Gamida Cell Reports Fourth Quarter and Full Year 2018 Financial Results and Provides Company Update

– Successfully Completed IPO Raising $53.2 Million in Gross Proceeds –

– Patient Enrollment in Phase 3 Study of NiCord® on
Track for Completion in Second Half of 2019; Topline Results Expected in
First Half of 2020 –

– Recently Presented NAM-NK and NiCord Data at 2019 TCT Annual
Meeting Demonstrate Clinical Progress and Potential of Proprietary NAM
Expansion Technology –

BOSTON–(BUSINESS WIRE)–lt;a href=”https://twitter.com/search?q=%24GMDA&src=ctag” target=”_blank”gt;$GMDAlt;/agt;–Gamida
Cell Ltd.
(Nasdaq:GMDA), a leading cellular and immune therapeutics
company, today reported financial results for the fourth quarter and
full year ended December 31, 2018, and provided a business update, which
highlights the company’s progress advancing its clinical development
candidates: NiCord®, an investigational advanced cell therapy
in Phase 3 clinical development designed to enhance and expand the
life-saving benefits of hematopoietic stem cell (bone marrow)
transplant, and NAM-NK, an investigational, cell-based cancer
immunotherapy in Phase 1 development in patients with non-Hodgkin
lymphoma and multiple myeloma.

Gamida Cell continues to anticipate completing patient enrollment in the
Phase 3 study of NiCord by the end of this year with topline data
anticipated in first half of 2020. The international, randomized,
multi-center study is designed to evaluate the safety and efficacy of
NiCord compared to standard umbilical cord blood for allogeneic bone
marrow transplant in approximately 120 patients with no available
matched donor. Additionally, the company continues to make progress in
its NAM-NK program, and expects to initiate a multi-center Phase 1/2
study of NAM-NK in patients with blood cancer in 2020.

“We are off to a strong start this year and are making meaningful
progress across every dimension of our company. The data presented last
week at the TCT Annual Meeting demonstrates the potential for NiCord to
offer a new cell therapy option for patients in need of a bone marrow
transplant,” stated Julian Adams, Ph.D., chief executive officer at
Gamida Cell. “Additionally, our collaboration with Be The Match
BioTherapies and the appointment of Tom Klima as chief commercial
officer underscore our commitment to advancing our clinical development
plans and beginning commercial readiness activities for the potential
approval and launch of NiCord.”

Dr. Adams continued, “We are committed to leveraging the transformative
potential of our proprietary nicotinamide-, or NAM-based, cell expansion
technology, to deliver a multi-product pipeline. Last week, we presented
encouraging data from our ongoing Phase 1 study of NAM-NK in patients
with non-Hodgkin lymphoma and multiple myeloma, and the emerging
clinical profile suggests it has the potential to address some of the
limitations of currently available cell therapies. In addition, we have
demonstrated the ability to cryopreserve our NK cells in a laboratory
setting, and we are working to scale up our manufacturing process to
enable the ability to evaluate multiple doses and schedules of NAM-NK
therapy in a multi-center, Phase 1/2 clinical study next year.”

Recent Company Highlights:

