On August 8, 2019 Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx, reported its financial results for the six-month period ended June 30, 2019 and provided a corporate update (Press release, Protalix, AUG 8, 2019, View Source;p=RssLanding&cat=news&id=2406197 [SID1234538438]).

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"We are very optimistic about our interactions with the U.S. Food and Drug Administration over the second quarter of 2019, which resulted in our decision to, together with our partner Chiesi Farmaceutici, begin preparing a biologics license application for pegunigalsidase alfa for the treatment of Fabry disease, which we expect to submit in the first quarter of 2020 through the FDA’s Accelerated Approval pathway," said Mr. Dror Bashan, Protalix’s President and Chief Executive Officer. "We are now focused on completing the anticipated filing."

Second Quarter 2019 and Recent Clinical and Corporate Highlights

The Company, together with its collaboration partner, Chiesi Farmaceutici S.p.A, or Chiesi, announced that, following a series of meetings and correspondence with the U.S. Food and Drug Administration (FDA), they plan to file a biologics license application (BLA) for pegunigalsidase alfa, or PRX-102, for the treatment of Fabry disease via the FDA’s Accelerated Approval pathway.

The Company and Chiesi have initiated preparations for the BLA submission based on data from the completed Phase I/II clinical trials of pegunigalsidase alfa and the ongoing Phase III BRIDGE clinical trial, and expect to hold a pre-BLA meeting with the FDA in the fourth quarter of 2019.

Enrollment completed in the Phase III BRIGHT clinical trial of pegunigalsidase alfa for the treatment of Fabry disease, via intravenous (IV) infusions of 2 mg/kg administered every 4 weeks.

To date, substantially all patients enrolled in the BRIGHT clinical trial remain on the 4‑week dosing regimen, and all of the patients that completed the study opted, with the advice of the treating physician, to continue treatment under the 4‑week dosing regimen in a long-term extension study.

The Company’s Phase III BALANCE clinical trial of pegunigalsidase alfa for the treatment of Fabry disease is currently 95% enrolled.

To date, more than 55 patients are being treated in the Company’s various extension studies after opting to continue treatment with pegunigalsidase alfa after completion of an initial study.

Results from the Company’s Phase I/II clinical trial of pegunigalsidase alfa were published in an article in the May 2019 edition of the Journal of Inherited Metabolic Disease.

Financial Results for the Six Months Ended June 30, 2019

The Company recorded total revenues of $22.7 million for the six-month period ended June 30, 2019, compared to $11.6 million for the same period of 2018. The increase is primary attributable to the recognition of $15.7 million of license revenues for the six-month period ended June 30, 2019 compared to the recognition of $5.0 million in the same period of 2018.

Research and development expenses, net were $25.0 million for the six months ended June 30, 2019, compared to $13.7 million for the same period in 2018. The increase is mainly due to advancement of the Phase III studies in PRX-102.

Selling, general and administrative expenses for the six months ended June 30, 2019 were $4.3 million, compared to $4.7 million for the same period in 2018.

Net loss for the six months ended June 30, 2019 was $15.0 million, or $0.10 per share, basic and diluted, compared to a net loss of $15.7 million, or $0.11 per share, basic and diluted, for the six months ended June 30, 2018.

On June 30, 2019, the Company had $25.1 million of cash and cash equivalents.

If the BLA submitted for pegunigalsidase alfa is approved by the FDA, the Company will be entitled to a milestone payment, which is currently expected to be payable around the fourth quarter of 2020 or first quarter of 2021.

Based on the Company’s current cash resources and commitments, the Company believes it may not be able to maintain its current planned development activities and the corresponding level of expenditures for at least 12 months from the date of approval of the financial statements as of June 30, 2019 in the absence of a refinancing or restructuring of its existing obligations. These factors raise substantial doubt as to the Company’s ability to continue as a going concern. Additional information will be included in Note 1(a) and other parts of the Company’s Quarterly Report on Form 10-Q which will be filed the day of the earnings call. The Company is evaluating various financing alternatives and related transactions.

