Gamida Cell Reports Full Year 2020 Financial Results and Provides Company Update
– Primary and secondary endpoints were met, key exploratory endpoints supported clinical benefit in Phase 3 study of omidubicel in patients with hematologic malignancies; BLA submission anticipated in fourth quarter of 2021-
— Omidubicel commercial preparation underway, including the creation of Gamida Cell Assist, to support potential launch —
– GDA-201 demonstrated significant clinical activity in Phase 1 study of patients with non-Hodgkin lymphoma, with multiple complete responses observed; Phase 1/2 clinical trial planned with IND submission anticipated in the second half of 2021—
– Strengthened financial position with sale of
– Company to host conference call at
“It has been a significant year for
“We believe our NAM-based cell expansion technology has potential for NK cell expansion and we are developing GDA-201, an NK-cell immunotherapy for the treatment of hematologic and solid tumors in combination with antibody therapies. This year, we made meaningful progress with GDA-201, which has demonstrated impressive early results in patients with heavily pre-treated NHL in a Phase 1 investigator-sponsored study. Following these results, we plan to submit an IND to the FDA in the second half of 2021 and initiate a Phase 1/2 study,”
Omidubicel, an investigational advanced cell therapy for allogeneic bone marrow transplant
During the year,
In May,
The Phase 3 study additionally met key secondary endpoints related to platelet engraftment, infections and hospitalization, all significant clinical measures in bone marrow transplant, as reported in
Recently, the results of the company’s Phase 3 study of omidubicel were presented at the Transplantation & Cellular Therapy Meetings of the
Additional omidubicel highlights:
- Presented new Phase 1 data from study of omidubicel in patients with severe aplastic anemia (SAA) at ASH: In a poster presentation at ASH,
Gamida Cell presented data demonstrating that patients with severe aplastic anemia treated with omidubicel achieved sustained early engraftment and robust immune reconstitution following reduced intensity conditioning. The data suggest that omidubicel can result in rapid engraftment and can achieve sustained hematopoiesis in patients who are at high risk for graft failure with conventional umbilical cord blood transplant. The study remains open for accrual of patients with SAA.
- Advanced commercial launch readiness:
Gamida Cell recently announced plans for the Gamida Cell Assist program. The transplant process can be challenging and complex for the patient, caregivers and the entire transplant care team. Gamida Cell Assist has been designed to focus on patient access and support of every individual and their caregivers at each step of the process. Once the program is launched, the Gamida Cell Assist case management team will provide a consistent, single point of contact for patients and health care professionals, and work with the transplant center to track production of omidubicel for each individual patient and provide real-time updates on the status of the therapy. The services provided will include coverage and reimbursement support, which may include financial, travel, and lodging assistance.Gamida Cell is committed to supporting a positive journey for patients and their transplant teams so they can focus on what matters most – the patient experience and successful clinical outcomes.
- Expanded collaboration with Be The Match BioTherapies®: In October,
Gamida Cell and Be The Match Therapies® expanded their existing strategic collaboration for omidubicel. In building upon the existing collaboration,Gamida Cell will work through Be The Match BioTherapies® for the supply of cord blood units, which serve as the starting material for omidubicel. The expanded agreement is designed to provide a smooth process throughout the omidubicel therapy supply chain.
GDA-201, an innate NK cell immunotherapy
- Presented updated Phase 1 data at the 62nd ASH Annual Meeting: In December,
Gamida Cell announced updated data from the ongoing Phase 1 study of GDA-201 in combination with monoclonal antibodies in patients with NHL and multiple myeloma at the ASH Annual Meeting. GDA-201 in combination with rituximab demonstrated significant clinical activity in relapsed and refractory NHL patients, with 13 complete responses and one partial response observed in the first 19 NHL patients, for an overall response rate of 74 percent. Overall survival and progression-free survival at one year in the NHL cohort suggest durable disease control, with a median follow-up of ten months (range 1–28 months), in heavily pretreated patients. Additionally, there were no dose-limiting toxicities, neurotoxic events, confirmed cytokine release syndrome, GvHD or marrow aplasia.
- Continued advancing Phase 1 study of GDA-201:
Gamida Cell continues to advance activities to enable the submission of an investigational new drug (IND) application for cryopreserved, off-the-shelf GDA-201 to enable a multi-center, Phase 1/2 clinical study in patients with NHL in the second half of 2021.Gamida Cell is pioneering a novel approach that harnesses the power of its cell expansion technology, which uniquely improves antibody-dependent cellular cytotoxicity and tumor targeting of NK cells.
