoma and multiple myeloma, and the emerging clinical profile suggests it has the potential to address some of the limitations of currently available cell therapies. In addition, we have demonstrated the ability to cryopreserve our NK cells in a laboratory setting, and we are working to scale up our manufacturing process to enable the ability to evaluate multiple doses and schedules of NAM-NK therapy in a multi-center, Phase 1/2 clinical study next year.”
Recent Company Highlights:
- Reported additional immune reconstitution data for NiCord
supporting clinical potential for bone marrow transplant: In
February, translational data from the completed Phase 1/2 study of
NiCord 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 Researchdemonstrating that recipients who received NiCord 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.
- Reported initial data from Phase 1/2 study of NiCord in severe aplastic anemia: At the 2019 TCT Annual meeting, data were reported from the ongoing Phase 1/2 study of NiCord in patients with severe aplastic anemia. In the initial cohort of three patients, all successfully underwent a bone marrow transplant consisting of NiCord plus a haploidentical stem cell graft. The rapid cord engraftment, sustained hematopoiesis and accelerated immune recovery in treatment refractory observed in these patients enable the initiation of a second cohort of patients to be treated with NiCord as a stand-alone graft.Patient enrollment in the second cohort is expected to begin in the first half of 2019.
- Announced encouraging data from Phase 1 study of NAM-NK in
non-Hodgkin Lymphoma and multiple myeloma: During the 2019 TCT
Annual Meeting, data reported from the ongoing Phase 1 study of NAM-NK
in patients with non-Hodgkin lymphoma (
NHL) and multiple myeloma (MM) demonstrated that NAM-NK 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 NAM-NK was generally well tolerated, support continued clinical development, and Gamida Cellis planning to initiate a multi-center, Phase 1/2 clinical study of NAM-NK in patients with blood cancers in 2020.
- Entered into agreement with
Editas Medicineto evaluate potential to gene edit NAM-NK cells: In February, Gamida Cellannounced an agreement with Editas Medicine, Inc.to evaluate the potential use of Editas Medicine’s CRISPR technology to edit NAM-NK cells. Through this agreement, the companies aim to rapidly discover optimized NAM-NK cells that could be used to improve the treatment of blood cancers and solid tumors.
- Formed strategic collaboration with Be The Match BioTherapies: In
January 2019, both organizations announced a collaboration to expand the use of bone marrow transplant to treat hematologic malignancies and serious blood disorders. Under the terms of the collaboration agreement, the organizations will explore opportunities to work together across Gamida Cell’s ongoing clinical development program for NiCord, including the ongoing Phase 3 clinical study. Be The Match BioTherapies has an extensive history of involvement in the delivery of cord blood units for transplant and broad access to cord blood banks globally.
Thomas Klimaas Chief Commercial Officer: In January, the company announced the appointment of Thomas Klimaas Chief Commercial Officer.Mr.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.
Nurit Benjaminito Board of Directors: 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.
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 NiCord as stand-alone graft in severe aplastic anemia in the first half of 2019
- Complete enrollment in Phase 3 study of NiCord in patients with hematologic malignancies in the second half of 2019
- Report topline data from the Phase 3 study of NiCord in patients with hematologic malignancies in the first half of 2020
- Complete BLA filing for NiCord 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 2020
Fourth Quarter and Full Year 2018 Financial Results:
December 31, 2018, Gamida Cellhad total cash, cash equivalents and available-for-sale securities of $60.7 million, compared to $41.1 millionat December 31, 2017.
Research and Development expenses in 2018 were
$22.0 million, compared to $15.0 millionin 2017. The increase was attributable mainly to a $5.4 millionincrease in clinical activities relating to the advancement of Gamida Cell’s clinical programs as well as an increase of $1.6 millionin other R&D expenses.
General and administrative expenses were
$11.6 millionin 2018, compared to $4.5 millionin 2017. The increase was attributable mainly to a $2.9 millionincrease in expenses related to expanding the management team and establishing the U.S. headquarters, an increase of $2.0 millionin non-cash stock-based compensation expenses, and $2.2 millionin professional services expenses incurred during the IPO process, rent and other expenses.
Finance expenses, net, were
$19.2 millionin 2018, compared to $0.5 millionin income, in 2017. The increase was primarily due to non-cash expenses resulting from revaluation of warrants and the revaluation of royalty-bearing grant IIA liability.
Net loss for 2018 was
$52.9 million, compared to a net loss of $19.1 millionfor 2017.
2019 Financial Guidance
NiCord, the company’s lead clinical program, is under development as a universal bone marrow transplant solution for patients with high-risk hematologic malignancies. NiCord has been granted Breakthrough Therapy designation by the
NAM-NK and NiCord are investigational therapies, and their safety and
efficacy have not been evaluated by the
This press release contains forward-looking statements as that term is
defined in the Private Securities Litigation Reform Act of 1995,
including with respect to the patient enrollment in and timing of
initiation and progress of and data reported from the clinical trials of
Gamida Cell’s product candidates, and Gamida Cell’s expectations
regarding its projected operating expenses and cash runway, which
statements are subject to a number of risks, uncertainties and
assumptions, including, but not limited to the scope, progress and
expansion of Gamida Cell’s clinical trials and variability, and
ramifications for the cost thereof; and clinical, scientific, regulatory
and technical developments. In light of these risks and uncertainties,
and other risks and uncertainties that are described in the Risk Factors
section of Gamida Cell’s public filing on Form 20-F, filed with
the SEC on
|CONSOLIDATED STATEMENTS OF FINANCIAL POSITION|
|U.S. dollars in thousands|
|Cash and cash equivalents||$||40,272||$||21,325|
|Available-for-sale financial assets||20,417||14,758|
|Short term deposits||-||5,000|
|Prepaid expenses and other current assets||1,502||2,539|
|Total current assets||62,191||43,622|
|Property and equipment, net||2,311||940|
|Total non-current assets||2,973||1,300|
|LIABILITIES AND SHAREHOLDERS' EQUITY|
|Employees and payroll accruals||2,888||1,517|
|Accrued expenses and other payables||1,832||669|
|Total current liabilities||6,705||4,576|
|Warrants presented at fair value||24,049||10,300|
|Employee benefit liabilities, net||183||200|
|Liability to Israel Innovation Authority (IIA)||9,540||6,890|
|Total non-current liabilities||33,772||17,390|
|CONTINGENT LIABILITIES AND COMMITMENTS|
|Capital reserve due to actuarial loss||(77)||(79)|
|Available for sale reserve||(43)||(34)|
|Total shareholders' equity||24,687||22,956|
|Total liabilities and shareholders' equity||$||65,164||$||44,922|
|CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS|
|U.S. dollars in thousands|
|Research and development expenses, net||$||22,045||$||15,018|
|General and administrative expenses||11,599||4,472|
|Loss before taxes on income||52,861||19,011|
|Taxes on income||70||-|
|Other comprehensive loss:||7||69|
|Total comprehensive loss||$||52,938||$||19,080|
|Loss per share - basic and diluted (in U.S. dollars)||$||10.53||$||27.56|
Jaren Irene Madden