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Turning Point Therapeutics and Almac Diagnostic Services Announce Approval of an Investigational Device Exemption for the Companion Diagnostic Assay to the Registrational TRIDENT-1 Clinical Study

On May 6, 2019 Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a precision oncology company developing novel drugs to address treatment resistance, and Almac Diagnostic Services, a global stratified medicine company, reported approval by the U.S. Food and Drug Administration (FDA) of an investigational device exemption (IDE) for the diagnostic assay that will be used in Turning Point’s registrational phase 2 clinical study (Press release, Turning Point Therapeutics, MAY 6, 2019, View Source [SID1234535761]).

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The next-generation sequencing (NGS) assay is intended for use in identifying patients with ROS1, NTRK1-3 and ALK gene fusions in advanced solid tumors. Clinical investigators will use the diagnostic test to determine molecular eligibility for enrollment into Turning Point’s registrational Phase 2 portion of the global TRIDENT-1 clinical study.

"We are pleased to achieve this important milestone with our partner Almac, moving us closer to the start of our pivotal study for repotrectinib in the second half of 2019," said Athena Countouriotis, M.D., president and chief executive officer. "With a clear unmet medical need for therapies that target genomic alterations in different cancers, the diagnostic assay will provide clinical investigators with timely information to confirm the presence of the oncogenic drivers we continue to study in patients with advanced solid tumors."

Almac has been developing the NGS diagnostic based on Archer Dx’s Anchored Multiplex PCR (AMP) chemistry, with the intent of submitting it for regulatory approval in the United States and using it initially at Almac’s CLIA-accredited laboratory in Durham, N.C. to identify tumors with the targeted gene fusions, enabling physicians to select appropriate patients for treatment with repotrectinib.

"We are very pleased to have been granted IDE approval by the FDA for the development and validation of this complex NGS diagnostic assay for its intended use as a clinical trial assay," said Paul Harkin, president and managing director, Almac Diagnostic Services. "We are delighted to assist Turning Point Therapeutics with this important upcoming clinical study, and furthering our partnership to progress such a promising investigational therapy as repotrectinib."

Repotrectinib is an investigational, next-generation tyrosine kinase inhibitor (TKI) developed for the treatment of patients with advanced solid tumors harboring ROS1, NTRK1-3 or ALK molecular rearrangements. Repotrectinib is a rationally designed, low molecular weight, macrocyclic TKI that is much smaller than current ROS1, TRK family and ALK inhibitors with the objective to systematically overcome the clinically acquired resistance mutations of ROS1, TRK family and ALK kinases, especially the gatekeeper and solvent front mutations.

Turning Point Therapeutics’ TRIDENT-1 Phase 2 open-label, multi-cohort study is planned for initiation in the second half of 2019.

About Almac Diagnostic Services

Almac Diagnostic Services is a global stratified medicine company specializing in biomarker driven clinical trials. The company is focused on the discovery, development and commercialization of diagnostic and companion diagnostic tests. Almac partners with biopharma companies to provide solutions ranging from biomarker discovery to CDx development including regulatory submissions and commercialization. Almac also facilitates biomarker clinical trial management and clinical test delivery from its CLIA-accredited labs. The tests developed at Almac Diagnostic Services have a wide range of applications including patient selection, and are utilized in phase I to phase III registrational clinical trials. For more information visit: www.almacgroup.com/diagnostics

Syndax Pharmaceuticals Reports First Quarter 2019 Financial Results and Provides Clinical and Business Update

On May 6, 2019 Syndax Pharmaceuticals, Inc. (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the first quarter ended March 31, 2019. In addition, the Company provided a clinical and business update (Press release, Syndax, MAY 6, 2019, View Source [SID1234535760]). As of March 31, 2019, Syndax had $92.7 million in cash, cash equivalents and short-term investments.

"We are pleased to report that E2112, our Phase 3 registration trial of entinostat plus exemestane in HR+, HER2- breast cancer, has passed its fourth interim overall survival analysis," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "E2112 was designed to replicate the compelling overall survival results obtained in the Phase 2b ENCORE 301 trial which led to Breakthrough Therapy designation. The next overall survival assessment is expected in the fourth quarter of this year. We remain confident in the potential that the addition of entinostat to exemestane will result in a positive survival benefit, which would allow us to file for full regulatory approval in this indication."

Dr. Morrison added, "We also look forward to filing an IND for SNDX-5613, our targeted menin inhibitor, later this quarter. Supported by a robust preclinical dataset, we believe this therapeutic class has the potential to make a meaningful impact for patients with genetically-defined acute leukemias for whom limited effective therapies exist."

Pipeline Updates

Entinostat

ECOG-ACRIN has informed the Company that following its fourth preplanned interim overall survival (OS) analysis, the E2112 trial will continue as planned until either an OS benefit is observed, or the final target number of events occur. E2112 is Syndax’s NCI-sponsored, ECOG-ACRIN led Phase 3 registration trial of entinostat, a Class I selective HDAC inhibitor, plus exemestane in advanced hormone receptor positive, human epidermal growth factor receptor 2 negative (HR+, HER2-) breast cancer. The next interim analysis for the OS endpoint is scheduled for 4Q19, with a final OS assessment, if necessary, to be conducted in 2Q20. Any positive OS assessment would enable the Company to file for full regulatory approval. The E2112 trial design was informed by the Phase 2b ENCORE 301 trial, the results of which led to entinostat’s Breakthrough Therapy designation in HR+, HER2- breast cancer, in which patients receiving the entinostat/exemestane combination demonstrated a statistically significant OS benefit.

