On May 3, 2018 Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), reported financial results for the three months ended March 31, 2018 and highlighted progress with the company’s first commercial product, XERMELO (telotristat ethyl), its pipeline drug candidates and its overall business (Press release, Lexicon Pharmaceuticals, MAY 3, 2018, View Source;2018.htm [SID1234526077]). The company will conduct a conference call and webcast today at 8:00 am EDT / 7:00 am CDT to discuss the financial results and to provide a business update.

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"Although we are treating more people affected with carcinoid syndrome diarrhea than ever before, we still have much work to do," said Lonnel Coats, Lexicon’s president and chief executive officer. "We continue to position the company for future growth and to build long-term sustainable value for shareholders. As such, we are excited about exploring the investigational use of telotristat ethyl in oncology indications this year. For sotagliflozin, we are a step closer to making the therapy available to people with type 1 diabetes, with the recent regulatory submissions in the U.S. and in Europe and the European Medicines Agency’s acceptance of the filing in Europe. Finally, we look forward to reporting clinical data later this year from our earlier-stage product candidates, LX2761 and LX9211, in diabetes and neuropathic pain, respectively, which we believe will create long-term value for the company."

First Quarter Product and Pipeline Highlights

XERMELO (telotristat ethyl) 250 mg

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Data from a poster highlighting time to sustained improvement in bowel movement frequency with XERMELO were presented at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO GI; San Francisco) in January.

Sotagliflozin

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Lexicon’s collaborator Sanofi submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in March for sotagliflozin, for use in combination with insulin therapy to improve glycemic control in adults with type 1 diabetes mellitus.
•
Sanofi submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in March for sotagliflozin, for use in combination with insulin therapy to improve glycemic control in adults with type 1 diabetes mellitus, and the review of the MAA under the Centralized Procedure began on March 29.

LX2761

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A Phase 1b clinical trial of LX2761, an orally administered drug candidate selectively targeted to inhibit SGLT1 (sodium-glucose cotransporter type 1) in the gastrointestinal tract, remains ongoing in people with type 2 diabetes.

LX9211

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A Phase 1a clinical trial of LX9211, an orally-administered drug candidate selectively targeted to inhibit AAK1 (adapter-associated kinase 1), remains ongoing in healthy volunteers.

First Quarter 2018 Financial Highlights

Revenues: Revenues for the first quarter of 2018 increased 38% to $25.2 million from $18.3 million for the corresponding period in 2017, primarily due to an increase in net product revenues recognized from the sale of XERMELO in the U.S. to $5.4 million from $0.7 million and increased revenue from collaborative agreements.

Cost of Sales: Cost of sales related to sales of XERMELO for the first quarter of 2018 increased 137% to $0.5 million from $0.2 million for the corresponding period in 2017.

Research and Development (R&D) Expenses: Research and development expenses increased 10% to $47.8 million for the first quarter of 2018 from $43.6 million for the corresponding period in 2017, primarily due to higher external clinical development expenses relating in substantial part to development of sotafliflozin in type 2 diabetes and professional and consulting fees related to sotagliflozin NDA preparation.

Selling, General and Administrative (SG&A) Expenses: Selling, general and administrative expenses for the first quarter of 2018 were flat year-over-year at $14.9 million.

Consolidated Net Loss: Net loss for the first quarter of 2018 was $42.1 million, or $0.40 per share, compared to a net loss of $34.9 million, or $0.33 per share, in the corresponding period in 2017. For the first quarter 2018 and 2017, net loss included non-cash, stock-based compensation expense of $3.1 million and $2.2 million, respectively.

Cash and Investments: As of March 31, 2018, Lexicon had $262.3 million in cash and investments, as compared to $310.8 million as of December 31, 2017.

