Gamida Cell Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Company Update
Gamida Cell Ltd. (Nasdaq: GMDA) provided a business update on March 15, 2022, highlighting a rolling BLA submission for omidubicel, anticipated to be completed in H1 2022. The company reported a net loss of $89.8 million for 2021, compared to $61.6 million in 2020, with cash reserves of approximately $96 million to fund operations into mid-2023. Gamida Cell is addressing an FDA clinical hold on its GDA-201 IND and advancing its NK cell pipeline. Expected milestones for 2022 include the completion of the BLA and initiation of a Phase 1/2 study for GDA-201.
- Initiated rolling BLA submission for omidubicel, expected to complete by H1 2022.
- Cash and cash equivalents of approximately $96 million, sufficient to fund operations into mid-2023.
- Expected 10-year overall survival of 48% for omidubicel-treated patients with hematologic malignancies.
- Net loss increased to $89.8 million in 2021 from $61.6 million in 2020.
- Increased expenses: R&D expenses rose to $50.2 million; commercial expenses increased to $20 million in 2021.
- Initiated rolling BLA submission for omidubicel in the first quarter of 2022 with full BLA submission on track for first half of 2022 -
- Finished fourth quarter of 2021 with approximately
- Company to host conference call at
During the past quarter and into 2022,
-
Continued to advance omidubicel, a potentially life-saving cell therapy treatment for patients with blood cancers in need of stem cell transplant. In the first quarter of 2022,
Gamida Cell initiated a rolling Biologics License Application (BLA) submission for omidubicel following the receipt of positive Type B meeting correspondence from theU.S. Food and Drug Administration (FDA). - Progressed activities to address the FDA’s clinical hold on the Investigational New Drug (IND) application for GDA-201, which was imposed based on FDA questions about donor eligibility procedures and assay qualification prior to the initiation of the study in patients with follicular and diffuse large B-cell lymphomas.
- Advanced the company’s NAM-enabled natural killer (NK) cell pipeline, including targets GDA-301, GDA-501 and GDA-601, which focus on solid-tumor and hematological cancers. These targets utilize CAR, membrane bound- and CRISPR-mediated technologies to increase targeting, potency and persistence against hematologic malignancies and solid tumors.
“Throughout 2021,
Fourth Quarter and Recent Developments
Omidubicel: Advanced Cell Therapy
-
BLA submission: Following the receipt of positive Type B meeting correspondence from the FDA confirming that analytical comparability has been established between Gamida Cell’s wholly-owned commercial manufacturing facility and the product that was manufactured for the Phase 3 study,
Gamida Cell initiated a rolling BLA submission for omidubicel in the first quarter of 2022. As part of the rolling BLA,Gamida Cell submitted the nonclinical and clinical modules of the omidubicel BLA and is on-track to complete submission of all remaining modules of the BLA by the first half of 2022. In parallel with the BLA submission, the company is assessing alternatives for the commercialization of omidubicel, including potentialU.S. or global partnerships. -
New data presented at ASH: At the 63rd
American Society of Hematology (ASH) Annual Meeting and Exposition inDecember 2021 ,Gamida Cell presented clinical updates and a health economic analysis on hospital resource utilization.- Data collected from a subset of patients in the omidubicel Phase 3 trial showed that, in addition to more rapid short-term hematopoietic recovery, omidubicel-treated patients had more rapid recovery of a wide variety of immune cells including CD4+ T cells, B cells, monocytes, NK cells, and dendritic cells than the control arm. The robust recovery of a broad range of the immune system correlated with and supported clinical data showing fewer severe bacterial, fungal, and viral infections in patients treated with omidubicel.
- Resource utilization data during the first 100 days after transplant showed that omidubicel-treated patients had significantly shorter durations of hospitalization, intensive care unit time, consultant visits, procedures, and transfusions than the control arm. These data provide further evidence of the clinical benefit associated with the more rapid hematopoietic recovery in patients treated with omidubicel and the corresponding reduction in healthcare resource utilization.