Corporate Highlights
- Strengthened financial position: In December 2020, the company executed an underwritten public offering raising approximately
$75 million before deducting underwriting discounts, commissions and offering expenses. Also, in February 2021, the company announced a$75 million financing withHighbridge Capital Management, LLC before deducting offering expenses. These capital infusions will be used to support manufacturing, regulatory and potential commercial development activities for omidubicel and to further the preclinical and clinical development of GDA-201.
Full Year 2020 Financial Results
- Research and development (R&D) expenses in 2020 were
$41.4 million , compared to$31.5 million in 2019. The increase was mainly due to advancing the GDA-201 clinical program and clinical activities relating to concluding our Phase 3 clinical trial, as well as additional headcount within the R&D organization.
- Commercial expenses in 2020 were
$8.7 million , compared to$4.7 million in 2019. The increase was attributed to an increase in omidubicel commercial readiness activities as well as additional headcount within the Commercial organization.
- General and administrative expenses were
$12.2 million in 2020, compared to$12.1 million in 2019. The increase was mainly due to a$1.3 million increase in professional services expenses, including Legal and Insurance, offset by decrease of$1.2 million in Travel and non-cash compensation expenses.
- Finance expenses, net, was
$10.4 million for 2020, compared to finance income, net, of$13.8 million for 2019. The decrease was primarily due to non-cash expenses resulting from revaluation of warrants owned by certain of the company’s shareholders and the revaluation of theIsraeli Innovation Authority royalty-bearing grant liability.
- Net loss for 2020 was
$72.7 million , compared to a net loss of$34.4 million in 2019.
- As of
December 31, 2020 ,Gamida Cell had total cash and cash equivalents of$127.2 million , compared to$55.4 million as ofDecember 31, 2019 . In addition, onFebruary 16, 2020 ,Gamida Cell announced the sale of$75 million exchangeable senior notes due in 2026 toHighbridge Capital Management, LLC .
2021 Financial Guidance
Expected 2021 Milestones
Omidubicel
- BLA submission to the FDA in the fourth quarter of 2021
- Commercial readiness activities underway for potential launch at approval
GDA-201
- Submit company-sponsored IND application to the FDA and initiate a Phase 1/2 clinical study in NHL in the second half of 2021
Conference Call Information
About Omidubicel
Omidubicel 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). In both Phase 1/2 and Phase 3 clinical studies (NCT01816230, NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.1,2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn®, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.
Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.
About GDA-201
GDA-201 is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.
About
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 timing of initiation and progress of and data reported from the clinical trials of Gamida Cell’s product candidates, anticipated regulatory filings, commercialization efforts and Gamida Cell’s expectations regarding its projected ongoing operating activities 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 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 and other sections of Gamida Cell’s Annual Report on Form 20-F, filed with the