Anticipated Upcoming Milestones

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2Q 2018 – Initiation of additional Phase 3 sotagliflozin study in type 2 diabetes by Sanofi
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3Q 2018 – Phase 1b data for LX2761 in type 2 diabetes
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June 2018 – Sotagliflozin data presentations at the American Diabetes Association 78th Scientific Sessions (ADA; June 22-26, 2018; Orlando, FL)
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October 2018 – Sotagliflozin data presentations at the 54th Annual Meeting of the European Association for the Study of Diabetes (EASD; October 1-5, 2018; Berlin, Germany)
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2H 2018 – Initiation of clinical studies for telotristat ethyl in neuroendocrine tumors and bilary tract cancers
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2H 2018 – Phase 1a data for LX9211 in neuropathic pain
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2018 – Manuscript publications for XERMELO and sotagliflozin
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2018 – Launch of XERMELO by Ipsen in additional European countries

Conference Call and Webcast Information

Lexicon management will hold a live conference call and webcast today at 8:00 am EDT / 7:00 am CDT to review its financial and operating results and to provide a general business update. The dial-in number for the conference call is 888-645-5785 (U.S./Canada) or 970-300-1531 (international). The conference ID for all callers is 3191269. The live webcast and replay may be accessed by visiting Lexicon’s website at www.lexpharma.com/investors. An archived version of the webcast will be available on the website for 14 days.

About XERMELO (telotristat ethyl)

Discovered using Lexicon’s unique approach to gene science, XERMELO (telostristat ethyl) is the first and only approved oral therapy for carcinoid syndrome diarrhea in combination with somatostatin analog (SSA) therapy in adults inadequately controlled by SSAs. XERMELO targets tryptophan hydroxylase, an enzyme that mediates the excess serotonin production within metastatic neuroendocrine tumor (mNET) cells. Lexicon has built the in-house

capability and infrastructure to launch and market XERMELO in the U.S., where it retains all commercialization rights. Lexicon also retains rights to market XERMELO in Japan. Lexicon has established a license and collaboration agreement with Ipsen to commercialize XERMELO in Europe and other countries outside of U.S. and Japan.

XERMELO was approved by the U.S. Food and Drug Administration on February 28, 2017 and by the European Commission on September 19, 2017 for the treatment of carcinoid syndrome diarrhea in combination with SSA therapy in adults inadequately controlled by SSA therapy. Carcinoid syndrome is a rare condition that occurs in patients living with metastatic NETs (mNETs) and is characterized by frequent and debilitating diarrhea. XERMELO targets the overproduction of serotonin inside mNET cells, providing an additional treatment option for patients suffering from carcinoid syndrome diarrhea.

XERMELO (telotristat ethyl) Important Safety Information

Warnings and Precautions: XERMELO may cause constipation, which can be serious. Monitor for signs and symptoms of constipation and/or severe, persistent, or worsening abdominal pain in patients taking XERMELO. Discontinue XERMELO if severe constipation or severe, persistent, or worsening abdominal pain develops.

Adverse Reactions: The most common adverse reactions (≥5%) include nausea, headache, increased gamma-glutamyl-transferase, depression, flatulence, decreased appetite, peripheral edema, and pyrexia.

Drug Interactions: If necessary, consider increasing the dose of concomitant CYP3A4 substrates, as XERMELO may decrease their systemic exposure. If combination treatment with XERMELO and short-acting octreotide is needed, administer short-acting octreotide at least 30 minutes after administering XERMELO.

For more information about XERMELO, see Full Prescribing Information at www.xermelo.com.

On May 3, 2018 Verastem, Inc. (NASDAQ:VSTM) (Verastem Oncology or the Company), focused on developing and commercializing drugs to improve the survival and quality of life of cancer patients, reported financial results for the quarter ended March 31, 2018 and provided an overview of certain corporate developments (Press release, Verastem, MAY 3, 2018, View Source;p=RssLanding&cat=news&id=2346916 [SID1234526096]).