-
An analysis of outcomes of patients with hematologic malignancies treated with omidubicel over a 10-year period showed long-term sustained bone marrow function and immune recovery, with a 10-year overall survival of
48% . These data provide further support for the long-term clinical benefit of omidubicel with long-lasting hematopoietic recovery.
GDA-201: NAM-Enabled NK Cell Therapy
-
IND for Phase 1/2 Study:
Gamida Cell is working diligently to address the clinical hold on the IND for a Phase 1/2 study of GDA-201.Gamida Cell expects to initiate a company-sponsored Phase 1/2 clinical study in patients with follicular and diffuse large B-cell lymphomas in 2022. -
New data presented at ASH:
Gamida Cell presented 2-year survival and correlation data with cytokine IL7 at the ASH Annual Meeting and Exposition inDecember 2021 . This analysis provided longer follow-up in the investigator-led study of GDA-201 in patients with non-Hodgkin lymphoma and demonstrated an overall survival rate of78% at two years with a median duration of response of 16 months.
NAM-Enabled NK Cell Pipeline Expansion
-
Advanced NAM-enabled genetically modified NK pipeline:
Gamida Cell continues to progress its NAM-enabled genetically modified NK pipeline, which utilizes CAR, membrane bound- and CRISPR-mediated technologies to increase targeting, potency and persistence against hematologic malignancies and solid tumors. The company plans to execute preclinical proof of concept studies for these genetically modified NK therapeutic targets and to select a product candidate for IND enabling studies by the end of 2022. These therapeutic targets include:- GDA-301: Knockout of CISH (cytokine inducible SH2 containing protein) in NK cells using CRISPR/Cas9 in combination with a membrane-bound IL-15/IL-15Ra;
- GDA-501: anti HER2 CAR-engineered NK cells to target solid tumors expressing HER2, based on a single-chain variable fragment of the widely used humanized monoclonal antibody trastuzumab; and
-
GDA-601: CRISPR Knockout of CD38 on NK cells combined with anti CD38 CAR. CD38 is an established immunotherapeutic target in multiple myeloma, but its expression on NK cells and its further induction during ex vivo NK cell expansion represents a barrier to the development of an anti CD38 CAR-NK cell therapy.
Gamida Cell is advancing this program with a collaboration with theDana-Farber Cancer Institute to study the in vitro cytotoxicity of GDA-601 in fresh samples from multiple myeloma patients. - GDA-401: A development candidate with the target still undisclosed.
Full Year 2021 Financial Results
-
Research and development expenses were
in 2021, compared to$50.2 million in 2020. The increase was primarily due to a$38.9 million increase in omidubicel commercial manufacturing readiness activities and advancing the NK programs, as well as an increase of$5.4 million in broadening the company scientific capabilities and talent.$5.9 million -
Commercial expenses in 2021 were
, compared to$20.0 million in 2020. The increase was attributable mainly to a$8.9 million increase in commercial readiness expenses and a$6.5 million increase in headcount within the commercial organization. Going forward, the company anticipates reducing its near-term commercial readiness expenses, as it is assessing alternatives for the commercialization of omidubicel, including potential$4.6 million U.S. or global partnerships. -
General and administrative expenses were
in 2021, compared to$17.0 million in 2020. The increase was mainly due to a$13.2 million increase in professional services expenses and a$2.6 million increase in headcount and related expenses.$1.2 million -
Finance expenses, net, were
for 2021, compared to$2.6 million for 2020. The increase was primarily due to a$0.6 million interest expenses from convertible notes, offset by a$4.4 million capitalization and other non-cash expenses, and a$2.1 million increase in interest income from cash management.$0.3 million -
Net loss for 2021 was
, compared to a net loss of$89.8 million in 2020.$61.6 million
2022 Financial Guidance
Expected Milestones in 2022
Omidubicel
- Completion of full BLA submission to the FDA in the first half of 2022
GDA-201
- Initiation of a company-sponsored Phase 1/2 clinical study in follicular and diffuse large B-cell lymphomas
