Gamida Cell Reports Second Quarter 2022 Financial Results and Provides Company Update
Gamida Cell Ltd. (Nasdaq: GMDA) announced the FDA's acceptance of its Biologics License Application (BLA) for omidubicel, granting Priority Review with a target action date set for January 30, 2023. The company reported a net loss of $18.6 million for Q2 2022, improving from $23.6 million in Q2 2021. Gamida Cell also initiated a Phase 1/2 study of GDA-201, targeting follicular and diffuse large B-cell lymphomas. With $55.1 million in cash, the company is funded through mid-2023, excluding commercialization costs for omidubicel.
- FDA acceptance of BLA for omidubicel with Priority Review.
- Initiated Phase 1/2 study for GDA-201, expanding treatment options.
- Reduced net loss from $23.6 million in Q2 2021 to $18.6 million in Q2 2022.
- Net loss of $18.6 million raises concerns for investors.
- Commercialization of omidubicel may require substantial additional funding.
- Received FDA acceptance of BLA for omidubicel with Priority Review; PDUFA target action date set for
- Dosed first patient in company-sponsored Phase 1/2 study of cryopreserved formulation of GDA-201 for the treatment of follicular and diffuse large B-cell lymphomas -
- Finished second quarter of 2022 with
sufficient funding for the company’s operations into mid-2023, excluding the cost of commercializing omidubicel -
- Company to host conference call at
Recently,
-
Received acceptance for filing from the
U.S. Food and Drug Administration (FDA) with priority review for its Biologics License Application (BLA) for omidubicel. The BLA has been assigned a Prescription Drug User Fee Act (PDUFA) target action date ofJanuary 30, 2023 . If approved, omidubicel will be the first allogeneic advanced stem cell therapy donor source for patients with blood cancers in need of a stem cell transplant. - Dosed the first patient in a company-sponsored Phase 1/2 study evaluating a cryopreserved formulation of GDA-201, a readily available cell therapy candidate for the treatment of follicular and diffuse large B-cell lymphomas.
-
Continued development of the company’s proprietary NAM-enabled NK cell pipeline, including genetically modified product candidates GDA-301, GDA-401, GDA-501 and GDA-601, which aim to treat solid-tumor and hematological cancers. These cell therapy candidates utilize CAR, membrane bound- and CRISPR-mediated technologies to increase the NK cell targeting, potency and persistence against hematologic malignancies and solid tumors. Promising new pre-clinical data on GDA-301 and GDA-601 were presented at the
International Society for Cell & Gene Therapy Meeting . The data demonstrated that both NAM-enabled cell therapy candidates represented a novel potent and cytotoxic approach in fighting cancer. - Advanced strategic evaluation for omidubicel commercialization, including assessing whether to commercialize omidubicel ourselves or to pursue strategic alternatives to commercialize omidubicel, upon receipt of regulatory approval. The company currently has sufficient cash to fund the company’s operations into mid-2023, excluding the cost of commercializing omidubicel.
“2022 is a potentially transformative year for
Second Quarter and Recent Developments
Omidubicel: Advanced Cell Therapy
-
BLA accepted by FDA with Priority Review: In
August 2022 , the FDA accepted for filing Gamida Cell’s BLA for omidubicel for the treatment of patients with blood cancers in need of an allogeneic hematopoietic stem cell transplant. The FDA granted Priority Review for the BLA and has set a PDUFA target action date ofJanuary 30, 2023 . In parallel,Gamida Cell is preparing for the commercialization of omidubicel in theU.S.
GDA-201: NAM-Enabled NK Cell Therapy
-
Dosed the first patient in Phase 1/2 study of cryopreserved formulation of GDA-201: In
August 2022 ,Gamida Cell completed the dosing of the first patient in a company-sponsored Phase 1/2 study evaluating a cryopreserved formulation of GDA-201 for the treatment of follicular and diffuse B-cell lymphomas.- The Phase 1 portion of the study is designed as a dose escalation phase to evaluate the safety of GDA-201, and will include patients with follicular lymphoma (FL), diffuse large B-cell lymphoma (DLBCL)/high grade B-cell lymphoma (HGBCL), marginal zone lymphoma or mantle cell lymphoma. The Phase 2 expansion phase is designed to evaluate the safety and efficacy of GDA-201 in 63 patients comprised of two patient cohorts, FL and DLBCL. The study will include patients who have relapsed or refractory lymphoma after at least two prior treatments, which may include CAR-T or stem cell transplant.
