Gamida Cell Reports Third Quarter 2019 Financial Results and Provides Company Update
– Completion of patient enrollment in Phase 3 clinical study of omidubicel expected by year-end; Topline data anticipated in first half of 2020 –
– Additional results from Phase 1 study of GDA-201 and new data on NAM technology platform to be presented at 61st Annual Meeting of the
“During the quarter, we made important progress toward our goal of developing next-generation cell therapies with the potential to redefine standards of care for patients with blood cancers and rare, serious hematologic diseases,” stated
“Our second cell therapy program, GDA-201, is also advancing, and next month we will report additional data from the ongoing Phase 1 study at the Annual Meeting of the
Program Highlights
-
Announced presentation of new data at the 61st Annual Meeting of the
American Society of Hematology (ASH): Last week,Gamida Cell announced that additional data from the ongoing Phase 1 clinical study of GDA-201 will be presented during an oral session at the ASH 2019 Annual Meeting, which is being heldDecember 7 – 10 inOrlando, FL. The presentation, “Results of a Phase 1 Trial of GDA-201, Nicotinamide-Expanded Allogeneic Natural Killer Cells (NAM-NK) in Patients with Refractory Non-Hodgkin Lymphoma (NHL ) and Multiple Myeloma (MM)” (Abstract #777), will take place onMonday, December 9 , at3:15 p.m. ET .
Additionally, new research on the mechanism of action of Gamida Cell’s NAM-based cell expansion platform, which is designed to enhance the number and functionality of allogeneic donor cells, will also be shared during the meeting. The poster presentation, “Nicotinamide (NAM) Modulates Transcriptional Signature of Ex Vivo Cultured UCB CD34+ Cells (Omidubicel) and Preserves Their Stemness and Engraftment Potential” (Abstract #3718), will take place onMonday, December 9 , from 6:00 –8:00 p.m. ET .
- Advanced the Phase 3 clinical study of omidubicel: Patient enrollment continued to progress in the Gamida Cell’s Phase 3 study of omidubicel in patients with high-risk hematologic malignancies. The international, randomized, multi-center study is designed to evaluate the safety and efficacy of omidubicel compared to standard umbilical cord blood for allogeneic bone marrow transplant in approximately 120 patients with no available matched donor. The company anticipates completing patient enrollment by the end of this year with topline data anticipated in first half of 2020.
-
Initiated health outcomes research for omidubicel: In September,
Gamida Cell and the CIBMTR® (Center for International Blood and Marrow Transplant Research ) announced a research agreement to evaluate outcomes of patients with hematological malignancies who undergo allogeneic hematopoietic stem cell transplant (bone marrow transplant) from various donor sources. The recently launched observational study includes both retrospective and prospective data contemporaneous to the Phase 3 study of omidubicel. The goal of this real-world, observational study is to better understand the variables that influence the health outcomes of patients receiving a transplant from a source other than a fully matched family donor.
-
Continued to focus on activities required to successfully bring omidubicel to patients:
Gamida Cell is continuing to advance key activities required to bring omidubicel to patients in a commercial setting, including building out manufacturing infrastructure, assembling an experienced commercial team with expertise in cell therapy and transplant, establishing hospital services and patient assistance programs, and exploring coverage and reimbursement models to enable access.
- Progressed enrollment in the Phase 1/2 study of omidubicel in patients with severe aplastic anemia: Enrollment is ongoing in a Phase 1/2 clinical study of omidubicel in patients with severe aplastic anemia, a rare, life-threatening bone marrow failure disease. Earlier this year, encouraging data from the first cohort of patients were reported at the 2019 Transplantation & Cellular Therapy (TCT) Meeting that demonstrated that all three patients in the cohort successfully underwent a bone marrow transplant consisting of omidubicel plus a haploidentical stem cell graft. The rapid engraftment, sustained hematopoiesis and accelerated immune recovery observed enabled the initiation of a second cohort, where patients will be treated with omidubicel as a stand-alone graft.
- Continued to prepare for the next clinical study of GDA-201: The company is continuing its work to enable a Phase 1/2 multi-dose, multi-center study of GDA-201 in patients with non-Hodgkin lymphoma, which is expected to begin in 2020. The decision to focus the next clinical study on non-Hodgkin lymphoma is based on the encouraging clinical data reported at the 2019 TCT Meeting which demonstrated the GDA-201 was generally well tolerated and clinically active, with multiple complete responses observed.