The Company’s management is in the process of evaluating refinancing and restructuring alternatives, including a restructuring of its outstanding convertible notes, and related transactions. However, there is no certainty about the Company’s ability to obtain such funding.
Conference Call and Webcast Information

The Company will host a conference call on Thursday, August 8, 2019, at 8:30 am ET to review the clinical, corporate and financial highlights.

To participate in the conference call, please dial the following numbers prior to the start of the call: United States: +1 (844) 358-6760; International: +1 (478) 219-0004. Conference ID number 1497319.

The conference call will also be broadcast live and available for replay for two weeks on the Company’s website, www.protalix.com, in the Events Calendar of the Investors section. Please access the Company’s website at least 15 minutes ahead of the conference to register, download, and install any necessary audio software.

On August 8, 2019 Physicians’ Education Resource (PER), a worldwide leading resource for continuing medical education (CME), named public health expert and former commissioner of the Food and Drug Administration (FDA) Scott Gottlieb, M.D., as the keynote speaker for the 37th Annual Chemotherapy Foundation Symposium Innovative Cancer Therapy for Tomorrow (Press release, Physicians’ Education Resource, AUG 8, 2019, View Source [SID1234538456]). This year’s keynote presentation will take place on Thursday, Nov. 7 at 11:50 AM, the New York Marriott Marquis in New York City.

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"We are excited to have Dr. Gottlieb as the keynote speaker for this year’s Chemotherapy Foundation Symposium (CFS) annual meeting," said Phil Talamo, president of PER. "Gottlieb is an expert in the field of health care access and public health advocacy, and his legacy at the FDA truly aligns with the meeting’s spirit of Innovative Cancer Therapy for Tomorrow as we pave the way for the future of cancer care."

Gottlieb is a physician and served as the 23rd commissioner of the FDA from 2017-2019. His work focuses on advancing public health through developing and implementing innovative approaches to improving medical outcomes, reshaping health care delivery and expanding consumer choice and safety. Under his leadership, the FDA advanced new frameworks for the modern, safe and effective oversight of gene therapies, cell-based regenerative medicines, targeted drugs and digital health devices.

"With improvements in detection and monitoring, plus the discovery of new therapeutic options, we are changing the way cancer is treated. Now, more than ever, we are creating new advancements for the care of all patients with this disease," said Dr. Gottlieb.

Gottlieb is widely published in leading medical journals and periodicals, including The Wall Street Journal, The New York Times and The Washington Post. Fortune recognized him as one of the World’s 50 Greatest Leaders in 2018 and 2019, and he was named one of Time’s Health Care 50, which celebrates those who have transformed health care, in 2018.

The 37th Annual CFS activity co-chairs — Adam M. Brufsky, M.D., Ph.D.; Benjamin P. Levy, M.D.; and William K. Oh, M.D. — will be joined by 100 world-renowned experts who will provide attendees with expert insights into the latest developments in cancer therapeutics, offering an unparalleled opportunity to learn how innovative approaches fit into existing treatment paradigms to optimize care and outcomes for their patients with cancer.

On August 8, 2019 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 financial results for the second quarter ended June 30, 2019 (Press release, Marker Therapeutics, AUG 8, 2019, View Source [SID1234538472]).

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"We are encouraged by the data generated to date with our MultiTAA T cell therapies—particularly in pancreatic cancer—a disease that has not seen meaningful improvements in treatment outcomes in more than 40 years," said Peter L. Hoang, President & CEO of Marker Therapeutics. "These data suggest that our therapy—which targets multiple antigens—continues to demonstrate epitope spreading, potentially contributing to the T cells’ ability to expand and produce a lasting anti-tumor effect, with no added toxicities. We will continue to follow these patients and enroll new patients to further evaluate durability."

Continued Mr. Hoang: "Encouraged by results from our investigator-sponsored trials, we are now looking forward to initiating our first Marker-sponsored trial with MultiTAA T cell therapies in patients with post-transplant acute myeloid leukemia, and look forward to continuing our discussions with the FDA regarding our Phase 2 trial."