CONSOLIDATED STATEMENTS OF FINANCIAL POSITION |
||||||||||
|
|
|
|
|
||||||
|
|
|
|
|
2020 |
|
|
2019 |
||
ASSETS |
|
|
|
|
|
|||||
|
|
|
|
|
|
|||||
CURRENT ASSETS: |
|
|
|
|
|
|||||
Cash and cash equivalents |
|
|
|
$ |
127,170 |
|
|
$ |
41,838 |
|
Marketable securities |
|
|
|
|
- |
|
|
13,559 |
||
Prepaid expenses and other current assets |
|
|
|
|
2,815 |
|
|
1,306 |
||
Total current assets |
|
|
|
|
129,985 |
|
|
56,703 |
||
|
|
|
|
|
|
|
||||
NON-CURRENT ASSETS: |
|
|
|
|
|
|
||||
Property, plant and equipment, net |
|
|
|
|
18,238 |
|
|
6,298 |
||
Right-of-use assets |
|
|
|
|
6,474 |
|
|
5,133 |
||
Other assets |
|
|
|
|
786 |
|
|
641 |
||
Total non-current assets |
|
|
|
|
25,498 |
|
|
12,072 |
||
Total assets |
|
|
|
$ |
155,483 |
|
|
$ |
68,775 |
|
LIABILITIES AND SHAREHOLDERS' EQUITY |
|
|
|
|
|
|||||
|
|
|
|
|
|
|||||
CURRENT LIABILITIES: |
|
|
|
|
|
|||||
Trade payables |
|
|
|
$ |
6,329 |
|
$ |
1,164 |
|
|
Employees and payroll accruals |
|
|
|
|
4,705 |
|
|
3,443 |
|
|
Current maturities of lease liabilities |
|
|
|
|
2,532 |
|
|
1,870 |
|
|
Accrued expenses and other payables |
|
|
|
|
7,988 |
|
|
4,918 |
|
|
|
|
|
|
|
21,554 |
|
|
11,395 |
|
|
NON-CURRENT LIABILITIES: |
|
|
|
|
|
|||||
Liabilities presented at fair value |
|
|
|
|
12,043 |
|
|
5,221 |
|
|
Employee benefit liabilities, net |
|
|
|
|
768 |
|
|
773 |
|
|
Lease liability |
|
|
|
|
5,378 |
|
|
4,101 |
|
|
Liability to |
|
|
|
|
17,003 |
|
|
12,302 |
|
|
|
|
|
|
|
35,192 |
|
|
22,397 |
|
|
CONTINGENT LIABILITIES AND COMMITMENTS |
|
|
|
|
|
|||||
|
|
|
|
|
|
|||||
SHAREHOLDERS' EQUITY: |
|
|
|
|
|
|||||
Share capital |
|
|
|
|
166 |
|
|
92 |
|
|
Share premium |
|
|
|
|
375,280 |
|
|
238,992 |
|
|
Capital reserve due to actuarial loss |
|
|
|
|
(441 |
) |
|
(541 |
) |
|
Reserve from financial assets measured at FVOCI |
|
|
|
|
- |
|
|
4 |
|
|
Accumulated deficit |
|
|
|
|
(276,268 |
) |
|
(203,564 |
) |
|
Total shareholders' equity |
|
|
|
|
98,737 |
|
|
34,983 |
|
|
|
|
|
|
|
|
|||||
Total liabilities and shareholders' equity |
|
|
|
$ |
155,483 |
|
$ |
68,775 |
|
|
CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS |
||||||||||||
|
|
Year ended |
||||||||||
|
|
|
2020 |
|
|
|
2019 |
|
|
|
2018 |
|
|
|
|
|
|
|
|
||||||
Operating expenses: |
|
|
|
|
|
|
||||||
Research and development expenses, net |
|
$ |
41,385 |
|
|
$ |
31,462 |
|
|
$ |
22,045 |
|
Commercial activities |
|
|
8,748 |
|
|
|
4,692 |
|
|
|
- |
|
General and administrative expenses |
|
|
12,167 |
|
|
|
12,091 |
|
|
|
11,599 |
|
|
|
|
|
|
|
|
||||||
Operating loss |
|
|
62,300 |
|
|
|
48,245 |
|
|
|
33,644 |
|
|
|
|
|
|
|
|
||||||
Financial expenses |
|
|
10,640 |
|
|
|
3,325 |
|
|
|
20,259 |
|
Financial income |
|
|
(236 |
) |
|
|
(17,149 |
) |
|
|
(1,042 |
) |
|
|
|
|
|
|
|
||||||
Loss before taxes on income |
|
|
72,704 |
|
|
|
34,421 |
|
|
|
52,861 |
|
Taxes on income |
|
|
- |
|
|
|
(70 |
) |
|
|
70 |
|
|
|
|
|
|
|
|
||||||
Net loss |
|
|
72,704 |
|
|
|
34,351 |
|
|
|
52,931 |
|
|
|
|
|
|
|
|
||||||
|
|
|
|
|
|
|
||||||
Net loss per share: |
|
|
|
|
|
|
||||||
|
|
|
|
|
|
|
||||||
Basic net loss per share |
|
$ |
1.66 |
|
|
$ |
1.17 |
|
|
$ |
10.53 |
|
|
|
|
|
|
|
|
||||||
Diluted net loss per share |
|
$ |
1.66 |
|
|
$ |
1.69 |
|
|
$ |
10.53 |
|
CONSOLIDATED STATEMENTS OF CASH FLOWS |
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|
|
Year ended |
||||||||||
|
|
|
2020 |
|
|
|
2019 |
|
|
|
2018 |
|
Cash flows from operating activities: |
|
|
|
|
|
|
||||||
|
|
|
|
|
|
|
||||||
Net loss |
|
$ |
(72,704 |
) |
|
$ |
