Gamida Cell Reports First Quarter 2021 Financial Results and Provides Company Update
- BLA submission for omidubicel, a potentially life-saving treatment for patients with blood cancers in need of stem cell transplant, expected in fourth quarter of 2021
- Pre-commercial and manufacturing activities underway to support potential launch of omidubicel in 2022
- Phase 1/2 clinical trial of allogeneic, off-the-shelf GDA-201 in NHL planned with IND submission anticipated in the second half of 2021
-
Strengthened financial position with sale of
$75M exchangeable senior notes inFebruary 2021 ; sufficient liquidity to fund the company’s operations into the second half of 2022 -
Company to host conference call at
8:00 a.m. ET today
“In the first quarter of this year, we made significant progress on key initiatives across all functions of our business, starting with omidubicel, a potentially transformative treatment option for patients with hematological malignancies,” said
“We also continue to expand our clinical pipeline with plans to submit an IND for our GDA-201 natural killer cell therapy, initiate a multi-center Phase 1/2 clinical study in NHL and continue to advance our R&D activities to pursue the development of genetically modified NAM-enabled NK cells in solid tumors. Importantly, we are well positioned to deliver our 2021 corporate goals and objectives toward improving the lives of the patients we serve,”
Omidubicel, a proprietary, investigational advanced cell therapy for allogeneic bone marrow transplant
Omidubicel is the foundational product based on
In February 2021, the company presented details of the results of the omidubicel Phase 3 study at the Transplantation & Cellular Therapy Meetings of the
In addition to the efficacy results described above, safety results were also presented, showing decreased incidence related to grade III/IV acute GvHD (14 percent for omidubicel, 21 percent for the comparator) and comparable results for all grades chronic GvHD at one year (35 percent for omidubicel, 29 percent for the comparator). Transplants with umbilical cord blood, the comparator, have been historically shown to result in low incidence of GvHD in relation to other graft sources and, in this study, omidubicel demonstrated a similar GvHD profile.
The data from the study relating to exploratory endpoints also supported the clinical benefit demonstrated by the study’s primary and secondary endpoints. The rate of infection was significantly reduced for patients randomized to omidubicel, with the cumulative incidence of first grade II or grade III bacterial or invasive fungal infection for patients randomized to omidubicel of 37 percent, compared to 57 percent for the comparator (p = 0.027). Additionally, the study demonstrated a reduction in the incidence of viral infections. Non-relapse mortality was 11 percent for patients randomized to omidubicel and 24 percent for patients randomized to the comparator (p=0.09). Overall survival at 15 months following randomization was 73 percent for patients randomized to omidubicel and 62 percent for patients randomized to control (p=0.16), median overall survival was not yet reached. Non-relapse mortality and overall survival were exploratory endpoints that were not powered for statistical significance. When considering the patient experience following transplant, faster hematopoietic recovery, fewer bacterial and viral infections and fewer days in hospital are all meaningful results and represent potentially important advancements in care. Learn more.
Additional omidubicel highlights:
-
Progress with commercial manufacturing readiness:
Gamida Cell is making important progress to address the clear feedback received during a Type B meeting with the FDA inDecember 2020 for commercial manufacturing facilities to be ready for BLA submission. These facilities include theGamida Cell facility inIsrael and a commercial facility for which the company has a contractual relationship with Lonza. Both of these facilities are currently on track to meet the FDA requirements that will be required for BLA submission. - Continued launch readiness: The company continues to progress commercial launch readiness activities for the potential launch of omidubicel in 2022, pending FDA approval. Based on market research insights, there is a clear opportunity to improve outcomes based on clinical needs with current donor sources, increase access for patients who are eligible and not matched for transplant, and increase patient eligibility based on the encouraging clinical profile of omidubicel.
-
Gamida Cell announced the Gamida Cell Assist program. The transplant process can be challenging and complex for patients, caregivers and the entire transplant care team. Gamida Cell Assist is designed to focus on patient access and support 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, 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. Learn more. -
Phase 1/2 study of omidubicel in patients with severe aplastic anemia:
Gamida Cell is actively evaluating omidubicel in an investigator-sponsored Phase 1/2 study in patients with severe aplastic anemia (SAA). Results to date have shown 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.
GDA-201, a proprietary innate NK cell immunotherapy
-
Continued advancement of Phase 1/2 study of GDA-201:
Gamida Cell is preparing for 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 this year.Gamida Cell is pioneering a potentially curative, novel approach that harnesses the power of its cell expansion technology, which improves antibody-dependent cellular cytotoxicity and tumor targeting of NK cells. Learn more. - Advancing NK cell R&D activities: The company continues to advance R&D activities to support pipeline growth, including the development of genetically modified NK cells.
Corporate Highlights
-
Strengthened financial position: In
February 2021 , the company completed a$75 million financing withHighbridge Capital Management, LLC , before deducting offering expenses. This financing 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.
First Quarter 2021 Financial Results
-
Research and development expenses in the first quarter of 2021 were
$11.4 million , compared to$7.9 million for the same period in 2020. The increase was mainly due to omidubicel commercial manufacturing readiness activities and advancing the GDA-201 program, including broadening the company’s scientific capabilities and talent.
