On March 2, 2020 KIYATEC, Inc. reported that VA Portland Health Care System (VAPORHCS) has joined the growing roster of institutions participating in its 3D-PREDICT clinical study to validate the company’s test as a patient-specific predictor of response to cancer therapies for patients with glioblastoma, an aggressive form of brain cancer (Press release, KIYATEC, MAR 2, 2020, View Source [SID1234555075]).

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"Glioblastoma, one of the most common and deadly forms of brain cancer, is notoriously difficult to treat. Patients and clinicians do not have the luxury of time to experiment with drug therapies they don’t know will work, so we developed our investigational glioblastoma drug response profile to help clinicians optimize therapeutic decision-making for their patients over the course of their disease," said Matthew Gevaert, CEO of KIYATEC. "We are honored to welcome VAPORHCS to the 3D-PREDICT study and salute the important commitment to cancer clinical studies demonstrated by all VA hospitals."

3D-PREDICT is a prospective, open-label, non-interventional study to validate KIYATEC’s glioblastoma drug response profile, which leverages the company’s ex vivo 3D cell culture technology platform to assess pre-treatment, patient-specific response to a panel of 12 drugs most commonly used to treat patients with newly diagnosed or recurrent glioblastoma. Details on the study can be found at View Source

On March 2, 2020 Gilead Sciences, Inc. (Nasdaq: GILD) and Forty Seven, Inc. (Nasdaq: FTSV) reported that the companies have entered into a definitive agreement pursuant to which Gilead will acquire Forty Seven for $95.50 per share in cash (Press release, Gilead Sciences, MAR 2, 2020, View Source [SID1234555015]). The transaction, which values Forty Seven at approximately $4.9 billion, was unanimously approved by both the Gilead and Forty Seven Boards of Directors and is anticipated to close during the second quarter of 2020, subject to regulatory approvals and other customary closing conditions.

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Through the addition of Forty Seven’s investigational lead product candidate, magrolimab, the acquisition will strengthen Gilead’s immuno-oncology research and development portfolio. Magrolimab is a monoclonal antibody in clinical development for the treatment of several cancers for which new, transformative medicines are urgently needed, including myelodysplastic syndrome (MDS), acute myeloid leukemia (AML) and diffuse large B-cell lymphoma (DLBCL). The investigational therapy targets CD47, a "do not eat me" signal that allows cancer cells to avoid destruction thereby permitting the patient’s own innate immune system to engulf and eradicate those cancer cells. Forty Seven presented promising results of a Phase 1b study of magrolimab in patients with MDS and AML at the American Society of Hematology (ASH) (Free ASH Whitepaper) meeting in December 2019. Magrolimab has the potential to be a first-in-class therapy.

"This agreement builds on Gilead’s presence in immuno-oncology and adds significant potential to our clinical pipeline," said Daniel O’Day, Chairman and Chief Executive Officer of Gilead Sciences. "Magrolimab complements our existing work in hematology, adding a non-cell therapy program that complements Kite’s pipeline of cell therapies for hematological cancers. With a profile that lends itself to combination therapies, magrolimab could potentially have transformative benefits for a range of tumor types. We are looking forward to working with the highly experienced team at Forty Seven to help patients with some of the most challenging forms of cancer."

"This is an exciting day for patients who may one day benefit from future anti-CD47 therapies and other immuno-oncology treatments based on our research and an exciting time for Forty Seven as this allows us to achieve our vision of helping patients defeat their cancer," commented Mark McCamish, MD, PhD, President and Chief Executive Officer of Forty Seven. "We are pleased to join Gilead and believe that by combining our scientific expertise with Gilead’s strength in developing treatments that modify the immune system, we will be able to more rapidly advance our therapies."