Third Quarter 2019 Financial Results
-
Research and development (R&D) expenses in the third quarter of 2019 were
$7.4 million compared to$5.1 million for the same period in 2018. R&D expenses were higher in the third quarter of 2019 compared to the same period in 2018 due to the advancement of omidubicel and GDA-201.
-
General and administrative expenses were
$4.6 million for the third quarter of 2019, compared to$2.4 million for the same period in 2018. The difference was attributable mainly to a$1.2 million increase in activities related to commercial readiness, as well as$1.0 million increase in professional services and other expenses, including an increase in expenses associated with being a publicly-traded company.
-
Finance income, net, was
$1.7 million for the third quarter of 2019, compared to finance expenses, net, of$2.2 million for the same period in 2018. The net increase was primarily due to non-cash income resulting from the re-valuation of warrants, offset by non-cash expenses from the re-valuation of theIsraeli Innovation Authority royalty-bearing grant liability.
-
Net loss for the third quarter of 2019 was
$10.1 million , compared to a net loss of$9.8 million for the same period in 2018.
-
As of
September 30, 2019 ,Gamida Cell had total cash, cash equivalents and available-for-sale assets of$68.1 million , compared to$60.7 million as ofDecember 31, 2018 .
2019 Financial Guidance
Conference Call Information
About Omidubicel
Omidubicel (formerly known as NiCord®), the company’s lead clinical program, 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). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the
About GDA-201
Omidubicel and GDA-201 are investigational therapies, and their safety and efficacy have not been evaluated by the
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 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
INTERIM CONSOLIDATED STATEMENTS OF FINANCIAL POSITION |
||||||||||||
U.S. dollars in thousands |
||||||||||||
|
|
September 30, |
|
Dec 31, |
||||||||
|
|
2019 |
|
2018 |
|
2018 |
||||||
|
|
Unaudited |
|
Audited |
||||||||
ASSETS |
|
|
|
|
|
|
||||||
|
|
|
|
|
|
|
||||||
CURRENT ASSETS: |
|
|
|
|
|
|
||||||
Cash and cash equivalents |
|
$ |
39,573 |
|
$ |
14,109 |
|
$ |
40,272 |
|||
Available-for-sale financial assets |
|
|
28,544 |
|
|
9,570 |
|
|
20,417 |
|||
Prepaid expenses and other current assets |
|
|
1,134 |
|
|
1,018 |
|
|
1,502 |
|||
|
|
|
|
|
|
|
||||||
Total current assets |
|
|
69,251 |
|
|
24,697 |
|
|
62,191 |
|||
|
|
|
|
|
|
|
||||||
NON-CURRENT ASSETS: |
|
|
|
|
|
|
||||||
Property and equipment, net |
|
|
4,209 |
|
|
1,743 |
|
|
2,311 |
|||
Right-of-use assets |
|
|
5,568 |
|
|
- |
|
|
- |
|||
Other assets |
|
|
651 |
|
|
354 |
|
|
662 |
|||
Deferred issuance cost |
|
|
- |
|
|
1,718 |
|
|
- |
|||
|
|
|
|
|
|
|
||||||
Total non-current assets |
|
|
10,428 |
|
|
3,815 |
|
|
2,973 |
|||
|
|
|
|
|
|
|
||||||
Total assets |
|
$ |