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"We’ve had a strong start to 2018, highlighted foremost by the acceptance of our duvelisib New Drug Application (NDA) by the U.S. Food and Drug Administration (FDA), including the receipt of Priority Review with an assigned target action date of October 5, 2018. This is exciting news for patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) and follicular lymphoma (FL)," said Robert Forrester, President and Chief Executive Officer of Verastem Oncology. "As we await the upcoming FDA review decision for duvelisib, we are building our U.S. commercial capabilities for our potential product launch in 2018. We are assembling a world-class commercial team, led by our Chief Commercial Officer, Joseph Lobacki, the former Chief Commercial Officer of Medivation, who has demonstrated success in commercializing oncology drugs."

Mr. Forrester added, "Yesterday, at our Analyst and Investor Day event in New York City, several key opinion leaders in the hematologic oncology field, including Drs. Jennifer Brown, Ian Flinn, Steven Horwitz, Brian Koffman and Lori Kunkel, joined Mr. Lobacki for an in-depth discussion regarding the unmet need among CLL/SLL and FL patients, where phosphoinositide-3-kinase (PI3K) inhibitors fit into the treatment paradigm, and the growing opportunity for duvelisib in CLL/SLL and FL, and beyond."

First Quarter 2018 and Recent Highlights:

Duvelisib

Duvelisib NDA Accepted by FDA with Priority Review – In April 2018, Verastem Oncology announced that the FDA accepted the duvelisib NDA for filing with Priority Review, with a target action date of October 5, 2018. In the accepted NDA, the Company is seeking full approval for duvelisib, its first-in-class investigational oral dual inhibitor of PI3K-delta and PI3K-gamma, for the treatment of relapsed or refractory CLL/SLL and accelerated approval for the treatment of relapsed or refractory FL. The duvelisib NDA is supported by clinical data from the randomized Phase 3 DUO study evaluating duvelisib as a monotherapy in patients with relapsed or refractory CLL/SLL, as well as the Phase 2 DYNAMO study evaluating patients with indolent non-Hodgkin lymphoma that are double-refractory to both rituximab and chemotherapy or radioimmunotherapy. Both DUO and DYNAMO achieved their primary endpoints.
Hosted Analyst and Investor Day Highlighting Commercial Potential of Duvelisib – In early May 2018, Verastem Oncology hosted an Analyst and Investor Day in New York City titled, "Duvelisib: Harnessing the Power of Dual PI3K Inhibition." Several key opinion leaders in the hematologic oncology field joined the Verastem Oncology executive leadership team for an in-depth discussion regarding the unmet need among CLL/SLL and FL patients, where PI3K-delta and PI3K-gamma inhibitors fit into the treatment paradigm, and the growing opportunity for duvelisib in CLL/SLL and FL, and beyond. The Company also provided an overview of its duvelisib commercial strategy and initiatives. An archived webcast of the event is available on the "Events & Presentations" page in the "Investors" section of the Company’s website at www.verastem.com.
Preclinical Duvelisib Data in Combination with Immunotherapy Presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Clinical Immuno-Oncology Symposium (ASCO-SITC) – In January 2018, the Company presented a poster at ASCO (Free ASCO Whitepaper)-SITC entitled "Dual PI3K-δ,γ Inhibitor Duvelisib Reduces Immunosuppressive Tregs and Myeloid Cells, Enhancing Efficacy of Checkpoint and Co-Stimulatory Antibodies in a B Cell Lymphoma Model," which highlighted the potential synergistic effects of duvelisib in combination with immune checkpoint or co-stimulatory antibodies in B-cell lymphoma. The poster is available on the "Publications" page in the "Media" section of the Company’s website at www.verastem.com.
Corporate and Financial