Magrolimab

Forty Seven is initially studying magrolimab in patients with MDS and AML. Additional studies are ongoing in non-Hodgkin lymphoma (NHL) and solid tumors. Magrolimab has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of MDS and AML, and for the treatment of relapsed or refractory DLBCL and follicular lymphoma, two forms of B-cell NHL. Magrolimab has also been granted Orphan Drug designation by the FDA for the treatment of MDS and AML and by the European Medicines Agency for the treatment of AML.

More than 400 patients have received the compound to date through clinical trials.

Ongoing Phase 1b Clinical Trial

In December 2019, Forty Seven presented promising results of a Phase 1b trial evaluating magrolimab in combination with azacitidine in untreated patients with higher risk MDS and untreated patients with AML, who are ineligible for induction chemotherapy. This has led to the initiation of a potential registrational cohort in MDS. All patients received a 1 mg/kg priming dose of magrolimab, coupled with intrapatient dose escalation to mitigate on-target anemia. Patients were then treated with full doses of azacitidine and magrolimab maintenance doses of 30 mg/kg weekly.

As of the data cutoff of November 18, 2019, 62 patients had been treated with the combination in the Phase 1b portion of the trial, including 35 patients with MDS and 27 patients with AML.

Clinical Activity Data

As of the data cutoff, 46 patients were evaluable for response assessment, including 24 patients with untreated higher-risk MDS and 22 patients with untreated AML, who were ineligible for induction chemotherapy.

In higher-risk MDS, the overall response rate (ORR) was 92 percent, with 12 patients (50 percent) achieving a complete response (CR), eight patients (33 percent) achieving a marrow CR and two patients (8 percent) achieving hematologic improvement. Two patients (8 percent) had stable disease.
In untreated AML, the ORR was 64 percent, with nine patients (41 percent) achieving a CR, three patients (14 percent) achieving a CR with complete blood count recovery (CRi) and one patient (5 percent) achieving a morphologic leukemia-free state (MLFS). Seven patients (32 percent) had stable disease and one patient (5 percent) had progressive disease.
The median time to response among MDS and AML patients treated with the combination was 1.9 months.
Median duration of response and median overall survival have not been reached for either MDS or AML patients, with a median follow-up of 6.4 months (range 2.0 to 14.4 months) for MDS and 8.8 months (range 1.9 to 16.9 months) for AML.
Safety Data

As of the data cutoff, the combination of magrolimab and azacitidine was well-tolerated, with no evidence of increased toxicities compared to azacitidine alone. Adverse events (AEs) were consistent with prior clinical experience. No deaths were observed in the first 60 days on combination treatment and only one patient out of 62 (1.6 percent) discontinued treatment due to a treatment-related AE.

Additional Programs

Beyond magrolimab, Forty Seven is preparing to advance two additional investigational compounds into clinical testing. FSI-174, an anti-cKIT antibody, is being developed in combination with magrolimab as a novel, all-antibody conditioning regimen to address the limitations of current stem cell transplantation conditioning regimens. FSI-189, an anti-SIRPα antibody, is being developed for the treatment of cancer, as well as certain non-oncology settings, including transplantation conditioning.

Terms of the Transaction

Under the terms of the merger agreement, a wholly-owned subsidiary of Gilead will promptly commence a tender offer to acquire all of the outstanding shares of Forty Seven’s common stock at a price of $95.50 per share in cash. Following successful completion of the tender offer, Gilead will acquire all remaining shares not tendered in the offer through a second step merger at the same price as in the tender offer.

Consummation of the tender offer is subject to a minimum tender of at least a majority of outstanding Forty Seven shares plus Forty Seven shares underlying vested options, the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act and other customary conditions.

Gilead plans to pay all cash consideration for the transaction. The tender offer is not subject to a financing condition.

Citi and J.P. Morgan are acting as joint financial advisors to Gilead. Centerview Partners LLC is acting as the exclusive financial advisor to Forty Seven. Skadden, Arps, Slate, Meagher & Flom LLP is serving as legal counsel to Gilead and Cooley LLP is serving as legal counsel to Forty Seven.

