On February 25, 2019 Sesen Bio (Nasdaq: SESN), a late-stage clinical company developing targeted fusion protein therapeutics for the treatment of people with cancer, reported that Company management will host a conference call and webcast on Monday, March 4, 2019 at 8:00 a.m. EST to review operating results for the fourth quarter and full year ended December 31, 2018 and new, preliminary analyses from the Phase 3 VISTA trial of Vicinium for patients with high-risk non-muscle invasive bladder cancer who have been previously treated with bacillus Calmette-Guérin (Press release, Sesen Bio, FEB 25, 2019, View Source [SID1234533640]).

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To participate in the conference call, please dial (844) 831-3025 (domestic) or (315) 625-6887 (international) and refer to conference ID 2179668. The webcast can be accessed in the Investor Relations section of the Company’s website at www.sesenbio.com. The replay of the webcast will be available in the investor section of the Company’s website at www.sesenbio.com for 60 days following the call.

On February 25, 2019 Veracyte, Inc. (Nasdaq: VCYT) reported financial results and business progress for the quarter and full year ended December 31, 2018, and provided financial guidance for 2019 (Press release, Veracyte, FEB 25, 2019, Veracyte Announces Fourth Quarter and Full-Year 2018 Financial Results and Provides 2019 Financial Outlook
[SID1234533639]).

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"We delivered exceptional growth in 2018 and are off to a strong start in 2019," said Bonnie Anderson, Veracyte’s chairman and chief executive officer. "Our 2018 performance was driven by several factors. Our Afirma GSC and Xpression Atlas launch broadened our thyroid offering to inform both diagnosis and treatment decisions, and the first full year of Percepta classifier adoption was strong with the fourth quarter acceleration in growth positioning us for a strong 2019. Clearly, our multi-product sales strategy that is designed to give us the leverage we need to be a profitable enterprise is working."

Anderson added, "In 2018, we also entered into biopharmaceutical collaborations with Loxo Oncology and Johnson & Johnson Innovation and the Johnson & Johnson Lung Cancer Initiative that recognize the value of our novel scientific platform and can help advance our pipeline. As we look to 2019 and beyond, we believe we are well-positioned for continued success as we further increase the number of patients whose lives can be improved by our innovative tests."

Fourth Quarter and Full-Year 2018 Financial Results

For the three- and twelve-month periods ended December 31, 2018, compared to the prior year:

Revenue was $25.8 million and $92.0 million, respectively, an increase of 31% and 28%;
Gross Margin was 66% and 64%, respectively, an increase of 6% and 3%;
Operating Expenses, Excluding Cost of Revenue, were $20.1 million and $81.2 million, respectively, an increase of 12% and 15%;
Net Loss and Comprehensive Loss was ($3.1) million and ($23.0) million, respectively, an improvement of 63% and 26%;
Basic and Diluted Net Loss Per Common Share was ($0.08) and ($0.62), respectively, an improvement of 67% and 32%;
Net Cash Used in Operating Activities was $1.2 million and $13.5 million, respectively, an improvement of 79% and 44%;
Cash Burn1 was $1.7 million and $15.4 million, respectively, an improvement of 73% and 39%; and
Cash and Cash Equivalents were $78.0 million at December 31, 2018.
2018 Full-Year and Recent Business Highlights

Commercial Expansion:

Grew total genomic test volume to 9,154 tests in the fourth quarter of 2018, representing 28% growth over 2017, which resulted in full-year 2018 growth of 22% over 2017, or 31,710 tests.
Transitioned all Afirma customers to the second-generation Afirma Genomic Sequencing Classifier (GSC) platform and launched the Afirma Xpression Atlas to provide a comprehensive solution that informs both thyroid cancer diagnosis and treatment decisions. Notably, 30% of Afirma GSC orders included Xpression Atlas in 2018, ahead of the company’s expectations.
Grew Percepta Bronchial Genomic Classifier volume to nearly 1,550 tests in its first full year of commercialization, with genomic volume accelerating 74% sequentially from the third quarter to the fourth quarter of 2018.
Established 20 leading Early Access Program (EAP) sites across the United States for Envisia in 2018, addressing physician demand for patient access to the classifier which improves idiopathic pulmonary fibrosis (IPF) diagnosis and builds a solid foundation for the company to commercially expand it in 2019.
Biopharmaceutical Collaborations