NAM-Enabled NK Cell Pipeline Expansion
-
Progressed 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 continues to conduct in vitro and in vivo preclinical proof-of-concept studies for these genetically modified NK therapeutic targets which are already showing encouraging results and plans to select the next NK pipeline 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-401: A development candidate with an undisclosed target;
- 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 in collaboration with theDana-Farber Cancer Institute to study the in vitro cytotoxicity of GDA-601 in fresh tumor tissue samples from multiple myeloma patients.
Corporate Updates
-
Appointed
Ivan M. Borrello , M.D. to Board of Directors:Dr. Borrello is an Associate Professor of Oncology at theSydney Kimmel Comprehensive Cancer Center atJohns Hopkins and a renowned physician and author who has made major contributions to better the understanding of immunotherapies and the treatment of hematologic malignancies as well as bone marrow transplant. The Company also announced the resignation ofOfer Gonen from its Board of Directors.
Second Quarter 2022 Financial Results
-
Research and development expenses were
in the second quarter of 2022, compared to$10.6 million in the same quarter in 2021. The decrease was attributable mainly to a$13.4 million decrease in clinical activities relating to the conclusion of our Phase 3 clinical trial and a decrease of$2.4 million in the GDA-201 clinical program.$0.4 million -
Commercial expenses were
in the second quarter of 2022, compared to$3.2 million in the second quarter of 2021. The decrease was attributable mainly to reducing near-term commercial readiness expenses, as we continued to assess strategic approaches for the commercialization of omidubicel.$5.0 million -
General and administrative expenses were
in the second quarter of 2022, compared to$4.3 million in the same period in 2021. The increase was mainly due to a$3.9 million increase in professional services expenses, offset by a decrease of$0.9 million in headcount related expenses.$0.5 million -
Finance expenses, net, were
in the second quarter of 2022, compared to$0.5 million in the same period in 2021. The decrease was primarily due to$1.3 million in non-cash expenses and an increase of$0.6 million in interest income from cash management.$0.2 million -
Net loss was
in the second quarter of 2022, compared to a net loss of$18.6 million in the second quarter of 2021.$23.6 million
2022 Financial Guidance
Expected Milestones in 2022 and Early 2023
Omidubicel
-
PDUFA target action date of
January 30, 2023 .
NK cell pipeline expansion
- Conduct 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 candidate developed as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant for patients with blood cancers. Omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment in comparison to standard umbilical cord blood in an international, multi-center, randomized Phase 3 study (NCT0273029) in patients with hematologic malignancies undergoing allogeneic bone marrow transplant. The Phase 3 study also showed reduced time to platelet engraftment, reduced infections and fewer days of hospitalization. One-year post-transplant data showed sustained clinical benefits with omidubicel as demonstrated by significant reduction in infectious complications as well as reduced non-relapse mortality and no significant increase in relapse rates nor increases in graft-versus-host-disease (GvHD) rates. Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the FDA and has also received Orphan Drug Designation in the US and EU.
Omidubicel is an investigational stem cell therapy candidate, and its safety and efficacy have not been established by the FDA or any other health authority. For more information about omidubicel, please visit https://www.gamida-cell.com.
About GDA-201
For more information about GDA-201, please visit https://www.gamida-cell.com. For more information on the Phase 1/2 clinical trial of GDA-201, please visit www.clinicaltrials.gov.
GDA-201 is an investigational cell therapy candidate, and its safety and efficacy have not been established by the FDA or any other health authority.
About NAM Technology
Our NAM-enabling technology, supported by positive omidubicel Phase 3 results, is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we can expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.