  • Reported additional immune reconstitution data for NiCord
    supporting clinical potential for bone marrow transplant:
    In
    February, translational data from the completed Phase 1/2 study of
    NiCord were reported at the 2019 Transplantation & Cellular Therapy
    (TCT) Meetings of American Society for Blood and Marrow
    Transplantation and Center for International Blood and Marrow
    Transplant Research demonstrating that recipients who received NiCord
    had rapid and robust reconstitution of key immune cells. Successful
    immune reconstitution is an important factor in the recovery of
    patients undergoing bone marrow transplant.
  • Reported initial data from Phase 1/2 study of NiCord in severe
    aplastic anemia:
    At the 2019 TCT Annual meeting, data were
    reported from the ongoing Phase 1/2 study of NiCord in patients with
    severe aplastic anemia. In the initial cohort of three patients, all
    successfully underwent a bone marrow transplant consisting of NiCord
    plus a haploidentical stem cell graft. The rapid cord engraftment,
    sustained hematopoiesis and accelerated immune recovery in treatment
    refractory observed in these patients enable the initiation of a
    second cohort of patients to be treated with NiCord as a stand-alone
    graft. Patient enrollment in the second cohort is expected to
    begin in the first half of 2019.
  • Announced encouraging data from Phase 1 study of NAM-NK in
    non-Hodgkin Lymphoma and multiple myeloma:
    During the 2019 TCT
    Annual Meeting, data reported from the ongoing Phase 1 study of NAM-NK
    in patients with non-Hodgkin lymphoma (NHL) and multiple myeloma (MM)
    demonstrated that NAM-NK was clinically active, with three complete
    responses observed in patients with NHL and one complete response in a
    patient with MM. These data, along with safety data showing that
    NAM-NK was generally well tolerated, support continued clinical
    development, and Gamida Cell is planning to initiate a multi-center,
    Phase 1/2 clinical study of NAM-NK in patients with blood cancers in
    2020.
  • Entered into agreement with Editas Medicine to evaluate potential
    to gene edit NAM-NK cells:
    In February, Gamida Cell announced an
    agreement with Editas Medicine, Inc. to evaluate the potential use of
    Editas Medicine’s CRISPR technology to edit NAM-NK cells. Through this
    agreement, the companies aim to rapidly discover optimized NAM-NK
    cells that could be used to improve the treatment of blood cancers and
    solid tumors.
  • Formed strategic collaboration with Be The Match BioTherapies: In
    January 2019, both organizations announced a collaboration to expand
    the use of bone marrow transplant to treat hematologic malignancies
    and serious blood disorders. Under the terms of the collaboration
    agreement, the organizations will explore opportunities to work
    together across Gamida Cell’s ongoing clinical development program for
    NiCord, including the ongoing Phase 3 clinical study. Be The Match
    BioTherapies has an extensive history of involvement in the delivery
    of cord blood units for transplant and broad access to cord blood
    banks globally.
  • Appointed Thomas Klima as Chief Commercial Officer: In January,
    the company announced the appointment of Thomas Klima as Chief
    Commercial Officer. Mr. Klima brings nearly 20 years of
    global experience in the pharmaceutical industry with expertise in
    cellular therapy, hematology, oncology and transplantation. During his
    career, he has played key roles in building commercial organizations
    and leading multiple successful product launches.
  • Appointed Nurit Benjamini to Board of Directors: In January,
    the company appointed Nurit Benjamini to Gamida Cell’s board of
    directors and chair of the board’s audit committee. Ms. Benjamini has
    served as chief financial officer of TabTale Ltd. since 2013.
    Previously, she held a number of chief financial officer positions,
    including at Wix.com Ltd., Sigma Designs Israel Ltd. and Compugen Ltd.

Anticipated 2019-2020 Milestones
Gamida Cell’s anticipated
program milestones in 2019-2020 are as follows:

Nicord®

  • Initiate Cohort 2 in the Phase 1/2 study evaluating NiCord as
    stand-alone graft in severe aplastic anemia in the first half of 2019
  • Complete enrollment in Phase 3 study of NiCord in patients with
    hematologic malignancies in the second half of 2019
  • Report topline data from the Phase 3 study of NiCord in patients with
    hematologic malignancies in the first half of 2020
  • Complete BLA filing for NiCord in hematologic malignancies in the
    second half of 2020, should Phase 3 data be positive

NAM-NK

  • Complete patient enrollment in the ongoing Phase 1 study in the second
    half of 2019
  • Present additional data at a medical meeting in the second half of 2019
  • Initiate multi-center, Phase 1/2 clinical study in 2020

Fourth Quarter and Full Year 2018 Financial Results:

  • At December 31, 2018, Gamida Cell had total cash, cash equivalents and
    available-for-sale securities of $60.7 million, compared to $41.1
    million at December 31, 2017.
  • Research and Development expenses in 2018 were $22.0 million, compared
    to $15.0 million in 2017. The increase was attributable mainly to a
    $5.4 million increase in clinical activities relating to the
    advancement of Gamida Cell’s clinical programs as well as an increase
    of $1.6 million in other R&D expenses.
  • General and administrative expenses were $11.6 million in 2018,
    compared to $4.5 million in 2017. The increase was attributable mainly
    to a $2.9 million increase in expenses related to expanding the
    management team and establishing the U.S. headquarters, an increase of
    $2.0 million in non-cash stock-based compensation expenses, and $2.2
    million in professional services expenses incurred during the IPO
    process, rent and other expenses.
  • Finance expenses, net, were $19.2 million in 2018, compared to $0.5
    million in income, in 2017. The increase was primarily due to non-cash
    expenses resulting from revaluation of warrants and the revaluation of
    royalty-bearing grant IIA liability.
  • Net loss for 2018 was $52.9 million, compared to a net loss of $19.1
    million for 2017.

2019 Financial Guidance
Gamida Cell expects cash used
for ongoing operating activities in 2019 to range from $35-$40 million,
reflecting anticipated expenditures to advance the company’s clinical
programs.

Gamida Cell expects that its cash, cash equivalents and
available-for-sale securities will support the company’s capital needs
through the data readout for the Phase 3 clinical study of NiCord, which
is expected in the first half of 2020. This cash runway guidance is
based on the company’s current operational plans and excludes any
additional funding that may be received or business development
activities that may be undertaken.