"Verastem Oncology" – In May 2018, the Company announced that it had changed its name to "Verastem Oncology" to reinforce its commitment to developing and commercializing treatment options for patients battling cancer.
Joseph Lobacki Appointed as Chief Commercial Officer – In January 2018, Joseph Lobacki was appointed as Verastem Oncology’s Executive Vice President and Chief Commercial Officer. Mr. Lobacki most recently served as the Chief Commercial Officer and Executive Council Member at Medivation and has previously held senior-level commercial positions at Micromet Inc. and Genzyme Corporation. Mr. Lobacki will lead Verastem Oncology’s commercial strategy and preparation for the potential launch of duvelisib.
Key Leadership Roles Filled – During the first quarter of 2018, Verastem Oncology hired key senior leadership team members in medical affairs, market access and sales management, including the appointment of Dr. Nadeem Mirza, as Vice President of Medical Affairs. Dr. Mirza most recently served as the Global Head Hematology, Global Medical Affairs at AbbVie Oncology.
Increased Debt Facility to up to $50.0 Million – In January 2018, Verastem Oncology amended its loan and security agreement with Hercules Capital, Inc., increasing its existing potential borrowing limit under the loan facility from up to $25.0 million to up to $50.0 million, subject to certain conditions of funding. Any additional proceeds received under the increased loan facility will be used to support the Company’s ongoing development programs, including regulatory and commercialization activities for duvelisib, and for general corporate purposes.
First Quarter 2018 Financial Results

Net loss for the three months ended March 31, 2018 (2018 Quarter) was $21.1 million, or $0.41 per share, as compared to a net loss of $13.0 million, or $0.35 per share, for the three months ended March 31, 2017 (2017 Quarter). Net loss includes non-cash stock-based compensation expense of $1.3 million and $1.2 million for the 2018 Quarter and 2017 Quarter, respectively.

Research and development expense for the 2018 Quarter was $10.9 million compared to $8.4 million for the 2017 Quarter. The $2.5 million increase from the 2017 Quarter to the 2018 Quarter was primarily related to an increase of $1.1 million in contract research organization expense for outsourced biology, development and clinical services, which includes our clinical trial costs, an increase of $0.9 million in personnel related costs, an increase of $0.2 million in stock-based compensation expense, and an increase in consulting fees of $0.2 million.

General and administrative expense for the 2018 Quarter was $9.8 million compared to $4.8 million for the 2017 Quarter. The increase of $5.0 million from the 2017 Quarter to the 2018 Quarter primarily resulted from increases in consulting and professional fees of $2.5 million, including $1.8 million related to commercial launch preparation, and personnel related costs of $2.0 million.

As of March 31, 2018, Verastem Oncology had cash and cash equivalents of $64.2 million compared to $86.7 million of cash, cash equivalents and investments as of December 31, 2017. From April 1, 2018 to May 3, 2018, the Company sold 5,903,073 shares of common stock under its at-the-market equity offering program (ATM) for net proceeds of approximately $21.9 million (after deducting commissions and other offering expenses). Giving effect to these sales under the ATM, the Company’s pro forma cash and cash equivalents balance at March 31, 2018 is approximately $86.1 million.

The number of outstanding common shares as of March 31, 2018, was 50,967,973.

Financial Guidance

Based on the Company’s current operating plans, it expects to have sufficient cash and cash equivalents to fund operations into 2019.

About Duvelisib

Duvelisib is a first-in-class investigational oral, dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells and T-cells. PI3K signaling may lead to the proliferation of malignant B- and T-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.1,2,3 Duvelisib was evaluated in late- and mid-stage extension trials, including DUO, a randomized, Phase 3 monotherapy study in patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL),4 and DYNAMO, a single-arm, Phase 2 monotherapy study in patients with refractory indolent non-Hodgkin lymphoma (iNHL).5 Both DUO and DYNAMO achieved their primary endpoints. Verastem Oncology’s New Drug Application (NDA) requesting the full approval of duvelisib for the treatment of patients with relapsed or refractory CLL/SLL, and accelerated approval for the treatment of patients with relapsed or refractory follicular lymphoma (FL) was accepted for filing by the U.S. Food and Drug Administration (FDA), granted Priority Review and assigned a target action date of October 5, 2018. Duvelisib is also being developed by Verastem Oncology for the treatment of peripheral T-cell lymphoma (PTCL), and is being investigated in combination with other agents through investigator-sponsored studies.6 Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