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, pre-clinical and clinical trials of Gamida Cell’s product candidates (including omidubicel and GDA-201), anticipated regulatory filings (including the timing of review of the BLA for omidubicel by the FDA), commercialization planning efforts, the potentially life-saving or curative therapeutic and commercial potential of Gamida Cell’s product candidates (including GDA-201 and omidubicel), Gamida Cell’s expectations for the clinical development milestones set forth herein, and Gamida Cell’s expectations regarding its projected cash, cash equivalents and investments to be used for operating activities. 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, including the scope, progress and expansion of Gamida Cell’s clinical trials and ramifications for the cost thereof; clinical, scientific, regulatory and technical developments; the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics; and 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 Quarterly Report on Form 10-Q, filed with the
CONDENSED CONSOLIDATED BALANCE SHEETS | |||||||||
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|
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|
2022 |
|
|
2021 |
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ASSETS |
|
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|
|
|
|
|||
|
|
|
|
|
|
|
|||
CURRENT ASSETS: |
|
|
|
|
|
|
|||
Cash and cash equivalents |
|
$ |
37,890 |
|
|
$ |
55,892 |
|
|
Marketable securities |
|
|
17,172 |
|
|
|
40,034 |
|
|
Prepaid expenses and other current assets |
|
|
2,294 |
|
|
|
2,688 |
|
|
|
|
|
|
|
|
|
|
|
|
Total current assets |
|
|
57,356 |
|
|
|
98,614 |
|
|
|
|
|
|
|
|
|
|
|
|
NON-CURRENT ASSETS: |
|
|
|
|
|
|
|
|
|
Restricted deposits |
|
|
3,591 |
|
|
|
3,961 |
|
|
Property, plant and equipment, net |
|
|
37,967 |
|
|
|
35,180 |
|
|
Operating lease right-of-use assets |
|
|
6,107 |
|
|
|
7,236 |
|
|
Severance pay fund |
|
|
1,579 |
|
|
|
2,148 |
|
|
Other long-term assets |
|
|
1,421 |
|
|
|
1,647 |
|
|
|
|
|
|
|
|
|
|
|
|
Total non-current assets |
|
|
50,665 |
|
|
|
50,172 |
|
|
|
|
|
|
|
|
|
|
|
|
Total assets |
|
$ |
108,021 |
|
|
$ |
148,786 |
|
|
LIABILITIES AND EQUITY |
|
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||
|
|
|
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CURRENT LIABILITIES: |
|
|
|
|
|
|
||
Trade payables |
|
$ |
2,738 |
|
|
$ |
8,272 |
|
Employees and payroll accruals |
|
|
4,978 |
|
|
|
4,957 |
|
Operating lease liabilities |
|
|
2,517 |
|
|
|
2,699 |
|
Accrued interest of convertible senior notes |
|
|
1,652 |
|
|
|
1,640 |
|
Accrued expenses and other current liabilities |
|
|
10,412 |
|
|
|
7,865 |
|
|
|
|
|
|
|
|
|
|
Total current liabilities |
|
|
22,297 |
|
|
|
25,433 |
|
|
|
|
|
|
|
|
|
|
NON-CURRENT LIABILITIES: |
|
|
|
|
|
|
|
|
Convertible senior notes, net |
|
|
71,801 |
|
|
|
71,417 |
|
Accrued severance pay |
|
|
1,840 |
|
|
|
2,396 |
|
Long-term operating lease liabilities |
|
|
4,233 |
|
|
|
5,603 |
|
|
|
|
|
|
|
|
|
|
Total non-current liabilities |
|
|
77,874 |
|
|
|
79,416 |
|
|
|
|
|
|
|
|
|
|
CONTINGENT LIABILITIES AND COMMITMENTS |
|
|
|
|
|
|
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SHAREHOLDERS’ EQUITY: |
|
|
|
|
|
|
|
|
Share capital - |
|
|
169 |
|
|
|
169 |
|
Additional paid-in capital |
|
|
383,915 |
|
|
|
381,225 |
|
Accumulated deficit |
|
|
(376,234 |
) |
|
|
(337,457 |
) |
|
|
|
|
|
|
|
|
|
Total shareholders’ equity |
|
|
7,850 |
|
|
|
43,937 |
|
|
|
|
|
|
|
|
|
|
Total liabilities and shareholders’ equity |
|
$ |
108,021 |
|
|
$ |
148,786 |
|
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS | ||||||||||||||||
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||||||||||||||||
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Three months ended
|
|
|
Six months ended
|
|
||||||||||
|
|
2022 |
|
|
2021 |
|
|
2022 |
|
|
2021 |
|
||||
|
|
Unaudited |
|
|||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
||||
Research and development expenses, net |
|
$ |
10,563 |
|
|
$ |
13,350 |
|
|
$ |
21,868 |
|
|
$ |
24,710 |
|