About NiCord
NiCord, the company’s lead clinical program, is
under development as a universal bone marrow transplant solution for
patients with high-risk hematologic malignancies. NiCord has been
granted Breakthrough Therapy designation by the U.S. Food and Drug
Administration, making it the first bone marrow transplant alternative
to receive this designation. It has also received U.S. and EU orphan
drug designation. A Phase 3 clinical study evaluating NiCord in patients
with leukemia and lymphoma is ongoing in the United States, Europe and
Asia.1 NiCord is also being evaluated in a Phase 1/2 clinical
study in patients with severe aplastic anemia.2 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 NiCord. For more information on
clinical trials of NiCord, please visit www.clinicaltrials.gov.

About NAM-NK
Gamida Cell applied the capabilities of its
NAM-based cell expansion technology to highly functional NK cells to
develop NAM-NK, an innate immunotherapy for the treatment of hematologic
and solid tumors in combination with standard of care antibody
therapies. NAM-NK addresses key limitations of NK cells by increasing
the cytotoxicity and in vivo retention and proliferation in the
bone marrow and lymphoid organs of NK cells expanded in culture. NAM-NK
is in Phase 1 development through an investigator-sponsored study in
patients with refractory non-Hodgkin lymphoma and multiple myeloma.3

NAM-NK and NiCord are investigational therapies, and their safety and
efficacy have not been evaluated by the U.S. Food and Drug
Administration or any other health authority.

About Gamida Cell
Gamida Cell is a clinical-stage
biopharmaceutical company committed to developing advanced cell
therapies with the potential to cure blood cancers and rare, serious
hematologic diseases. We are leveraging our proprietary
nicotinamide-based, or NAM-based, cell expansion technology to develop
product candidates designed to address the limitations of cell
therapies. For additional information, please visit www.gamida-cell.com.

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 SEC on February 25, 2019, and other filings that Gamida Cell makes
with the SEC from time to time (which are available at http://www.sec.gov),
the events and circumstances discussed in such forward-looking
statements may not occur, and Gamida Cell’s actual results could differ
materially and adversely from those anticipated or implied thereby. Any
forward-looking statements speak only as of the date of this press
release and are based on information available to Gamida Cell as of the
date of this release.

 
CONSOLIDATED STATEMENTS OF FINANCIAL POSITION
U.S. dollars in thousands    
      December 31,
2018 2017
ASSETS
 
CURRENT ASSETS:
Cash and cash equivalents $ 40,272 $ 21,325
Available-for-sale financial assets 20,417 14,758
Short term deposits 5,000
Prepaid expenses and other current assets   1,502   2,539
Total current assets   62,191   43,622
 
NON-CURRENT ASSETS:
Property and equipment, net 2,311 940
Other assets   662   360
Total non-current assets   2,973   1,300
 
Total assets $ 65,164 $ 44,922
 
LIABILITIES AND SHAREHOLDERS’ EQUITY
 
CURRENT LIABILITIES:
Trade payables $ 1,985 $ 2,390
Employees and payroll accruals 2,888 1,517
Accrued expenses and other payables   1,832   669
Total current liabilities   6,705   4,576
 
NON-CURRENT LIABILITIES:
Warrants presented at fair value 24,049 10,300
Employee benefit liabilities, net 183 200
Liability to Israel Innovation Authority (IIA)   9,540   6,890
Total non-current liabilities   33,772   17,390
 
CONTINGENT LIABILITIES AND COMMITMENTS
 
SHAREHOLDERS’ EQUITY:
Ordinary shares 67 2
Preferred shares 38
Share premium 193,953 139,311
Capital reserve due to actuarial loss (77) (79)
Available for sale reserve (43) (34)
Accumulated deficit   (169,213)   (116,282)
Total shareholders’ equity   24,687   22,956
 
Total liabilities and shareholders’ equity $ 65,164 $ 44,922
 
 
CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS
U.S. dollars in thousands
      Year ended
December 31,
2018     2017
 
Operating expenses:
Research and development expenses, net $ 22,045 $ 15,018
General and administrative expenses   11,599   4,472
Operating loss   33,644   19,490
 
Financial expenses 20,259 718
Financial income   (1,042)   (1,197)
Loss before taxes on income   52,861   19,011
 
Taxes on income 70
   
Net Loss   52,931   19,011
 
Other comprehensive loss: 7 69
   
Total comprehensive loss $ 52,938 $ 19,080
 
Loss per share – basic and diluted (in U.S. dollars) $ 10.53 $ 27.56
 

1ClinicalTrials.gov
identifier NCT02730299.
2ClinicalTrials.gov
identifier NCT03173937.
3ClinicalTrials.gov
identifier NCT03019666.

Contacts

Jaren Irene Madden
[email protected]
1-617-286-6264

Media Inquiries
Krystle Gibbs
[email protected]
508-479-6358

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