(34,351 |
) |
|
$ |
(52,931 |
) |
|
|
|
|
|
|
|
||||||
Adjustments to reconcile net loss to net cash used in operating activities: |
|
|
|
|
|
|
||||||
|
|
|
|
|
|
|
||||||
Adjustments to the profit or loss items: |
|
|
|
|
|
|
||||||
Depreciation of property, plant and equipment and right-of-use assets |
|
|
2,397 |
|
|
|
2,143 |
|
|
|
269 |
|
Financial loss (income), net |
|
|
483 |
|
|
|
(775 |
) |
|
|
(858 |
) |
Share-based compensation |
|
|
2,864 |
|
|
|
4,868 |
|
|
|
3,575 |
|
Change in employee benefit liabilities, net |
|
|
94 |
|
|
|
126 |
|
|
|
(15 |
) |
Amortization of premium on marketable securities |
|
|
4 |
|
|
|
184 |
|
|
|
272 |
|
Revaluation of financial derivatives |
|
|
6,822 |
|
|
|
(15,904 |
) |
|
|
17,600 |
|
Revaluation of liability to IIA |
|
|
4,302 |
|
|
|
2,531 |
|
|
|
2,037 |
|
|
|
|
|
|
|
|
||||||
|
|
|
16,966 |
|
|
|
(6,827 |
) |
|
|
22,880 |
|
Changes in asset and liability items: |
|
|
|
|
|
|
||||||
|
|
|
|
|
|
|
||||||
Increase (decrease) in other receivables, prepaid expenses and other assets |
|
|
(1,626 |
) |
|
|
(150 |
) |
|
|
942 |
|
Increase (decrease) in trade payables |
|
|
5,083 |
|
|
|
(821 |
) |
|
|
(405 |
) |
Increase in accrued expenses and other payables |
|
|
3,454 |
|
|
|
2,807 |
|
|
|
2,296 |
|
|
|
|
|
|
|
|
||||||
|
|
|
6,911 |
|
|
|
1,836 |
|
|
|
2,833 |
|
Cash received during the year for: |
|
|
|
|
|
|
||||||
|
|
|
|
|
|
|
||||||
Interest received |
|
|
361 |
|
|
|
1,546 |
|
|
|
792 |
|
Interest paid |
|
|
(161 |
) |
|
|
(134 |
) |
|
|
- |
|
|
|
|
|
|
|
|
||||||
Net cash used in operating activities |
|
|
(48,627 |
) |
|
|
(37,930 |
) |
|
|
(26,426 |
) |
|
|
|
|
|
|
|
||||||
Cash flows from investing activities: |
|
|
|
|
|
|
||||||
|
|
|
|
|
|
|
||||||
Purchase of property, plant and equipment |
|
|
(11,804 |
) |
|
|
(3,055 |
) |
|
|
(1,645 |
) |
Purchase of marketable securities |
|
|
- |
|
|
|
(32,021 |
) |
|
|
(10,905 |
) |
Proceeds from bank deposits |
|
|
- |
|
|
|
- |
|
|
|
5,000 |
|
Investment in restricted bank deposits |
|
|
(158 |
) |
|
|
- |
|
|
|
(150 |
) |
Proceeds from maturity of marketable securities |
|
|
13,551 |
|
|
|
38,742 |
|
|
|
- |
|
Proceeds from sale of marketable securities |
|
|
- |
|
|
|
- |
|
|
|
4,949 |
|
|
|
|
|
|
|
|
||||||
Net cash provided by (used in) investing activities |
|
|
1,589 |
|
|
|
3,666 |
|
|
|
(2,751 |
) |
CONSOLIDATED STATEMENTS OF CASH FLOWS |
||||||
|
|
Year ended |
||||
|
|
2020 |
|
2019 |
|
2018 |
|
|
|
|
|
|
|
Cash flows from financing activities: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Proceeds from secondary offerings, net |
|
133,316 |
|
37,140 |
|
- |
Receipt of grants from the IIA |
|
399 |
|
224 |
|
612 |
Proceeds from issuance of shares, initial public offering (payment of issuance expenses), net |
|
- |
|
(238) |
|
47,479 |
Payment of lease liabilities |
|
(1,985) |
|
(1,529) |
|
- |
Proceeds from exercise of options |
|
650 |
|
132 |
|
2 |
|
|
|
|
|
|
|
Net cash provided by financing activities |
|
132,380 |
|
35,729 |
|
48,093 |
|
|
|
|
|
|
|
Exchange differences on balances of cash and cash equivalents |
|
(10) |
|
101 |
|
31 |
|
|
|
|
|
|
|
Increase in cash and cash equivalents |
|
85,332 |
|
1,566 |
|
18,947 |
Cash and cash equivalents at beginning of year |
|
41,838 |
|
40,272 |
|
21,325 |
|
|
|
|
|
|
|
Cash and cash equivalents at end of year |
|
|
|
|
|
|
|
|
|
|
|
|
|
1 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.
2
3 Clinicaltrials.gov identifier NCT03019666
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