PROGRAM HIGHLIGHTS AND CURRENT UPDATES

Multi-Antigen Targeted (MultiTAA) T Cell Therapies

Encouraging Interim Pancreatic Cancer Data Reported at AACR (Free AACR Whitepaper) Meeting in July
Marker recently reported interim data from an ongoing investigator-sponsored Phase 1/2 clinical trial led by Baylor College of Medicine (BCM), evaluating the Company’s MultiTAA T cell therapy in patients with pancreatic adenocarcinoma. Investigators plan to enroll a total of 45 patients with advanced or borderline resectable pancreatic cancer in the three-arm trial and a total of 19 patients had received infusions of MultiTAA T cell therapy as of July 5, 2019. The results from these patients—which were reviewed in an oral presentation during a plenary session at AACR (Free AACR Whitepaper)’s Cell Therapy meeting in July—suggested that MultiTAA T cell therapy may contribute to more durable responses without added toxicity when used in combination with standard-of-care chemotherapy, or as a second-line therapy for patients who are chemo-refractory. Additionally, in patients with borderline surgically resectable disease—a challenging setting due to the associated dense fibrotic tissue—interim data suggest that MultiTAA T cells are capable of meaningfully infiltrating the tumor. Marker plans to follow these patients and enroll new patients to further evaluate durability.

Marker Preparing for Company-Sponsored Phase 2 Clinical Trial in AML
The multicenter trial will evaluate clinical efficacy of Marker’s MultiTAA T cell therapy in patients with AML in both the adjuvant and active disease setting following an allogeneic hematopoietic stem cell transplant (HSCT). The dose in the Phase 2 trial is expected to be the maximum tolerated dose currently determined in the BCM-sponsored Phase 1 trial. In the adjuvant setting, patients will be randomized to either MultiTAA T cell therapy at approximately 90 days post-transplant or standard-of-care observation, while the active disease patients will receive MultiTAA T cells upon relapse as part of a single-arm group.

T Cell-Based Vaccines

The Company continues to advance its T cell-based vaccine programs in ovarian cancer and triple negative breast cancer.

Phase 2 Ovarian Cancer Clinical Trial Highlights

TPIV200 is being studied as a maintenance therapy for patients in their first remission after surgery and platinum-based chemotherapy.
Trial is fully enrolled, with a total of 120 patients randomized and treated at 17 clinical sites.
Company expects to reach planned interim analysis trigger of 55 patients who have progressed before the end of 2019 and to report results of this interim analysis in the fourth quarter of 2019.
Phase 2 Triple Negative Breast Cancer Trial Highlights

Based on preliminary analysis of 34 patients evaluated to date in the dose-finding, four-arm trial—including low and high-dose TPIV200 with or without cyclophosphamide—31 showed meaningful immune response to the vaccine treatment (subject to final review by independent biostatistical analysis).
Of 80 patients treated at 11 clinical sites, 14 have shown disease progression, as of June 30, 2019, following treatment with TPIV200.
CORPORATE UPDATE

On August 6, Steve Elms was appointed to Marker’s Board of Directors. Mr. Elms currently serves as Managing Partner at Aisling Capital.
SECOND QUARTER 2019 FINANCIAL RESULTS

Net loss for the quarter ended June 30, 2019 was $5.6 million, compared to a net loss of $4.8 million for the quarter ended June 30, 2018.

Research and development expenses were $3.2 million for the quarter ended June 30, 2019, an increase of $1.4 million, compared to $1.8 million for the quarter ended June 30, 2018. The increase was primarily attributable to increases in personnel-related expenses, including stock-based compensation expenses and consulting expenses, relating to the build-up of Marker’s internal infrastructure as the Company advances the clinical development of its MultiTAA T cell product candidates.