About Defactinib

Defactinib is an investigational inhibitor of focal adhesion kinase (FAK), a non-receptor tyrosine kinase that mediates oncogenic signaling in response to cellular adhesion and growth factors.7 Based on the multi-faceted roles of FAK, defactinib is used to treat cancer through modulation of the tumor microenvironment and enhancement of anti-tumor immunity.8,9 Defactinib is currently being evaluated in three separate clinical collaborations in combination with immunotherapeutic agents for the treatment of several different cancer types including pancreatic cancer, ovarian cancer, non-small cell lung cancer (NSCLC), and mesothelioma. These studies are combination clinical trials with pembrolizumab and avelumab from Merck & Co. and Pfizer/Merck KGaA, respectively.10,11,12 Information about these and additional clinical trials evaluating the safety and efficacy of defactinib can be found on www.clinicaltrials.gov.

On May 3, 2018 Nicox SA (Euronext Paris: FR0013018124, COX), an international ophthalmology company, reported a poster presentation highlighting scientific data for NCX 667, a novel nitric oxide (NO) donating compound, at the Association for Research in Vision and Ophthalmology (ARVO) 2018 Annual Meeting, one of the key scientific events in the ophthalmology calendar, being held on April 29 – May 3, 2018 in Honolulu, Hawaii, United States.

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Synthesized and characterized by Nicox, NCX 667 is a lead molecule among the Company’s future generation of stand-alone NO-donors which are designed to optimize NO dosing and can be used alone or in combination with existing standard-of-care drugs, as either ophthalmic solutions or extended release formulations, to enable robust intraocular pressure (IOP)-lowering in patients with open-angle glaucoma or ocular hypertension.

The ARVO 2018 abstract by Francesco Impagnatiello, Ph.D., et al. describes results following single dose administration of various doses (0.1%, 0.3% and 1.0% solution) of NCX 667 in several ocular normotensive and ocular hypertensive animal models. These results demonstrate that NCX 667 lowers IOP in a dose-dependent manner. Furthermore, an in vitro bioengineered human trabecular meshwork/Schlemm’s canal (TM/SC) system was used to study the effects of NCX 667 on the conventional outflow facility. These data support the hypothesis that the IOP-lowering effects of NCX 667 are likely due to an increase in outflow facility via the TM/SC outflow pathway.

Michael Bergamini, Ph.D., Executive Vice President, Chief Scientific Officer of Nicox, commented: "The results presented at ARVO this year show a clear, dose-dependent, and meaningful lowering of IOP in both ocular normotensive and hypertensive models. These results, combined with the in vitro data, continue to build upon the growing body of scientific evidence supporting the development of NCX 667, a lead molecule among our future generation of stand-alone NO-donors. We are continuing to generate new compounds in this class and are testing multiple leads using topical and sustained release dosing."

An estimated 3.5% of the worldwide population between 40 and 80 years of age are affected by the most common forms of glaucoma1.

The ARVO 2018 abstracts have been published in the meeting website located at View Source and details for the poster presentation are as follows:

Title: NCX 667, a novel nitric oxide (NO) donor lowers intraocular pressure (IOP) via stimulation of trabecular meshwork/Schlemm’s canal outflow facility

Date and time: Wednesday, May 2, 2018 from 11:15 am to 1:00 pm HAST

Presenter: Francesco Impagnatiello, Ph.D. , Nicox Research Institute

Session n° 448, Title: Glaucoma – Trabecular Meshwork

Abstract n°: 4707 / Poster n°: B0131

Location: Hawaii Convention Center, Exhibit Hall

On May 3, 2018 OncoSec Medical Incorporated (OncoSec) (NASDAQ:ONCS), a company developing intratumoral cancer immunotherapies, reported that Daniel J. O’Connor, Chief Executive Officer of OncoSec, will present at the Eighth Annual BioNJ BioPartnering Conference, developed in partnership with J.P. Morgan, being held Thursday, May 3, 2018 at the The Palace at Somerset Park in Somerset, NJ (Press release, OncoSec Medical, MAY 3, 2018, View Source [SID1234526023]).