Conference Call

At 8:00 a.m. Eastern Time today, Gilead’s management will host a conference call and a simultaneous webcast to discuss the transaction. A live webcast of the call can be accessed at Gilead’s Investors page at View Source Please connect to the website at least 15 minutes prior to the start of the call to allow adequate time for any software download that may be required. Alternatively, please call 877-359-9508 (U.S.) or 224-357-2393 (international) and dial the conference ID 8479332 to access the call.

Telephone replay will be available approximately two hours after the call through 8:30 a.m. Eastern Time, March 4, 2020. To access the replay, please call 855-859-2056 (U.S.) or 404-537-3406 (international) and dial the conference ID 8479332. The webcast will be archived on www.gilead.com for one year.

On March 2, 2020 Marker Therapeutics, Inc. (NASDAQ:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported that the Company has entered into a Common Stock Purchase Agreement (the "Agreement") of up to $30 million with Aspire Capital Fund, LLC ("Aspire"), a Chicago-based institutional investor and long-term Marker shareholder (Press release, TapImmune, MAR 2, 2020, View Source [SID1234555044]).

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Under the terms of the Agreement, Aspire has committed to purchase up to $30 million of the Company’s common stock at Marker’s discretion from time to time during a 30-month period at prices based on the market price at the time of each sale. Marker will retain full control as to the timing and amount of any sale of shares of common stock to Aspire, subject to certain limitations specified in the Agreement.

There are no warrants, options, financing swaps, derivatives or other securities associated with this Agreement. Additionally, there are no financial covenants or restrictions on future financings and there are no rights of first refusal, participation rights, penalties or liquidated damages. Lastly, Marker maintains the right to terminate the Agreement at any time, at its discretion, without any additional cost or penalty. Proceeds from the Agreement will be used to further advance the Company’s pipeline including its post-transplant acute myeloid leukemia (AML) Phase 2 trial, which is expected to begin in 2020, as well as for general corporate purposes.

"This Agreement with Aspire provides Marker with the opportunity to access capital in an efficient manner," stated Peter L. Hoang, President and CEO of Marker. "The financial flexibility provided by this transaction will further support the advancement of our clinical programs including the first Marker-sponsored clinical trial this year investigating our novel MultiTAA cell therapy."

As consideration for Aspire’s entering into the Agreement, Marker issued 345,357 shares to Aspire as a commitment fee. Additional detail regarding the Agreement is set forth in Marker’s Current Report on Form 8-K, filed today with the SEC.

On March 2, 2020 Ziopharm Oncology, Inc. ("Ziopharm" or the "Company") (Nasdaq: ZIOP), reported its financial results for the fourth quarter and year ended December 31, 2019 and provided a corporate update (Press release, Ziopharm, MAR 2, 2020, View Source [SID1234555060]).

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"In the 16 months since forging our corporate independence, we have advanced all our clinical programs, licensed critical intellectual property, recruited key personnel to the Company and Board, and fortified our financial position," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer. "In 2020, we will use this foundation to accelerate our commercial pathway, through enrollment at NCI to TCR-T trial, preparing TCR-T trials with MD Anderson Cancer Center, data readouts on Controlled IL-12 in recurrent GBM and enrollment to trial of CAR-T infused day after gene transfer."

Significant 2019 Achievements

NCI Phase 2 TCR Study. Under the T-cell receptor (TCR) T-cell therapy program, an investigational new drug (IND) application submitted by the National Cancer Institute (NCI) received clearance from the U.S. Food and Drug Administration (FDA) for a phase 2 clinical trial in multiple solid tumors to evaluate use of the Sleeping Beauty platform for TCR-T therapy.