Commercial expenses |
|
|
3,193 |
|
|
|
4,988 |
|
|
|
7,072 |
|
|
|
9,219 |
|
General and administrative expenses |
|
|
4,290 |
|
|
|
3,874 |
|
|
|
8,429 |
|
|
|
7,387 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total operating loss |
|
|
18,046 |
|
|
|
22,212 |
|
|
|
37,369 |
|
|
|
41,316 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Financial expenses, net |
|
|
508 |
|
|
|
1,345 |
|
|
|
1,408 |
|
|
|
1,427 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Loss |
|
$ |
18,554 |
|
|
$ |
23,557 |
|
|
$ |
38,777 |
|
|
$ |
42,743 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share, basic and diluted |
|
|
0.31 |
|
|
|
0.40 |
|
|
|
0.65 |
|
|
|
0.72 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted average number of shares |
|
|
59,546,273 |
|
|
|
59,253,315 |
|
|
|
59,510,918 |
|
|
|
59,188,504 |
|
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS | |||||||||
|
|||||||||
|
|
|
Six months ended
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|
|||||
|
|
|
2022 |
|
|
2021 |
|
||
Cash flows from operating activities: |
|
|
|
|
|
|
|
||
Loss |
|
|
$ |
(38,777 |
) |
|
$ |
(42,743 |
) |
Adjustments to reconcile loss to net cash used in operating activities: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Depreciation of property, plant and equipment |
|
|
|
224 |
|
|
|
206 |
|
Financing expense (income), net |
|
|
|
(273 |
) |
|
|
1,694 |
|
Share-based compensation |
|
|
|
2,530 |
|
|
|
2,025 |
|
Amortization of issuance costs |
|
|
|
385 |
|
|
|
269 |
|
Operating lease right-of-use assets |
|
|
|
1,226 |
|
|
|
1,032 |
|
Operating lease liabilities |
|
|
|
(1,649 |
) |
|
|
(1,187 |
) |
Accrued severance pay, net |
|
|
|
14 |
|
|
|
- |
|
Increase in prepaid expenses and other assets |
|
|
|
(19 |
) |
|
|
(358 |
) |
Decrease in trade payables |
|
|
|
(5,535 |
) |
|
|
(884 |
) |
Increase (decrease) in accrued expenses and current liabilities |
|
|
|
2,285 |
|
|
|
(622 |
) |
|
|
|
|
|
|
|
|
|
|
Net cash used in operating activities |
|
|
|
(39,589 |
) |
|
|
(40,568 |
) |
|
|
|
|
|
|
|
|
|
|
Cash flows from investing activities: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Purchase of property, plant and equipment |
|
|
|
(1,540 |
) |
|
|
(6,118 |
) |
Purchase of marketable securities |
|
|
|
(3,708 |
) |
|
|
(68,151 |
) |
Proceeds from maturity of marketable securities |
|
|
|
26,175 |
|
|
|
17,824 |
|
Proceeds (investments) from restricted deposits |
|
|
|
500 |
|
|
|
(1,000 |
) |
|
|
|
|
|
|
|
|
|
|
Net cash provided by (used in) investing activities |
|
|
$ |
21,427 |
|
|
$ |
(57,445 |
) |
|
|
|
|
|
|
|
|
||
Cash flows from financing activities: |
|
|
|
|
|
|
|
||
|
|
|
|
|
|
|
|
||
Proceeds from exercise of options |
|
|
$ |
76 |
|
|
$ |
556 |
|
Proceeds from share issuance, net |
|
|
|
84 |
|
|
|
- |
|
Proceeds from issuance of convertible senior notes, net |
|
|
|
- |
|
|
|
70,777 |
|
|
|
|
|
|
|
|
|
|
|
Net cash provided by financing activities |
|
|
|
160 |
|
|
|
71,333 |
|
|
|
|
|
|
|
|
|
|
|
Decrease in cash and cash equivalents |
|
|
|
(18,002 |
) |
|
|
(26,680 |
) |
Cash and cash equivalents at beginning of period |
|
|
|
55,892 |
|
|
|
127,170 |
|
|
|
|
|
|
|
|
|
|
|
Cash and cash equivalents at end of period |
|
|
$ |
37,890 |
|
|
$ |
100,490 |
|
|
|
|
|
|
|
|
|
|
|
Significant non-cash transactions: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Purchase of property, plant and equipment on credit |
|
|
|
282 |
|
|
|
1,563 |
|
|
|
|
|
|
|
|
|
|
|
Supplemental disclosures of cash flow information: |
|
|
|
|
|
|
|
|
|
Cash paid for interest |
|
|
$ |
(2,203 |
) |
|
$ |
- |
|
View source version on businesswire.com: https://www.businesswire.com/news/home/20220815005184/en/
For investors:
Stern Investor Relations, Inc.
Courtney.Turiano@sternir.com
1-212-362-1200
For media:
Chief of Staff
Heather@gamida-cell.com
1-617-892-9083
Source:
FAQ
What is the significance of the FDA's acceptance of Gamida Cell's BLA for omidubicel?
When is the PDUFA target action date for omidubicel?
What were Gamida Cell's financial results for Q2 2022?
What is GDA-201 and its current status?