-
Commercial expenses in the first quarter of 2021 were
$4.4 million compared to$1.5 million for the first quarter of 2020. The increase was mainly attributed to progress with commercial readiness activities, including the hiring of an experienced commercial leadership team.
-
General and administrative expenses were
$3.4 million for the first quarter of 2021 compared to$3.0 million for the same period in 2020. The increase was mainly due to the hiring of key management positions to support the growth of the business.
-
Finance income, net, was
$0.7 million for the first quarter of 2021, compared to finance income, net, of$1.7 million for the first quarter of 2020. The decrease was primarily due to interest expenses following the recent$75M financing withHighbridge Capital Management , and non-cash expense resulting from revaluation of warrants, andIsraeli Innovation Authority royalty-bearing grant liability.
-
Net loss for the first quarter of 2021 was
$18.0 million , compared to a net loss of$10.6 million for the same period in 2020.
-
As of
March 31, 2021 ,Gamida Cell had total cash and cash equivalents of$174.8 million , compared to$127.2 million as ofDecember 31, 2020 .
2021 Financial Guidance
Expected 2021-2022 Milestones and Key Events
Omidubicel
- BLA submission to the FDA in the fourth quarter of 2021
- Manufacturing and launch readiness activities ongoing for potential FDA approval in 2022
GDA-201
- Submit company-sponsored IND application to the FDA and initiate a Phase 1/2 clinical study in NHL patients 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
___________________ |
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 |
INTERIM CONSOLIDATED STATEMENTS OF FINANCIAL POSITION | |||||||||
|
2021 |
2020 |
2020 |
||||||
Unaudited | Audited | ||||||||
ASSETS | |||||||||
CURRENT ASSETS: | |||||||||
Cash and cash equivalents |
$ |
174,798 |
$ |
40,292 |
$ |
127,170 |
|||
Prepaid expenses and other current assets |
|
3,352 |
|
1,637 |
|
2,815 |
|||
Total current assets |
|
178,150 |
|
41,929 |
|
129,985 |
|||
NON-CURRENT ASSETS: | |||||||||
Property, plant and equipment, net |
|
21,162 |
|
8,543 |
|
18,238 |
|||
Right-of-use assets |
|
5,920 |
|
5,820 |
|
6,474 |
|||
Other assets |
|
772 |
|
637 |
|
786 |
|||
Total non-current assets |
|
28,854 |
|
15,000 |
|
25,498 |
|||
Total assets |
$ |
206,004 |
$ |
56,929 |
$ |
155,483 |
|||
2021 |
|
2020 |
|
2020 |
|||||
Unaudited | Audited | ||||||||
LIABILITIES AND EQUITY | |||||||||
CURRENT LIABILITIES: | |||||||||
Trade payables |
$ |
7,204 |
$ |
3,098 |
$ |
6,329 |
|||
Employees and payroll accruals |
|
3,854 |
|
2,549 |
|
4,705 |
|||
Current maturities of lease liabilities |
|
2,076 |
|
1,535 |
|
2,532 |
|||
Accrued interest |
|
634 |
|
- |
|
- |
|||
Accrued expenses and other payables |
|
6,099 |
|
2,922 |
|
7,988 |
|||
Total current liabilities |
|
19,867 |
|
10,104 |
|
21,554 |
|||
NON-CURRENT LIABILITIES: | |||||||||
Liabilities presented at fair value |
|
9,758 |
|
2,773 |
|
12,043 |
|||
Employee benefit liabilities, net |
|
768 |
|
773 |
|
768 |
|||
Other long-term liabilities |
|
4,988 |
|
4,920 |
|
5,378 |
|||
Liability to |
|
18,080 |
|
13,077 |
|
17,003 |
|||
Convertible senior notes, net |
|
68,646 |
|
- |
|
- |
|||
Total non-current liabilities |
|
102,240 |
|
21,543 |
|
35,192 |
|||
SHAREHOLDERS' EQUITY: | |||||||||
Share capital - | |||||||||
Ordinary shares of |
|
167 |
92 |
|
166 |
||||
Authorized: 100,000,000 shares at |
|||||||||
Share premium |
|
378,478 |
239,897 |
|
375,280 |
||||
Capital reserve |
|
-441 |
-541 |
|
-441 |
||||
Accumulated deficit |
|
-294,307 |
-214,166 |
|
-276,268 |
||||
Total shareholders' equity |
|
83,897 |
25,282 |
|
98,737 |
||||
Total liabilities and shareholders' equity |
$ |
206,004 |
|
$ |
155,483 |
||||
INTERIM CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS | ||||||
Three months ended |
Year ended |
|||||
2021 |
2020 |
2020 |
||||
Unaudited | Audited | |||||
Operating expenses: | ||||||
Research and development, net |
|
|
|
|||
Commercial activities |