79,679 |
|
$ |
28,512 |
|
$ |
65,164 |
LIABILITIES AND EQUITY |
|
|
|
|
|
|
||||||
|
|
|
|
|
|
|
||||||
CURRENT LIABILITIES: |
|
|
|
|
|
|
||||||
Trade payables |
|
$ |
2,105 |
|
$ |
3,060 |
|
|
$ |
1,985 |
||
Employees and payroll accruals |
|
|
3,096 |
|
|
2,128 |
|
|
|
2,888 |
||
Current maturities of lease liabilities |
|
|
1,926 |
|
|
- |
|
|
|
- |
||
Accrued expenses and other payables |
|
|
1,979 |
|
|
2,111 |
|
|
|
1,832 |
||
|
|
|
|
|
|
|
||||||
Total current liabilities |
|
|
9,106 |
|
|
7,299 |
|
|
|
6,705 |
||
|
|
|
|
|
|
|
||||||
NON-CURRENT LIABILITIES: |
|
|
|
|
|
|
||||||
Liabilities presented at fair value |
|
|
5,434 |
|
|
15,400 |
|
|
|
24,049 |
||
Employee benefit liabilities, net |
|
|
280 |
|
|
194 |
|
|
|
183 |
||
Lease liability |
|
|
4,342 |
|
|
- |
|
|
|
- |
||
Liability to Israel Innovation Authority (IIA) |
|
|
11,594 |
|
|
10,474 |
|
|
|
9,540 |
||
|
|
|
|
|
|
|
||||||
Total non-current liabilities |
|
|
21,650 |
|
|
26,068 |
|
|
|
33,772 |
||
|
|
|
|
|
|
|
||||||
SHAREHOLDERS' EQUITY: |
|
|
|
|
|
|
||||||
Total shareholders' equity |
|
|
48,923 |
|
|
(4,855 |
) |
|
|
24,687 |
||
|
|
|
|
|
|
|
||||||
Total liabilities and shareholders' equity |
|
$ |
79,679 |
|
$ |
28,512 |
|
|
$ |
65,164 |
INTERIM CONSOLIDATED STATEMENTS OF COMPREHENSIVE INCOME |
|||||||||||||||||||||
U.S. dollars in thousands (except share and per share data) |
|||||||||||||||||||||
|
|
Nine months ended
|
|
Three months ended
|
|
Year ended
|
|||||||||||||||
|
|
2019 |
|
2018 |
|
2019 |
|
2018 |
|
2018 |
|||||||||||
|
|
Unaudited |
|
Audited |
|||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|||||||||||
Research and development, net |
|
$ |
21,682 |
|
|
$ |
17,169 |
|
|
$ |
7,363 |
|
|
$ |
5,132 |
|
|
$ |
22,045 |
|
|
General and administrative |
|
|
12,195 |
|
|
|
7,008 |
|
|
|
4,621 |
|
|
|
2,438 |
|
|
|
11,599 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
Operating loss |
|
|
33,877 |
|
|
|
24,177 |
|
|
|
11,984 |
|
|
|
7,570 |
|
|
|
33,644 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
Finance expenses |
|
|
2,499 |
|
|
|
6,560 |
|
|
|
895 |
|
|
|
2,356 |
|
|
|
20,259 |
|
|
Finance income |
|
|
(16,665 |
) |
|
|
(434 |
) |
|
|
(2,613 |
) |
|
|
(104 |
) |
|
|
(1,042 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
Loss (income) before taxes on income |
|
|
19,711 |
|
|
|
30,303 |
|
|
|
10,266 |
|
|
|
9,822 |
|
|
|
52,861 |
|
|
Taxes on income (benefit) |
|
|
(70 |
) |
|
|
- |
|
|
|
(170 |
) |
|
|
- |
|
|
|
70 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
Net loss |
|
|
19,641 |
|
|
|
30,303 |
|
|
|
10,096 |
|
|
|
9,822 |
|
|
|
52,931 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
Net loss (income) per share: |
|
|
|
|
|||||||||||||||||
|
|
|
|
|
|||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
Basic net loss per share |
|
$ |
0.70 |
|
|
$ |
43.92 |
|
|
$ |
0.30 |
|
|
$ |
14.23 |
|
|
$ |
10.53 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||