NK cell pipeline expansion
- Establish preclinical proof of concept studies of the NAM-enabled, genetically modified NK therapeutic targets
- Select pipeline candidate for IND-enabling studies
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 for patients with blood cancers. Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the
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 (including GDA-201), anticipated regulatory filings (including the timing of submission of the BLA for omidubicel to the FDA), commercialization planning efforts, and the potentially life-saving or curative therapeutic and commercial potential of omidubicel, and Gamida Cell’s expectations regarding its projected cash to be used for operating activities and cash runway. Any statement describing Gamida Cell’s goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to a number of risks, uncertainties and assumptions, including those related to the impact that the COVID-19 pandemic could have on our business, and including the scope, progress and expansion of Gamida Cell’s clinical trials and ramifications for the cost thereof; clinical, scientific, regulatory and technical developments; and those inherent in the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such product candidates. 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 BALANCE SHEETS |
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2021 |
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2020 |
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ASSETS |
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||
|
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|
|
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||
CURRENT ASSETS: |
|
|
|
|
||
Cash and cash equivalents |
|
$ |
55,892 |
|
$ |
127,170 |
Marketable securities |
|
|
40,034 |
|
|
- |
Prepaid expenses and other current assets |
|
|
2,688 |
|
|
3,087 |
|
|
|
|
|
||
Total current assets |
|
|
98,614 |
|
|
130,257 |
|
|
|
|
|
||
NON-CURRENT ASSETS: |
|
|
|
|
||
Restricted deposits |
|
|
3,961 |
|
|
- |
Property, plant and equipment, net |
|
|
35,180 |
|
|
18,238 |
Operating lease right-of-use assets |
|
|
7,236 |
|
|
6,841 |
Severance pay fund |
|
|
2,148 |
|
|
2,191 |
Other long-term assets |
|
|
1,647 |
|
|
786 |
|
|
|
|
|
||
Total non-current assets |
|
|
50,172 |
|
|
28,056 |
|
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|
|
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||
Total assets |
|
$ |
148,786 |
|
$ |
158,313 |
CONSOLIDATED BALANCE SHEETS |
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2021 |
|
2020 |
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LIABILITIES AND SHAREHOLDERS’ EQUITY |
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CURRENT LIABILITIES: |
|
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|
||||
Trade payables |
|
$ |
8,272 |
|
|
$ |
6,331 |
|
Employees and payroll accruals |
|
|
4,957 |
|
|
|
4,705 |
|
Operating lease liabilities |
|
|
2,699 |
|
|
|
2,475 |
|
Accrued interest of convertible senior notes |
|
|
1,640 |
|
|
|
- |
|
Accrued expenses and current liabilities |
|
|
7,865 |
|
|
|
7,988 |
|
|
|
|
|
|
||||
|
|
|
25,433 |
|
|
|
21,499 |
|
|
|
|
|
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||||
NON-CURRENT LIABILITIES: |
|
|
|
|
||||
Convertible senior notes, net |
|
|
71,417 |
|
|
|
- |
|
Accrued severance pay |
|
|
2,396 |
|
|
|
2,426 |
|
Long-term operating lease liabilities |
|
|
5,603 |
|
|
|
5,517 |
|
|
|
|
|
|
||||
Total non-current liabilities |
|
|
79,416 |
|
|
|
7,943 |
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CONTINGENT LIABILITIES AND COMMITMENTS |
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SHAREHOLDERS’ EQUITY: |
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Ordinary shares of
Authorized: 150,000,000 shares at |
|
|
169 |
|
|
|
166 |
|
Additional paid-in capital |
|
|
381,225 |
|
|
|
376,369 |
|
Accumulated deficit |
|
|
(337,457 |
) |
|
|
(247,664 |
) |
Total shareholders’ equity |
|
|
|
|
||||
|
|
|
43,937 |
|
|
|
128,871 |
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Total liabilities and shareholders’ equity |
|
|
|
|
||||
|
|
$ |
148,786 |
|
|
$ |
158,313 |
|
CONSOLIDATED STATEMENTS OF OPERATIONS |
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Year ended
|
||||
|
|
2021 |
|
2020 |
||
|
|
|