This trial represents a first-in-human non-viral TCR-T trial at NCI

NCI has begun to screen patients for enrollment with patients going through the TCR identification and procurement process; with tumor resection, neoantigen identification, and TCRs made ready for infusion

Study protocol details are available on Clinicaltrials.gov: A Phase 2 Study Using the Administration of Autologous T-Cells Engineered Using the Sleeping Beauty Transposon/Transposase System to Express T-Cell Receptors Reactive Against Mutated Neoantigens in Patients With Metastatic Cancer (NCT0402436)

New MD Anderson Agreement. In October, the Company entered into a lease agreement to expand its R&D footprint and entered into a new Research and Development Agreement with MD Anderson Cancer Center which:

Enables Ziopharm-sponsored clinical studies at MD Anderson based on the Sleeping Beauty non-viral gene transfer platform to generate T cells targeting neoantigens in solid tumors; initially, two approaches will be evaluated infusing autologous T cells expressing

TCRs with specificities for multiple private neoantigens and administering autologous T cells expressing TCRs from a library targeting shared neoantigens in hotspots

Accelerates expansion of the library, licensed from NCI earlier in 2019, of TCRs against neoantigens in hotspots including mutated KRAS, TP53 and EGFR

Phase 2 Combination Study for Controlled IL-12. Under the Controlled IL-12 program, the Company initiated a phase 2 combination trial with Regeneron’s Libtayo to treat patients with recurrent glioblastoma (rGBM).

As prescribed in the study protocol, the Company expects to enroll up to 36 patients at approximately 10 sites. Enrollment is anticipated to be completed in 1H 2020.

This phase 2 trial builds on experience from a phase 1 combination study with another PD-1 inhibitor, OPDIVO, which completed enrollment last year, with additional data expected this year.

Controlled IL-12 Clinical Data. Data publications in 2019 at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and Society for Neuro-Oncology (SNO) annual meetings showed Controlled IL-12, as monotherapy or in combination with a PD-1 inhibitor, resulted in immune-mediated anti-tumor effects in the setting of recurrent GBM. Encouraging results from the phase 1 monotherapy main trial were published in Science Translational Medicine. These publications and presentations are available on Ziopharm’s website.

IND Cleared for CAR-T Study. Under the CAR-T program, an IND was cleared by the FDA for a phase 1 clinical trial to assess CD19-specific CAR-T, produced using Rapid Personalized Manufacturing (RPM).

Up to 24 patients will be enrolled to evaluate infusion of donor-derived RPM CAR-T in patients with CD19+ leukemias and lymphomas who have relapsed after allogeneic bone marrow transplant. This study is being conducted at MD Anderson Cancer Center as an investigator-initiated trial.

The Company also expects partners at Eden BioCell to file an IND for an autologous RPM CD19 trial this year in Taiwan.

Expanded Team and Capabilities. The Company experienced significant growth in 2019, with strategic hires expanding capabilities and increasing staff nearly 50 percent to 73 employees at year end.

Notably, Ziopharm welcomed Sath Shukla, formerly of Vertex Pharmaceuticals, as Chief Financial Officer and Dr. Drew Deniger, who previously worked under Dr. Steven Rosenberg at the NCI, to lead the TCR program.

Chris Bowden, M.D., and Heidi Hagen were appointed as Directors, completing the repopulation of the Board as part of Ziopharm’s reemergence as an independent company.

Strengthened Balance Sheet. Ziopharm completed 2019 with approximately $79.7 million in cash, with another $20.3 million in capital pre-funded at MD Anderson available for the Company’s program.

Subsequent to the close of 2019, the Company raised approximately an additional $98 million through recent financing activities, Ziopharm has dramatically extended its funding horizon and can accelerate the buildout of its TCR program in Houston and the launch of Ziopharm-led TCR clinical trials for patients with solid tumors.