Diluted net loss per share |
|
$ |
1.24 |
|
|
$ |
43.92 |
|
|
$ |
0.30 |
|
|
$ |
14.23 |
|
|
$ |
10.53 |
|
|
INTERIM CONSOLIDATED STATEMENTS OF CASH FLOWS |
||||||||||||||||||||
U.S. dollars in thousands |
||||||||||||||||||||
|
|
Nine months ended
|
|
Three months ended
|
|
Year ended
|
||||||||||||||
|
|
2019 |
|
2018 |
|
2019 |
|
2018 |
|
2018 |
||||||||||
|
|
Unaudited |
|
Audited |
||||||||||||||||
Cash flows from operating activities: |
|
|
|
|
|
|
|
|
|
|
||||||||||
|
|
|
|
|
|
|
|
|
|
|
||||||||||
Net loss |
|
$ |
(19,641 |
) |
|
$ |
(30,303 |
) |
|
$ |
(10,096 |
) |
|
$ |
(9,822 |
) |
|
$ |
(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 |
|
|
1,792 |
|
|
|
146 |
|
|
|
547 |
|
|
|
49 |
|
|
|
269 |
|
Financial (income) expenses, net |
|
|
(768 |
) |
|
|
(161 |
) |
|
|
(199 |
) |
|
|
214 |
|
|
|
(858 |
) |
Cost of share-based compensation |
|
|
3,731 |
|
|
|
2,503 |
|
|
|
1,321 |
|
|
|
880 |
|
|
|
3,575 |
|
Change in employee benefit liabilities, net |
|
|
14 |
|
|
|
(6 |
) |
|
|
6 |
|
|
|
(23 |
) |
|
|
(15 |
) |
Amortization of premium on available-for-sale financial assets |
|
|
150 |
|
|
|
191 |
|
|
|
49 |
|
|
|
200 |
|
|
|
272 |
|
Revaluation of financial derivatives |
|
|
(15,691 |
) |
|
|
5,100 |
|
|
|
(2,220 |
) |
|
|
1,700 |
|
|
|
17,600 |
|
Revaluation of liability to IIA |
|
|
1,852 |
|
|
|
3,167 |
|
|
|
653 |
|
|
|
567 |
|
|
|
2,037 |
|
|
|
|
|
|
|
|
|
|
|
|
||||||||||
|
|
|
(8,920 |
) |
|
|
10,940 |
|
|
|
157 |
|
|
|
3,587 |
|
|
|
22,880 |
|
Changes in asset and liability items: |
|
|
|
|
|
|
|
|
|
|
||||||||||
|
|
|
|
|
|
|
|
|
|
|
||||||||||
Decrease (increase) in prepaid expenses and other current assets and other assets |
|
|
113 |
|
|
|
(1,266 |
) |
|
|
(4 |
) |
|
|
(1,637 |
) |
|
|
942 |
|
Increase (decrease) in trade payables |
|
|
120 |
|
|
|
670 |
|
|
|
(124 |
) |
|
|
1,902 |
|
|
|
(405 |
) |
Increase (decrease) in accrued expenses and other payables and employee and payroll accrual |
|
|
680 |
|
|
|
1,071 |
|
|
|
518 |
|
|
|
(8 |
) |
|
|
2,296 |
|
|
|
|
|
|
|
|
|
|
|
|
||||||||||
|
|
|
913 |
|
|
|
475 |
|
|
|
390 |
|
|
|
257 |
|
|
|
2,833 |
|
|
|
|
|
|
|
|
|
|
|
|
||||||||||
Cash received during the period for: |
|
|
|
|
|
|
|
|
|
|
||||||||||
Interest received |
|
|
1,132 |
|
|
|
570 |
|
|
|
302 |
|
|
|
179 |
|
|
|
792 |
|
Interest paid |
|
|
(92 |
) |
|
|
- |
|
|
|
(41 |
) |
|
|
- |
|
|
|
- |
|
|
|
|
|
|
|
|
|
|
|
|
||||||||||
|
|
|
1,040 |
|
|
|
570 |
|
|
|
261 |
|
|
|
179 |
|
|
|
792 |
|
|
|
|
|
|
|
|
|
|
|
|
||||||||||
Net cash used in operating activities |
|
|
(26,608 |
) |
|
|
(18,318 |
) |
|
|
(9,288 |
) |
|
|
(5,799 |
) |
|
|
(26,426 |
) |
|
|
|
|
|
|
|
|
|
|
|
||||||||||
Cash flows from investing activities: |
|
|
|
|
|
|
|
|
|
|
||||||||||
|
|
|
|
|
|
|
|
|
|
|
||||||||||
Purchase of property and equipment |
|
|
(2,139 |
) |
|
|
(949 |
) |
|
|
(1,261 |
) |
|
|
(246 |
) |
|
|
(1,645 |
) |
Purchase of available-for-sale financial assets |
|
|
(32,021 |
) |
|
|
- |
|
|