Executed a long-term strategic collaboration with Johnson & Johnson, LLC and Johnson & Johnson’s Lung Cancer Initiative to advance diagnostics, including a nasal swab test, for early lung cancer detection. Veracyte estimates the combined monetary and non-monetary value of the collaboration to be more than $50 million. The company believes this collaboration expands its addressable lung cancer diagnostic market to a more than $30 billion global opportunity.
Entered into a research collaboration with Loxo Oncology, through which Loxo has access to data from Veracyte’s Afirma Xpression Atlas platform to help in its development of therapies for patients with genetically defined cancers, including thyroid cancer.
Reimbursement Progress:

Received draft Medicare coverage for the Envisia Genomic Classifier through the MolDX program, with a final positive coverage decision expected in early 2019.
Achieved in-network status as a service provider with the last of the major commercial health plans, which Veracyte believes will facilitate coverage and reimbursement for its Percepta and Envisia classifiers.
Evidence Development:

Afirma – Published clinical validation data for the Afirma GSC in JAMA Surgery, demonstrating the next-generation test’s ability to help approximately 70% of patients with indeterminate thyroid nodules avoid unnecessary surgery. Presented 12 Afirma studies at three endocrinology conferences, including real-world data showing that the Afirma GSC is helping even more patients avoid unnecessary surgery than is suggested by the clinical validation study findings.
Percepta – Presented early, interim results at the 2018 CHEST Annual Meeting from the ongoing registry clinical utility study showing the test changed clinical decision-making and reduced invasive procedures at every evaluation time point up to 12 months post-testing.
Envisia – Published a study quantifying and qualifying the challenges in obtaining timely, accurate diagnosis of IPF and other interstitial lung diseases, thus underscoring the clinical need for the Envisia classifier. Presented data at a leading pulmonology conference demonstrating the test’s ability to improve the diagnosis of IPF without the need for surgery.
Financing and Debt Facility:

In July 2018, Veracyte issued and sold 5,750,000 shares of common stock in a registered public offering, including the underwriters’ exercise in full of their option to purchase an additional 750,000 shares, at a price to the public of $10.25 per share. Net proceeds from the offering were approximately $55.0 million.
In January 2019, the company used $12.5 million of cash and cash equivalents to reduce its principal debt balance from $25.0 million to $12.5 million.
2019 Outlook

Veracyte is guiding to full-year 2019 revenue in the range of $113 million to $117 million and full-year 2019 net cash used in operating activities in the range of $4 million to $6 million.

Conference Call and Webcast Details

Veracyte will host a conference call and webcast today at 5:00 p.m. Eastern Time to discuss the company’s financial results and provide a general business update. The conference call will be webcast live from the company’s website and will be available via the following link: View Source The webcast should be accessed 10 minutes prior to the conference call start time. A replay of the webcast will be available for one year following the conclusion of the live broadcast and will be accessible on the company’s website at View Source approximately two hours following the completion of the call.

The conference call can be accessed as follows:

U.S./Canada participant dial-in number (toll-free): (855) 541-0980
International participant dial-in number: (970) 315-0440
Conference I.D.: 5498321

On February 25, 2019 Medpace Holdings, Inc. (Nasdaq: MEDP) ("Medpace") reported financial results for the fourth quarter and full year ended December 31, 2018 (Press release, Medpace, FEB 25, 2019, View Source [SID1234533638]).

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Fourth Quarter and Full Year 2018 Financial Results under ASC 606

Revenue for the three and twelve months ended December 31, 2018 was $192.1 million and $704.6 million, respectively. Backlog as of December 31, 2018 was $1.1 billion and net new business awards were $231.2 million, representing a net book-to-bill ratio of 1.20x for the fourth quarter of 2018. For the full year 2018, net new business awards were $899.4 million, representing a net book-to-bill ratio of 1.28x. The Company calculates the net book-to-bill ratio by dividing net new business awards by revenue.

For the fourth quarter of 2018 and full year 2018, total direct costs were $131.1 million and $489.1 million, respectively. Adjusted direct costs were $131.9 million and $492.2 million in the fourth quarter of 2018 and full year 2018, respectively. Selling, general and administrative (SG&A) expenses were $20.6 million and Adjusted SG&A expenses were $20.8 million for the fourth quarter of 2018. For the full year 2018, SG&A expenses were $75.7 million and Adjusted SG&A expenses were $75.7 million.

