Gamida Cell Reports First Quarter 2019 Financial Results and Provides Company Update

– Announces USAN selection of omidubicel as nonproprietary name for
company’s investigational NAM-expanded hematopoietic stem cells
(formerly known as NiCord
®) –

– Patient enrollment in Phase 3 study of omidubicel expected
to be completed in second half of 2019; Topline results expected in
first half of 2020 –

– Phase 1 clinical study of GDA-201 (formerly known as NAM-NK)
continues to progress, with additional data expected in 2H19 –

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 quarter
ended March 31, 2019. The company also highlighted continued progress in
advancing its clinical development candidates: omidubicel1
(formerly known as NiCord®), an investigational advanced cell
therapy in Phase 3 clinical development designed to enhance the
life-saving benefits of hematopoietic stem cell (bone marrow)
transplant, and GDA-201 (formerly known as NAM-NK), an investigational,
natural killer (NK) cell-based cancer immunotherapy in Phase 1
development in patients with non-Hodgkin lymphoma and multiple myeloma.

“Gamida Cell is focused on transforming the treatment landscape for
patients with blood cancers and rare, serious hematologic diseases. We
are pleased that omidubicel has been selected as the nonproprietary name
for NiCord, highlighting our progress toward bringing this important
cell therapy to patients in need of a bone marrow transplant,” stated
Julian Adams, Ph.D., chief executive officer of Gamida Cell. “We have
also made several key personnel appointments this year that reflect our
strategic focus on commercial preparedness, including hiring our first
chief commercial officer and nominating new board members who bring
commercial, operational and financial experience to Gamida Cell’s board
of directors.”

Dr. Adams continued, “We are pleased that the multi-center, randomized
Phase 3 study of omidubicel is progressing, with patient enrollment
expected to be complete by the end of this year and topline data
anticipated in the first half of 2020. Positive data from the study
would enable the submission of our first biologics license application
next year, which would be a significant achievement.”

“Earlier this year, we also reported encouraging data from the Phase 1
clinical study of our natural killer cell product candidate, GDA-201,
previously known as NAM-NK. The multiple complete responses observed
emboldened us to begin scaling up our manufacturing process to enable
the evaluation of a cryopreserved formulation of GDA-201 in a
multi-center, multi-dose Phase 1/2 clinical study in patients with
non-Hodgkin lymphoma next year,” Dr. Adams concluded.

Company Highlights

  • Omidubicel selected as nonproprietary name for NiCord: Today
    Gamida Cell announced that the United States Adopted Names (USAN)
    Council selected omidubicel as the nonproprietary name for Gamida
    Cell’s investigational hematopoietic stem cell expanded through the
    company’s proprietary nicotinamide-based, or NAM, technology. The USAN
    Council aims for global standardization and unification of drug
    nomenclature to ensure that drug information is communicated
    accurately and unambiguously. Gamida Cell’s lead investigational
    product has two components: omidubicel (hematopoietic stem cells
    expanded through the company’s proprietary nicotinamide-based, or NAM,
    technology) and differentiated immune cells, including T cells. Gamida
    Cell refers to the two components collectively as “omidubicel.” Going
    forward, Gamida Cell will use the name “omidubicel” in publications
    and public statements, at conferences and other forums, and in medical
    and commercial-related materials.
  • Reported encouraging data for omidubicel and GDA-201 at TCT Annual
    Meeting:
    In February, data from the omidubicel and GDA-201
    clinical programs 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 from the completed Phase 1/2 clinical study of
    omidubicel demonstrated that recipients who received omidubicel 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.

    Data were also reported
    from the ongoing Phase 1/2 study of omidubicel in patients with severe
    aplastic anemia. In the initial cohort of three patients, all
    successfully underwent a bone marrow transplant consisting of
    omidubicel plus a haploidentical stem cell graft. The results enable
    the initiation of a second cohort of patients to be treated with
    omidubicel as a stand-alone graft. Patient enrollment in the
    second cohort is expected to begin in the first half of 2019.

    Additionally,
    data reported from the ongoing Phase 1 study of GDA-201 in patients
    with non-Hodgkin lymphoma (NHL) and multiple myeloma (MM) demonstrated
    that GDA-201 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 GDA-201 was
    generally well tolerated, support continued clinical development.
    Gamida Cell is planning to initiate a multi-center, Phase 1/2 clinical
    study of GDA-201 in patients with NHL in 2020.

  • Evolved Board of Directors to reflect company’s progress toward
    commercialization:
    In March, the company announced the
    nominations of Shawn Cline Tomasello and Stephen T. Wills to its board
    of directors. These nominations require approval at the Annual
    Shareholders Meeting, which will take place in June 2019. Ms.
    Tomasello has extensive experience in commercializing first-in-class
    medicines for the treatment of cancer, including Yescarta®
    (at Kite Pharma, now part of Gilead Sciences) and Imbruvica®
    (at Pharmacyclics, now part of AbbVie). Mr. Wills has extensive
    operational, financial and transactional experience over nearly three
    decades in the life sciences and accounting industries. He has served
    as chief financial officer of Palatin Technologies, a publicly-traded
    biotechnology company developing peptide therapeutics, since 1997 and
    also serves as Palatin’s chief operating officer and executive vice
    president.

    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.

  • Appointed Thomas Klima as chief commercial officer: In January,
    the company announced the appointment of Thomas Klima as chief
    commercial officer. In this newly created role, Mr. Klima will
    be responsible for building the team and executing the strategy to
    potentially bring omidubicel to patients, including oversight of
    reimbursement and patient services. 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.