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As part of his presentation, Mr. O’Connor will discuss OncoSec’s corporate growth strategy; provide a comprehensive overview of the Company’s ongoing and anticipated clinical programs involving ImmunoPulse IL-12 (or Intratumoral tavo-EP) in metastatic melanoma and triple-negative breast cancer (TNBC); and the potential of ImmunoPulse IL-12 as a monotherapy and in combination with anti-PD-1 antibody therapy in metastatic melanoma and triple-negative breast cancer (TNBC). In addition to the presentation, management will participate in one-on-one meetings with qualified members of the investor community who are registered to attend the conference.

BioNJ’s Eighth Annual BioPartnering Conference, developed in partnership with J.P. Morgan Chase, will bring together nearly 400 public and private company investors, life sciences professionals and academic partners from the Northeast to Mid-Atlantic states. Pharmaceutical sponsors for the conference include The Janssen Pharmaceutical Companies of Johnson & Johnson, Merck, and Pfizer.

On May 3, 2018 Celyad (Euronext Brussels and Paris, and NASDAQ: CYAD) a clinical-stage biopharmaceutical company focused on the development of CAR-T cell therapies, reported the publication later today of a patient case study from the hematological arm of its THINK Phase I trial in the journal Haematologica1 (Press release, Celyad, MAY 3, 2018, View Source [SID1234526052]). The publication, entitled "NKG2D-based Chimeric Antigen Receptor Therapy Induced Remission in a Relapsed/Refractory Acute Myeloid Leukemia Patient" is authored by the trial investigators at the Moffitt Cancer Center and Research Institute in Tampa, Fla. and by Celyad’s scientific team.

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The publication details the first objective response to CAR-T in relapsed/refractory AML using CYAD-01, Celyad’s Natural Killer Group 2D (NKG2D) chimeric antigen receptor T-cell therapy, without pre-conditioning lymphodepletion. The patient received CYAD-01 infusions at the initial dose level of 3×108 cells every 2 weeks for 3 administrations, achieving a morphologic leukemia-free state (MLFS2) at 3-months which enabled the patient to benefit from an allo-hematopoietic stem cell transplant (allo-HSCT). The patient achieved a complete molecular remission and remains in remission 9 months post study enrollment. CYAD-01 was well tolerated with no significant toxicities. The demonstrated first objective response to any CAR-T in relapsed/refractory AML without preconditioning chemotherapy highlights the potential of CYAD-01 as a treatment for AML.

"Our results demonstrate the validity of NKG2D as a target, in particular in the context of refractory AML and without other intervening treatments nor preconditioning", commented Frédéric Lehmann, VP Clinical Development and Medical Affairs at Celyad. "We look forward to continue our clinical development plan for our NKG2D CAR based platform and explore the various conditions within which this therapy could provide benefits to patients with end stage cancers."

Dr. David Sallman, Assistant Member in the Malignant Hematology Department of Moffitt Cancer Center, added: "The THINK study case report provides the first clinical validity of CYAD-01 as a tumor-specific antigen-receptor and AML as a disease sensitive to gene-engineered cell therapies. As antigen targeting offers significant challenges in AML, this outcome brings hope for the further use of gene-engineered T-cells for patients with AML that have run out of therapeutic options. It’s all the more striking that this outcome was observed without any prior lymphodepletion highlighting the potential of using a physiologic antigen-receptor."