GAAP net income for the fourth quarter of 2018 was $22.8 million, or $0.61 per diluted share, which resulted in a net income margin of 11.8%. GAAP net income for the full year of 2018 was $73.2 million, or $1.97 per diluted share, which resulted in a net income margin of 10.4%. Adjusted EBITDA for the fourth quarter of 2018 was $39.7 million, or 20.7% of revenue. Adjusted EBITDA for the full year of 2018 was $137.8 million, or 19.6% of revenue. Adjusted Net Income was $28.1 million, and Adjusted Net Income per diluted share was $0.76 for the fourth quarter of 2018. Adjusted Net Income was $95.5 million, and Adjusted Net Income per diluted share was $2.59 for the full year of 2018.

Fourth Quarter and Full Year 2018 Financial Results under ASC 605

Net service revenue for the three months ended December 31, 2018 increased 28.6% to $127.9 million, compared to $99.4 million for the comparable prior-year period. Net service revenue for the year ended December 31, 2018 increased 23.7% to $478.1 million, compared to $386.5 million for the year ended December 31, 2017. On a constant currency basis, net service revenue for the fourth quarter of 2018 increased 29.1% compared to the fourth quarter of 2017 and increased 23.4% for the year ended December 31, 2018 compared to the year ended December 31, 2017.

Backlog as of December 31, 2018 grew 19.4% to $626.1 million from $524.4 million as of December 31, 2017. Net new business awards were $146.7 million, representing a net book-to-bill ratio of 1.15x for the fourth quarter of 2018, as compared to $114.7 million for the comparable prior-year period. For the year ended December 31, 2018, net new business awards were $581.0 million, representing a net book-to-bill ratio of 1.22x, compared to $426.1 million for the year ended December 31, 2017.

For the fourth quarter of 2018, Direct service costs, excluding depreciation and amortization, were $67.9 million, compared to $55.6 million in the fourth quarter of 2017. Adjusted Direct service costs were $68.7 million for the fourth quarter 2018, compared to $56.4 million in the fourth quarter of 2017. For the full year 2018, Direct service costs, excluding depreciation and amortization, were $252.3 million, compared to $211.8 million in the full year 2017. Adjusted Direct service costs were $255.4 million for the full year 2018, compared to $215.0 million in the full year 2017.

SG&A expenses were $20.6 million in the fourth quarter of 2018, compared to $16.8 million in the fourth quarter of 2017. Adjusted SG&A expenses were $20.8 million for the fourth quarter 2018 versus $16.4 million in the fourth quarter of 2017. For the full year 2018, SG&A expenses were $75.7 million, compared to $63.4 million for the full year 2017. Adjusted SG&A expenses were $75.7 million for the full year 2018 versus $63.1 million for the full year 2017.

GAAP net income for the fourth quarter of 2018 was $22.5 million, or $0.60 per diluted share, versus GAAP net income of $11.3 million, or $0.30 per diluted share, for the fourth quarter of 2017. This resulted in a net income margin of 17.6% and 11.4% for the fourth quarter of 2018 and 2017, respectively. GAAP net income for full year 2018 was $81.6 million, or $2.20 per diluted share, versus GAAP net income of $39.1 million, or $0.98 per diluted share, for the full year 2017. This resulted in a net income margin of 17.1% and 10.1% for the full year 2018 and 2017, respectively.

Adjusted EBITDA for the fourth quarter of 2018 increased 43.0% to $38.6 million, or 30.2% of net service revenue, compared to $27.0 million, or 27.2% of net service revenue, for the comparable prior-year period. Adjusted EBITDA for the full year 2018 increased 37.0% to $148.0 million, or 31.0% of net service revenue, compared to $108.0 million, or 28.0% of net service revenue, for the prior year. On a constant currency basis, Adjusted EBITDA for the fourth quarter of 2018 increased 38.0% from the fourth quarter of 2017 and increased 36.2% for the full year 2018 compared to the full year 2017.

Adjusted Net Income for the fourth quarter of 2018 increased 88.2% to $27.8 million, compared to $14.8 million for the comparable prior-year period. Adjusted Net Income per diluted share for the fourth quarter of 2018 was $0.75 compared to Adjusted Net Income per diluted share of $0.39 for the comparable prior-year period. Adjusted Net Income for the full year 2018 increased 71.7% to $103.8 million, compared to $60.5 million for the prior year. Adjusted Net Income per diluted share for the full year 2018 was $2.81 compared to Adjusted Net Income per diluted share of $1.52 for the prior year.

A reconciliation of the Company’s non-GAAP financial measures, including EBITDA, Adjusted EBITDA, Adjusted EBITDA margin, Adjusted Direct costs, Adjusted Selling, general and administrative expenses, Adjusted Net Income, and Adjusted Net Income per diluted share to the corresponding GAAP measures is provided below.