|
(32,021 |
) |
|
|
- |
|
|
|
(10,905 |
) |
Proceed from sale of available-for-sale financial assets |
|
|
- |
|
|
|
4,984 |
|
|
|
- |
|
|
|
- |
|
|
|
4,949 |
|
Proceed from maturity of available-for-sale financial assets |
|
|
23,789 |
|
|
|
- |
|
|
|
8,049 |
|
|
|
- |
|
|
|
- |
|
Proceeds from bank deposits |
|
|
- |
|
|
|
5,000 |
|
|
|
- |
|
|
|
- |
|
|
|
5,000 |
|
Investment in restricted bank deposits |
|
|
- |
|
|
|
(150 |
) |
|
|
- |
|
|
|
(150 |
) |
|
|
(150 |
) |
|
|
|
|
|
|
|
|
|
|
|
||||||||||
Net cash provided by (used in) investing activities |
|
|
(10,371 |
) |
|
|
8,885 |
|
|
|
(25,233 |
) |
|
|
(396 |
) |
|
|
(2,751 |
) |
INTERIM CONSOLIDATED STATEMENTS OF CASH FLOWS |
||||||||||||||||||||
U.S. dollars in thousands |
||||||||||||||||||||
|
|
Nine months ended September 30, |
|
Three months ended September 30, |
|
Year ended December 31, |
||||||||||||||
|
|
2019 |
|
2018 |
|
2019 |
|
2018 |
|
2018 |
||||||||||
|
|
Unaudited |
|
|
||||||||||||||||
Cash flows from financing activities: |
|
|
|
|
|
|
|
|
|
|
||||||||||
|
|
|
|
|
|
|
|
|
|
|
||||||||||
Proceeds from secondary offering, net |
|
|
37,235 |
|
|
|
- |
|
|
|
37,343 |
|
|
|
- |
|
|
|
- |
|
Receipt of grants from the IIA |
|
|
202 |
|
|
|
2,953 |
|
|
|
35 |
|
|
|
1,300 |
|
|
|
612 |
|
Proceeds from issuance of shares, initial public offering (payment of issuance expenses), net |
|
|
(238 |
) |
|
|
- |
|
|
|
- |
|
|
|
- |
|
|
|
47,479 |
|
Payment of lease liabilities |
|
|
(1,144 |
) |
|
|
- |
|
|
|
(380 |
) |
|
|
- |
|
|
|
- |
|
Exercise of options |
|
|
120 |
|
|
|
- |
|
|
|
3 |
|
|
|
- |
|
|
|
2 |
|
Increase in deferred issuance cost |
|
|
- |
|
|
|
(736 |
) |
|
|
- |
|
|
|
- |
|
|
|
||
|
|
|
|
|
|
|
|
|
|
|
||||||||||
Net cash provided by financing activities |
|
|
36,175 |
|
|
|
2,217 |
|
|
|
37,001 |
|
|
|
1,300 |
|
|
|
48,093 |
|
|
|
|
|
|
|
|
|
|
|
|
||||||||||
Exchange differences on balances of cash and cash equivalents |
|
|
105 |
|
|
|
- |
|
|
|
15 |
|
|
|
- |
|
|
|
31 |
|
|
|
|
|
|
|
|
|
|
|
|
||||||||||
Increase (decrease) in cash and cash equivalents |
|
|
(699 |
) |
|
|
(7,216 |
) |
|
|
2,495 |
|
|
|
(4,895 |
) |
|
|
18,947 |
|
Cash and cash equivalents at beginning of period |
|
|
40,272 |
|
|
|
21,325 |
|
|
|
37,078 |
|
|
|
19,004 |
|
|
|
21,325 |
|
|
|
|
|
|
|
|
|
|
|
|
||||||||||
Cash and cash equivalents at end of period |
|
$ |
39,573 |
|
|
$ |
14,109 |
|
|
$ |
39,573 |
|
|
$ |
14,109 |
|
|
$ |
40,272 |
|
|
|
|
|
|
|
|
|
|
|
|
||||||||||
|
|
|
|
|
|
|
|
|
|
|
||||||||||
|
|
|
|
|
|
|
|
|
|
|
1Horwitz 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.
2ClinicalTrials.gov identifier NCT02730299.
3ClinicalTrials.gov identifier NCT03173937.
4ClinicalTrials.gov identifier NCT03019666.
View source version on businesswire.com: https://www.businesswire.com/news/home/20191113005671/en/
Source:
Jaren Irene Madden
jaren@gamida-cell.com
1-617-286-6264
Krystle Gibbs (media)
krystle@tenbridgecommunications.com
508-479-6358