General and administrative expenses were $2.7 million for the quarter ended June 30, 2019, a decrease of $0.4 million, compared to $3.1 million for the quarter ended June 30, 2018. The decrease was primarily attributable to $1.2 million of merger-related expenses incurred during the three months ended June 30, 2018, offset by increased expenses relating to $0.3 million of headcount-related expenses, $0.4 million of non-merger-related legal and other professional expenses and $0.1 million of office-related and insurance expenses.

CASH POSITION AND GUIDANCE
At June 30, 2019, Marker had cash and cash equivalents of $53.4 million. The Company believes that its existing cash and cash equivalents will fund the Company’s current operations into late 2020.

On August 8, 2019 Geron Corporation (Nasdaq: GERN) reported the opening of patient screening and enrollment for the Phase 3 portion of IMerge to evaluate imetelstat, a first-in-class telomerase inhibitor, in lower risk myelodysplastic syndromes (MDS) (Press release, Geron, AUG 8, 2019, View Source [SID1234538391]).

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"The start of the Phase 3 portion of IMerge is a significant milestone for Geron and imetelstat," said John A. Scarlett, M.D., Chairman and Chief Executive Officer. "We are hopeful that the Phase 3 will confirm the encouraging results from the Phase 2 portion, and that imetelstat could become a much-needed treatment alternative for patients with lower risk MDS."

IMerge is a two-part Phase 2/3 clinical trial of imetelstat in transfusion dependent patients with lower risk MDS who are relapsed after or refractory to erythroid stimulating agents (ESAs). The Phase 3 portion is planned to enroll approximately 170 patients in a randomized, double-blind, placebo-controlled clinical trial to test the hypothesis that imetelstat improves the rate of red blood cell transfusion independence (TI). The trial is planned to be conducted at multiple medical centers globally, including North America, Europe, Middle East and Asia. The primary efficacy endpoint is 8-week TI rate, which is defined as the proportion of patients achieving transfusion independence during any consecutive eight weeks since entry into the trial. Key secondary endpoints include the rate of transfusion independence lasting at least 24 weeks, or 24-week TI rate, durability of transfusion independence and the amount and relative change in transfusions.

Many key aspects from the Phase 2 portion of IMerge remain the same for the Phase 3 portion. A target patient population of non-del(5q) lower risk MDS patients who are naïve to treatment with hypomethylating agents (HMAs) and lenalidomide was identified from the Phase 2 portion, and will be enrolled in the Phase 3. In addition, the primary and secondary endpoints, the dose and schedule of imetelstat administration and many of the clinical sites remain the same as in the Phase 2. Recently reported Phase 2 data highlighted the meaningful and durable transfusion independence, disease-modifying activity, and efficacy responses across MDS patient subgroups potentially achievable with imetelstat treatment.

Based upon current planning assumptions, Geron expects top-line results for the IMerge Phase 3 trial to be available by mid-year 2022.

To learn more about IMerge and whether the study is enrolling patients in your area, please visit www.clinicaltrials.gov.

About Myelodysplastic Syndromes

Myelodysplastic syndromes are a group of diverse blood disorders that develop because bone marrow cells do not mature into healthy blood cells. Many patients develop chronic anemia, the predominant clinical problem in lower risk MDS, and become dependent on red blood cell transfusions. There are approximately 60,000 people living with the disease in the United States.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Early clinical data suggest imetelstat may have disease-modifying activity through the suppression of malignant progenitor cell clone proliferation, which allows potential recovery of normal hematopoiesis. Ongoing clinical studies of imetelstat consist of IMerge, a Phase 2/3 trial in lower risk myelodysplastic syndromes (MDS) and IMbark, a Phase 2 trial in Intermediate-2 or High-risk myelofibrosis. Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for the treatment of patients with transfusion dependent anemia due to non-del(5q) lower risk MDS who are refractory or resistant to an erythroid stimulating agent.

On August 8, 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 financial results for the second quarter ended June 30, 2019 and recent business highlights (Press release, Magenta Therapeutics, AUG 8, 2019, View Source [SID1234538423]).