Balance Sheet and Liquidity

The Company’s Cash and cash equivalents were $23.3 million at December 31, 2018, and the Company generated $39.4 million in cash flow from operating activities during the fourth quarter of 2018.

Financial Guidance

For full year 2019, the Company is providing guidance under ASC 606. The Company forecasts 2019 revenue in the range of $783.0 million to $807.0 million, representing growth of 11.1% to 14.5% over 2018 revenue of $704.6 million. GAAP net income for full year 2019 is forecasted in the range of $85.2 million to $89.2 million. Additionally, full year 2019 Adjusted EBITDA is expected in the range of $137.0 million to $145.0 million.

Based on forecasted 2019 revenue of $783.0 million to $807.0 million and GAAP net income of $85.2 million to $89.2 million, diluted earnings per share (GAAP) is forecasted in the range of $2.27 to $2.38. Adjusted Net Income for 2019 is forecasted in the range of $97.0 million to $101.0 million, compared to Adjusted Net Income of $95.5 million for 2018. Furthermore, Adjusted Net Income per diluted share for 2019 is expected in the range of $2.58 to $2.69 per share.

Conference Call Details

Medpace will host a conference call at 9:00 a.m. ET, Tuesday, February 26, 2019, to discuss its fourth quarter and full year 2018 results.

To participate in the conference call, dial 800-219-7113 (domestic) or 574-990-1030 (international) using the passcode 5975717.

To access the conference call via webcast, visit the "Investors" section of Medpace’s website at medpace.com. The webcast replay of the call will be available at the same site approximately one hour after the end of the call.

A supplemental slide presentation will also be available at the "Investors" section of Medpace’s website prior to the start of the call.

A recording of the call will be available at 12:00 p.m. ET on Tuesday, February 26, 2019 until 12:00 p.m. ET on Tuesday, March 12, 2018. To hear this recording, dial 855-859-2056 (domestic) or 404-537-3406 (international) using the passcode 5975717.

On February 25, 2019 Gamida Cell Ltd. (Nasdaq:GMDA), a leading cellular and immune therapeutics company, 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 (Press release, Gamida Cell, FEB 25, 2019, View Source [SID1234533637]).

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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.

On February 25, 2019 Magenta Therapeutics (NASDAQ:MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, reported that the Company highlighted preclinical research on its targeted conditioning programs in four presentations and posters at the Transplant and Cellular Therapy (TCT) annual meeting (Press release, Magenta Therapeutics, FEB 25, 2019, View Source [SID1234533636]).

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Patients currently undergoing stem cell transplant or stem cell gene therapy are first prepared, or conditioned, with non-specific, genotoxic chemotherapy alone or in combination with total body irradiation. This process is associated with significant side effects, including infertility and mortality risks, which can prevent many eligible patients from undergoing a stem cell transplant or gene therapy. Magenta is developing a portfolio of targeted antibody drug conjugates (ADCs), including non-genotoxic agents, that selectively remove the specific cells needed to enable a successful transplant or gene therapy procedure without the toxicities of chemotherapy and radiation. Magenta’s C100 program targets CD45, expressed on both stem and immune cells, with the goal of enabling stem cell transplant in blood cancer and autoimmune diseases. Magenta’s C200 program targets CD117, expressed on stem cells, with potential applicability as a conditioning agent before gene therapy for genetic diseases and stem cell transplant for blood cancers.

"Stem cell transplant and gene therapy are potential cures for many diseases, including sickle cell disease, blood cancers, and autoimmune diseases, such as multiple sclerosis and systemic sclerosis. However, the toxicity of the current conditioning regimens prevents a significant percentage of patients from considering a transplant or gene therapy and requires the patients who do undergo the procedure to trade their disease for the prospect of infertility, organ damage, secondary cancers and even death," said Michael Cooke, Ph.D., Chief Scientific Officer, Magenta Therapeutics. "Data in non-human primates from Magenta’s two distinct lead conditioning programs show potent and selective depletion of the target cells, and the ADCs were well tolerated. Based on these promising data, we selected a development candidate for the C200 program and have moved into IND-enabling studies. We expect to declare a candidate for the C100 program this year and move into IND-enabling studies in 2020."