4,430 |
1,468 |
8,748 |
|||
General and administrative |
3,413 |
2,994 |
12,167 |
|||
Operating loss |
19,209 |
12,341 |
62,300 |
|||
Finance expense |
1,690 |
919 |
10,640 |
|||
Finance income |
-2,413 |
-2,658 |
-236 |
|||
Loss before tax benefit |
18,486 |
10,602 |
72,704 |
|||
Tax benefit |
-447 |
- |
- |
|||
Net loss |
18,039 |
10,602 |
72,704 |
|||
Other comprehensive loss: | ||||||
Items that will be reclassified subsequently to profit or loss: | ||||||
Actuarial net gain of defined benefit plans |
- |
- |
-100 |
|||
Changes in the fair value of marketable securities |
- |
4 |
4 |
|||
Total comprehensive loss |
|
10,606 |
|
|||
Net loss per share: | ||||||
Basic loss per share |
|
|
|
|||
Diluted loss per share |
|
|
|
|||
Weighted average share count |
60,011,038 |
33,674,506 |
43,725,584 |
|||
INTERIM CONSOLIDATED STATEMENTS OF CASH FLOWS | |||||||||
Three months ended |
Year ended |
||||||||
2021 |
|
2020 |
|
2020 |
|
||||
Unaudited | Audited | ||||||||
Cash flows from operating activities: | |||||||||
Net loss |
$ (18,039 |
) |
$ (10,602 |
) |
$ (72,704 |
) |
|||
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 |
635 |
|
550 |
|
2,397 |
|
|||
Financial income (expense) net |
485 |
|
-132 |
|
483 |
|
|||
Share-based compensation |
1,014 |
|
899 |
|
2,864 |
|
|||
Change in employee benefit liabilities, net |
- |
|
- |
|
94 |
|
|||
Amortization of premium on available-for-sale financial assets |
- |
|
4 |
|
4 |
|
|||
Revaluation of liabilities presented at fair value |
-2,285 |
|
-2,448 |
|
6,822 |
|
|||
Revaluation of liability to IIA |
1,026 |
|
722 |
|
4,302 |
|
|||
Deferred income taxes |
-447 |
|
- |
|
- |
|
|||
428 |
|
-405 |
|
16,966 |
|
||||
Changes in asset and liability items: | |||||||||
Increase in prepaid expenses, other current assets and other assets |
-523 |
|
-458 |
|
-1,626 |
|
|||
Increase in trade payables |
875 |
|
1,934 |
|
5,083 |
|
|||
Increase (decrease) in accrued expenses and other payables |
-2,724 |
|
-3,096 |
|
3,454 |
|
|||
-2,372 |
|
-1,620 |
|
6,911 |
|
||||
Cash received during the period for: | |||||||||
Interest received |
- |
|
348 |
|
361 |
|
|||
Interest paid |
-51 |
|
-47 |
|
-161 |
|
|||
Net cash used in operating activities |
-20,034 |
|
-12,326 |
|
-48,627 |
|
|||
Cash flows from investing activities: | |||||||||
Purchase of property, plant and equipment |
-2,806 |
|
-2,119 |
|
-11,804 |
|
|||
Proceeds from maturity of marketable securities |
- |
|
- |
|
-158 |
|
|||
Proceeds from sale of marketable securities |
- |
|
13,551 |
|
13,551 |
|
|||
Net cash provided by (used in) investing activities |
$ (2,806 |
) |
|
|
|
|
|||
Three months ended |
Year ended |
||||||||
2021 |
|
2020 |
|
2020 |
|
||||
Unaudited | Audited | ||||||||
Cash flows from financing activities: | |||||||||
Proceeds from secondary offering, net |
- |
|
- |
|
133,316 |
|
|||
Receipt of grants from the IIA |
52 |
|
53 |
|
399 |
|
|||
Proceeds from issuance of convertible senior notes, net of issuance costs |
71,012 |
|
- |
|
- |
|
|||
Payment of lease liabilities |
-664 |
|
-787 |
|
-1,985 |
|
|||
Exercise of options |
502 |
|
6 |
|
650 |
|
|||
Payment of issuance costs related to public offering |
-468 |
|
- |
|
- |
|
|||
Net cash (used in) provided by financing activities |
70,434 |
|
-728 |
|
132,380 |
|
|||
Exchange differences on balances of cash and cash equivalents |
34 |
|
76 |
|
-10 |
|
|||
Increase (decrease) in cash and cash equivalents |
47,628 |
|
-1,546 |
|
85,332 |
|
|||
Cash and cash equivalents at beginning of period |
127,170 |
|
41,838 |
|
41,838 |
|
|||
Cash and cash equivalents at end of period |
|
|
|
|
|
|
|||
Supplemental disclosure of non-cash financing activities: | |||||||||
Significant non-cash transactions: | |||||||||
Lease liabilities arising from new right-of-use asset |
$ - |
|
$ - |
|
|
|
|||
IIA liability for grants to be received |
|
|
$ - |
|
|
|
|||
Issuance expenses on credit |
|
|
$ - |
|
|
|
|||
Purchase of property, plant and equipment on credit |
|
|
|
|
|
|
|||
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