|
|
||
Research and development expenses, net |
|
$ |
50,177 |
|
$ |
38,873 |
Commercial expenses |
|
|
20,013 |
|
|
8,894 |
General and administrative expenses |
|
|
16,977 |
|
|
13,158 |
|
|
|
|
|
||
Total operating loss |
|
|
87,167 |
|
|
60,925 |
|
|
|
|
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Financial expenses, net |
|
|
2,626 |
|
|
648 |
|
|
|
|
|
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Loss |
|
|
89,793 |
|
|
61,573 |
|
|
|
|
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Net loss per share attributable to ordinary shareholders, basic and diluted |
|
$ |
1.52 |
|
$ |
1.41 |
|
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|
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Weighted average number of shares used in computing net loss per share attributable to ordinary shareholders, basic and diluted |
|
|
59,246,803 |
|
|
43,725,584 |
CONSOLIDATED STATEMENTS OF CASH FLOWS |
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Year ended
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2021 |
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2020 |
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Cash flows from operating activities: |
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|
|
|
|
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Loss |
|
$ |
(89,793 |
) |
|
$ |
(61,573 |
) |
|
|
|
|
|
||||
Adjustments to reconcile loss to net cash used in operating activities: |
|
|
|
|
||||
|
|
|
|
|
||||
Depreciation of property, plant and equipment |
|
|
431 |
|
|
|
357 |
|
Financing expense, net |
|
|
359 |
|
|
|
166 |
|
Share-based compensation |
|
|
4,233 |
|
|
|
3,368 |
|
Amortization of debt discount and issuance costs |
|
|
638 |
|
|
|
- |
|
Operating lease right-of-use assets |
|
|
2,109 |
|
|
|
1,891 |
|
Operating lease liabilities |
|
|
(2,193 |
) |
|
|
(1,318 |
) |
Accrued severance pay, net |
|
|
12 |
|
|
|
- |
|
Decrease (increase) in prepaid expenses and other assets |
|
|
1,008 |
|
|
|
(1,630 |
) |
Increase in trade payables |
|
|
1,941 |
|
|
|
5,066 |
|
Increase (decrease) in accrued expenses and current liabilities |
|
|
(505 |
) |
|
|
3,454 |
|
|
|
|
|
|
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Net cash used in operating activities |
|
|
(81,760 |
) |
|
|
(50,219 |
) |
|
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Cash flows from investing activities: |
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|
|
|
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|
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|
|
|
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Purchase of property, plant and equipment |
|
|
(15,054 |
) |
|
|
(11,804 |
) |
Purchase of marketable securities |
|
|
(102,179 |
) |
|
|
- |
|
Proceeds from maturity of marketable securities |
|
|
61,534 |
|
|
|
13,551 |
|
Investment in restricted deposits |
|
|
(5,222 |
) |
|
|
(158 |
) |
|
|
|
|
|
||||
Net cash provided by (used in) investing activities |
|
|
(60,921 |
) |
|
|
1,589 |
|
|
|
|
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|
|
|
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Cash flows from financing activities: |
|
|
||||||
|
|
|
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|
||||
Proceeds from issuance of ordinary shares, net |
|
|
- |
|
|
|
133,312 |
|
Proceeds from exercise of options |
|
|
626 |
|
|
|
650 |
|
Proceeds from issuance of convertible senior notes, net |
|
|
70,777 |
|
|
|
- |
|
|
|
|
|
|
||||
Net cash provided by financing activities |
|
|
71,403 |
|
|
|
133,962 |
|
|
|
|
|
|
||||
Increase (decrease) in cash and cash equivalents |
|
|
(71,278 |
) |
|
|
85,332 |
|
Cash and cash equivalents at beginning of year |
|
|
127,170 |
|
|
|
41,838 |
|
|
|
|
|
|
||||
Cash and cash equivalents at end of year |
|
$ |
55,892 |
|
|
$ |
127,170 |
|
View source version on businesswire.com: https://www.businesswire.com/news/home/20220315005423/en/
For investors:
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Courtney.Turiano@sternir.com
1-212-362-1200
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1-978-417-1946
Source:
FAQ
What are the financial results of Gamida Cell for 2021?
When is the full BLA submission for omidubicel expected?
What is the cash runway for Gamida Cell?
What are the key milestones for Gamida Cell in 2022?