Non-Genotoxic Conditioning Using Amanitin Antibody-Drug Conjugates Targeting CD45 Effectively Deplete Human and Non-Human Primate Hematopoietic Stem Cells and Immune Cells (C100 Program)

Key results, presented by Rahul Palchaudhuri, Ph.D., Magenta Therapeutics:

A single dose of an anti-CD45 ADC achieved efficient depletion of immune cells in the periphery and hematopoietic stem cells in the bone marrow in preclinical models.
An anti-CD45 amanitin ADC with engineered fast half-life demonstrated potent depletion of peripheral immune cells as well as immune cells and hematopoietic stem cells in the bone marrow of non-human primates.
An anti-CD45 amanitin ADC with engineered fast half-life demonstrated potent stem and immune cell depletion and rapid clearance, providing optimal pharmacokinetics for patient preparation for stem cell transplant.
These ADCs were well tolerated at the efficacious doses.
Simultaneous depletion of immune and hematopoietic stem cells using an anti-CD45 ADC with rapid clearance may enable safer conditioning for allogeneic transplant and enable an immune-reset in autoimmune disease transplantation, broadening patient access to this potentially curative therapy.
Magenta plans to optimize the anti-CD45 ADC and select a development candidate in 2019, with IND-enabling studies to begin in 2020.
Magenta is testing anti-CD45 ADCs in preclinical models of autoimmune disease, with data expected later in 2019.
A CD117-Amanitin Antibody Drug Conjugate Effectively Depletes Human and Non-Human Primate Hematopoietic Stem and Progenitor Cells: Targeted Non-Genotoxic Conditioning for Bone Marrow Transplant (C200 Program)

Key results, presented by Brad Pearse, Ph.D., Magenta Therapeutics:

An anti-CD117 ADC conjugated with amanitin potently depleted both human and non-human primate hematopoietic stem cells and progenitors in vivo.
An anti-CD117 amanitin ADC with engineered fast half-life demonstrated potent stem cell depletion and rapid clearance, representing optimal pharmacokinetics and pharmacodynamics for patient preparation for stem cell transplant or gene therapy.
The ADCs were well tolerated at the efficacious doses.
Potent and selective depletion of stem cells with rapid clearance of the ADC has the potential to provide a significant improvement over current approaches to patient preparation prior to stem cell transplant and gene therapies, broadening patient access to curative therapies.
Magenta has declared a lead for development for the C200 program and moved into IND-enabling studies.
Magenta is undertaking studies of C200 in preclinical models for gene therapy conditioning, with data expected later this year.
Single Doses of Antibody Drug Conjugates (ADCs) Targeted to CD117 or CD45 Have Potent In Vivo Anti-Leukemia Activity and Survival Benefit in Patient-Derived AML Models

Key results, presented by Jennifer Proctor, Magenta Therapeutics:

CD117 is expressed on human hematopoietic stem and progenitor cells and on leukemia cells in 80% of patients with acute myeloid leukemia (AML) and in 65% of patients with myelodysplastic syndromes (MDS); CD45 is expressed on all lympho-hematopoietic cells in many blood cancers.
Both the anti-CD117 amanitin ADC and the anti-CD45 amanitin ADC demonstrated potent killing of human hematopoietic stem cells and leukemia cell lines in vitro.
A single dose of either ADC demonstrated potent in vivo anti-leukemia activity in mice bearing established human leukemia cell lines.
Both ADCs significantly improved the survival of mice engrafted with human leukemia cells from AML patients, which included leukemias that were resistant to multiple lines of therapy.
Magenta Therapeutics’ C200 and C100 programs are designed with the dual intent of selectively eliminating the necessary cells to enable a successful transplant and reducing disease burden.
Magenta continues to progress ADC-based conditioning approaches targeting CD45 and CD117 toward the clinic.
CD45-Targeted Antibody-Drug Conjugate Plus Post-Transplant Cytoxan is Sufficient to Enable Allogeneic Bone Marrow Transplant in a Minor Mismatch Mouse Model (C100 Program)

Key results, presented by Sharon Hyzy, M.S., Magenta Therapeutics:

ADCs targeted to mouse CD45 have been shown to effectively condition immunocompetent mice for autologous stem cell transplant.
To investigate the utility of a murine-specific tool ADC conjugated to saporin to enable allogenic transplant, Magenta assessed this ADC as a single agent or in combination with immunosuppressive agents to facilitate transplant in a murine allogeneic transplant model.
A single dose of anti-CD45 saporin ADC, when combined with post-transplant cyclophosphamide to prevent graft-versus-host-disease, enabled successful engraftment across minor histocompatibility antigens.
The anti-CD45 saporin ADC was more effective than an unconjugated anti-CD45 antibody, pre-transplant cyclophosphamide, or sublethal irradiation in combination with post-transplant cyclophosphamide.
Magenta continues to investigate additional linker-toxins as well as ADC-based conditioning in multiple allogeneic mouse models.