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

Omidubicel

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

GDA-201

  • 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 patients with NHL
    in 2020

First Quarter 2019 Financial Results

  • As of March 31, 2019, Gamida Cell had total cash, cash equivalents and
    available-for-sale securities of $50.3 million, compared to $60.7
    million as of December 31, 2018.
  • Research and development expenses in the first quarter of 2019 were
    $7.3 million, compared to $5.1 million in the same period in 2018. The
    difference was attributable mainly to a $1.2 million increase in
    clinical activities relate to the advancement of omidubicel and
    GDA-201, $0.5 million reduction in grants received from the Israeli
    Innovation Authority (IIA) and an increase of $0.5 million in
    compensation and other R&D expenses.
  • General and administrative expenses were $3.8 million for the first
    quarter of 2019, compared to $1.7 million in the same period in 2018.
    The increase was due mainly to a $1.0 million increase in expenses
    related to hiring and establishing the U.S. headquarters, an increase
    of $0.5 million in non-cash stock-based compensation expenses, and
    $0.6 million in professional services, rent and other expenses.
  • Finance expenses, net, were $4.4 million for the three months ended
    March 31, 2019, compared to $0.7 million in income in the same period
    in 2018. The increase was primarily due to noncash expenses resulting
    from revaluation of warrants and the revaluation of royalty-bearing
    grant IIA liability.
  • Net loss for the first quarter of 2019 was $15.5 million, compared to
    a net loss of $7.4 million in the same period in 2018.

2019 Financial Guidance
Gamida Cell continues to expect 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, available-for-sale
securities and short-term debt will support the company’s capital needs
through the data readout for the Phase 3 clinical study of omidubicel,
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.

Conference Call Information
Gamida Cell will host a
conference call today, May 7, 2019, at 8:30 a.m. ET to discuss these
financial results and company updates. A live webcast of the conference
call can be accessed in the “Investors” section of Gamida Cell’s website
at www.gamida-cell.com.
To participate in the live call, please dial 866-930-5560 (domestic) or
409-216-0605 (international) and refer to conference ID number 2277888.
A replay of the webcast will be available for approximately 30 days.

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).1 Omidubicel is the first bone
marrow transplant product to receive Breakthrough Therapy Designation
from the U.S. Food and Drug Administration and has also received Orphan
Drug Designation in the U.S. and EU. In a Phase 1/2 clinical study,
omidubicel demonstrated rapid and durable time to engraftment and was
generally well-tolerated.2 A Phase 3 study evaluating
omidubicel in patients with leukemia and lymphoma is ongoing in the
U.S., Europe and Asia.3 Omidubicel is also being evaluated in
a Phase 1/2 clinical study in patients with severe aplastic anemia.4
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.

About GDA-201
Gamida Cell applied the capabilities of its
NAM-based cell expansion technology to develop GDA-201 (formerly known
as NAM-NK), an innate natural killer (NK) cell immunotherapy for the
treatment of hematologic and solid tumors in combination with standard
of care antibody therapies. GDA-201 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. GDA-201 is in Phase 1 development through an
investigator-sponsored study in patients with refractory non-Hodgkin
lymphoma and multiple myeloma.5

Omidubicel and GDA-201 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.

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 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.

           
INTERIM CONSOLIDATED STATEMENTS OF FINANCIAL POSITION
 
U.S. dollars in thousands
 
March 31, December 31,
2019 2018
 

ASSETS

 

CURRENT ASSETS:

 
Cash and cash equivalents $ 43,749 $ 40,272
Available-for-sale financial assets 6,507 20,417
Prepaid expenses and other current assets   684   1,502
 

Total current assets

  50,940   62,191
 

NON-CURRENT ASSETS:

Property and equipment, net 2,782 2,311
Right-of-use assets 6,668
Other assets   657   662
 

Total non-current assets

  10,107   2,973
 

Total assets

$ 61,047 $ 65,164
 
 

LIABILITIES AND EQUITY

 

CURRENT LIABILITIES:

Trade payables $ 1,341 $ 1,985
Employees and payroll accruals 2,580 2,888
Current maturities of lease liabilities 2,156
Accrued expenses and other payables   1,739   1,832
 

Total current liabilities

  7,816   6,705
 

NON-CURRENT LIABILITIES:

Liabilities presented at fair value 25,031 24,049
Employee benefit liabilities, net 276 183
Lease Liabilities 4,671
Liability to Israel Innovation Authority (IIA)   10,108   9,540
 

Total non-current
liabilities

  40,086   33,772
 

SHAREHOLDERS’ EQUITY:

Share capital 68 67
Share premium 197,967 193,953
Capital reserve due to actuarial gains (160) (77)
Available-for-sale reserve (10) (43)
Accumulated deficit   (184,720)   (169,213)
 

Total shareholders’ equity

  13,145   24,687
 

Total liabilities and
shareholders’ equity

$ 61,047 $ 65,164

1 Gamida Cell’s lead development candidate consists of
omidubicel (expanded hematopoietic stem cells) and differentiated immune
cells, including T cells. Gamida Cell refers to the two components
collectively as “omidubicel.”

2 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.

3 ClinicalTrials.gov identifier NCT02730299.

4 ClinicalTrials.gov identifier NCT03173937.

5 ClinicalTrials.gov identifier NCT03019666.

Contacts

Jaren Irene Madden
jaren@gamida-cell.com
617-892-9084

Krystle Gibbs (media)
krystle@tenbridgecommunications.com
508-479-6358

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