Anticipated Milestones for the First Half of 2020

Sleeping Beauty Cell Therapy Programs

Patient dosing in the NCI-led phase 2 TCR-T trial targeting solid tumors

Initiation of the CD19-specific CAR-T RPM phase 1 trial with membrane bound IL-15 at MD Anderson

Controlled IL-12 Gene Therapy Program

Complete enrollment and initial data readout for phase 2 combination trial with Libtayo

Interim data readout of phase 1 combination trial with OPDIVO

Interim data readout from phase 1 monotherapy trial in expanded cohort

Fourth Quarter 2019 Financial Results

Net loss of the fourth quarter of 2019, was $15.7 million, or $(0.09) per share, compared to net income of $194.5 million, or $1.29 per share, for the fourth quarter of 2018. Net income for the fourth quarter of 2018 reflects the forfeiture and return of all the Company’s Series 1 preferred stock held by a former corporate partner and the relinquishment of Ziopharm’s obligations under a separate agreement, accounting for approximately $207 million.

Research and development expenses were $10.2 million for the fourth quarter of 2019, compared to $8.2 million for the fourth quarter of 2018, primarily reflecting increased clinical trial activity.

General and administrative expenses were $5.8 million for the fourth quarter of 2019, compared to $4.6 million for the fourth quarter of 2018. The increase in general and administrative expenses for the fourth quarter of 2019 is primarily due to increased headcount, growth of intellectual property activity and expanded clinical activity.

The Company ended the fourth quarter 2019 with unrestricted cash resources of approximately $79.7 million.

In addition, a prepayment of approximately $20.3 million remains for work to be conducted by the Company at MD Anderson under the Company’s Research and Development Agreements.

Full Year 2019 Financial Results

Net loss applicable to the common shareholders for the year ended December 31, 2019 was $117.8 million, or $(0.70) per share, basic and diluted, compared to net income applicable to the common shareholders of $137.2 million, or $0.96 per share, basic and diluted, for the year ended December 31, 2018. Net income in 2018 reflects the forfeiture and return of all of the Company’s Series 1 preferred stock held by a former corporate partner and the relinquishment of Ziopharm’s obligations under a separate agreement, accounting for approximately $207 million.

Research and development expenses were $38.3 million for the year ended December 31, 2019, compared to $34.1 million for the year ended December 31, 2018. The increase in research and development expenses for the year ended December 31, 2019 is primarily due to expanded clinical trial activity.

General and administrative expenses were $19.5 million for the year ended December 31, 2019, compared to $19.9 million for the year ended December 31, 2018.

Conference Call and Webcast

Scheduled for today at 4:30 p.m. ET, the conference call can be accessed by dialing 1-844-309-0618 (U.S. and Canada) or 1-661-378-9465 (international). The passcode for the conference call is 6773016. To access the live webcast or the subsequent archived recording, visit the "Investors" section of the Ziopharm website at

www.ziopharm.com. The webcast will be recorded and available for replay on the Company’s website for two weeks.

On March 2, 2020 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to finding cures for blood cancers and serious blood diseases, reported that updated results from a Phase 1 clinical study of GDA-201, an investigational, natural killer (NK) cell-based cancer immunotherapy for the treatment of patients with non-Hodgkin lymphoma and multiple myeloma, will be presented during an oral session at the 46th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT), which is being held March 22 through March 25, 2020, in Madrid, Spain (Press release, Gamida Cell, MAR 2, 2020, View Source [SID1234555076]).

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Details about the presentation are as follows:

Time: Wednesday, March 25, 2020, at 11:00 a.m. CET
Abstract Number: OS22-1
Title: Results of a Phase 1 Trial of GDA-201, Nicotinamide-Expanded Allogeneic Natural Killer Cells (NAM-NK) in Patients with Refractory Non-Hodgkin Lymphoma (NHL) and Multiple Myeloma (MM)
Lead Author: Veronika Bachanova, M.D., Ph.D., Division of Hematology, Oncology and Transplantation, University of Minnesota, Minneapolis, MN
Location: Room N118

About GDA-201
Gamida Cell applied the capabilities of its NAM-based cell expansion technology to develop GDA-201, 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.1

GDA-201 is an investigational therapy, and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.