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"In the first half of 2019, Magenta made meaningful progress in executing on our goals, including advancing our second program into the clinic and extending our cash runway through a follow-on financing," said Jason Gardner, D. Phil., Chief Executive Officer and President, Magenta Therapeutics. "We look forward to sharing results of our progress through a number of clinical and preclinical data sets in the second half of the year."

Upcoming Anticipated Milestones:

The Company plans to achieve the following key milestones in 2019:

Present clinical data from the Phase 1 study of MGTA-145
Present additional clinical data from the Phase 2 study of MGTA-456 in inherited metabolic disorders (IMDs)
Present preclinical data on anti-CD45 targeted conditioning program in autoimmune disease and declare a development candidate
Present preclinical data on anti-CD117 targeted conditioning program in gene therapy
Recent Business Highlights:

Initiated Phase 1 study of MGTA-145 as first-line stem cell mobilization therapy: In April 2019, Magenta announced that it had dosed the first subjects in a Phase 1 study of MGTA-145. Magenta intends to develop MGTA-145 in autoimmune diseases, blood cancers and genetic diseases. The Phase 1 study will investigate the safety and tolerability of MGTA-145 alone and in combination with plerixafor in healthy volunteers and establish recommended Phase 2 doses as well as measure the number of hematopoietic stem cells in the blood after dosing with MGTA-145 alone and in combination with plerixafor. Study enrollment is on track, and Magenta expects to present data from the study in the second half of 2019. Depending on the Phase 1 data, the Company plans to move MGTA-145 into a Phase 2 study in multiple myeloma and non-Hodgkin lymphoma in 2020.

Updated clinical data for MGTA-456 cell therapy showed continued signs of durable clinical benefit in patients with IMDs: Magenta presented updated data from the Phase 2 clinical study of MGTA-456 in patients with IMDs at the American Academy of Neurology (AAN) annual meeting in May 2019. Patients with cerebral adrenoleukodystrophy (cALD) treated with MGTA-456 in the study showed stable neurological function scores and persistent resolution of brain inflammation by MRI at 6 months post-transplant, suggesting that the progression of disease has been halted. Magenta expects to update longer term results in these patients in the second half of 2019.

Submitted multiple abstracts from across the pipeline to the 2019 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting: Magenta submitted abstracts from across its conditioning, mobilization and cell therapy programs for presentation at the 2019 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting.

Appointed Anne McGeorge to board of directors: In June 2019, Magenta announced that it had appointed Anne McGeorge to the board of directors and named her chair of the audit committee. Ms. McGeorge is an operating partner at Havencrest Healthcare Partners, a growth equity fund specializing in the healthcare industry, and is on the advisory board at Dioko Ventures, a healthcare venture fund, and is a member of the board of directors of The Be The Match Foundation and a member of the audit/finance committee of The National Marrow Donor Program, both of which are non-profit organizations dedicated to helping patients access life-saving stem cell transplants.

Financial Results:

Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2019, were $172.3 million compared to $142.6 million on December 31, 2018. In addition, in May 2019 Magenta announced that it completed a public offering of common stock and raised net proceeds of $60.3 million. Magenta anticipates that its cash, cash equivalents and marketable securities, including the proceeds from this recent financing, will be sufficient to fund operations and capital expenditures into the second half of 2021.

Research and Development Expenses: Research and development (R&D) expenses were $13.4 million in the second quarter of 2019, compared to $9.7 million in the second quarter of 2018. The increase was driven primarily by investments in clinical activities for MGTA-145, as well as manufacturing related to our conditioning programs.

General and Administrative Expenses: General and administrative (G&A) expenses were $5.9 million for the second quarter of 2019, compared to $4.3 million for the second quarter in 2018. The increase was primarily due to increased personnel and facility costs associated with the growth of the Company.

Net Loss: Net loss was $17.7 million for the second quarter of 2019, compared to net loss of $13.7 million for the second quarter of 2018.