At the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting held March 29 – April 3, 2019, Syndax presented data from the non-small cell lung cancer (NSCLC) and melanoma cohorts of the ENCORE 601 trial of entinostat in combination with KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 (programmed death receptor-1) therapy. These data provided further evidence that the addition of entinostat to pembrolizumab may overcome resistance to immunotherapy in melanoma and NSCLC patients whose disease progressed on or after anti-PD-1 therapy. As the Company has previously indicated, following availability of positive E2112 OS results, it will determine whether to advance its entinostat-PD-1 combination programs into one or more registration trials.

SNDX-5613

Syndax continues to expect to file an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for its targeted menin inhibitor, SNDX-5613, later this quarter, with the initiation of a Phase 1 clinical trial in a defined subset of acute leukemia patients expected to follow shortly thereafter.

SNDX-6352

The Company continues to anticipate initial results from the Phase 1 dose escalation trial of SNDX-6352, Syndax’s anti-CSF-1R monoclonal antibody, in patients with chronic graft versus host disease (cGVHD) in the second half of the year. The objectives of this trial are to evaluate the safety and preliminary efficacy of SNDX-6352 in cGVHD and to identify a recommended Phase 2 dose and schedule.

First Quarter 2019 Financial Results

As of March 31, 2019, Syndax had cash, cash equivalents and short-term investments of $92.7 million and 31.6 million shares and share equivalents issued and outstanding.

In March 2019, Syndax issued 4.5 million shares of its common stock and prefunded warrants at an offering price of $6.00, as well as warrants to purchase up to 4.5 million shares of its common stock, with half at an exercise price of $12.00 per share and the remaining half at an exercise price of $18.00 per share. As a result of the offering, Syndax received aggregate net proceeds of approximately $27.4 million.

First quarter 2019 research and development expenses decreased to $11.3 million from $15.3 million. The first quarter decrease was primarily due to reduced CMC activities and decreased clinical activities.

General and administrative expenses for the first quarter 2019 decreased to $3.9 million from $4.8 million. The decrease was primarily due to decreased pre-commercialization expenses and decreased professional fees.

For the three months ended March 31, 2019, Syndax reported a net loss attributable to common stockholders of $14.3 million or $0.53 per share compared to $19.4 million or $0.79 per share for the prior year period.

Financial Guidance

Today the Company provided operating expense guidance for the second quarter and full year 2019. For the second quarter and full year 2019, research and development expenses are expected to be $9 to $10 million and $46 to $50 million, respectively, and total operating expenses are expected to be $13 to $14 million and $60 to $64 million, respectively.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Monday, May 6, 2019.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

Conference ID: 4292817
Domestic Dial-in Number: 855-251-6663
International Dial-in Number: 281-542-4259
Live Webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors section of the Company’s website, www.syndax.com.

Protalix BioTherapeutics Reports 2019 First Quarter Results and Provides Corporate Update

On May 6, 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 three months ended March 31, 2019 and provided a corporate update (Press release, Protalix, MAY 6, 2019, View Source;p=RssLanding&cat=news&id=2397172 [SID1234535759]).

"In the first three months of 2019, we have continued to execute on enrollment in our PRX-102 studies and have worked to prepare ourselves for a potential accelerated approval path," said Mr. Moshe Manor, Protalix’s President and Chief Executive Officer. "In addition, we are encouraged by the initial pharmacokinetic (PK) data from our BRIGHT study, which were presented at the 15th Annual WORLDSymposiumTM 2019 in February 2019, that demonstrate the potential for PRX-102 to be infused once-monthly, compared to the current treatment regimen of every two weeks."

First Quarter 2019 and Recent Clinical and Corporate Highlights

The Company’s BRIGHT phase III clinical trial of pegunigalsidase alfa, or PRX-102, for the treatment of Fabry disease is currently one patient away from completion of enrollment.

The Company’s BALANCE phase III clinical trial of pegunigalsidase alfa for the treatment of Fabry disease is currently eleven patients away from completion of enrollment.

The Company presented results from the BRIGHT Study at the 15th Annual WORLD Symposium showing that infusion of pegunigalsidase alfa every 4 weeks results in the presence of continuous active enzyme throughout the entire infusion interval.

The Company is scheduled to present three posters during the 6th Update on Fabry Disease international conference being held in Prague, Czech Republic, on May 26-28, 2019.

To date, more than 40 patients are being treated in the Company’s various extension studies after opting to continue treatment with pegunigalsidase alfa after they completed an initial study.

The Company plans to meet with the U.S. Food and Drug Administration (FDA) for a follow up meeting during the second quarter of 2019 in connection with the potential accelerated approval filing path for pegunigalsidase alfa.
First Quarter 2019 Financial Results

The Company recorded total revenues of $10.4 million during the three months ended March 31, 2019, compared to $6.7 million for the same period of 2018. The increase is primary attributable to the recognition of $6.9 million of license revenues in the three months ended on March 31, 2019 compared to the recognition of $2.2 million in the same period of 2018.

Research and development expenses for the three months ended March 31, 2019, were $11.7 million, compared to $7.3 million for the same period in 2018. Selling, general and administrative expenses for the three months ended March 31, 2019 were $2.2 million, compared to $2.5 million incurred during the same period in 2018.

Net loss for the three months ended March 31, 2019 was $7.3 million compared to $7.2 million for the three months ended March 31, 2018.
On March 31, 2019, the Company had $30.4 million of cash and cash equivalents, compared to $37.8 million at March 31, 2018, which is currently projected to fund operations into mid-2020. As of March 31, 2019, the Company had outstanding $57.9 million of its 7.50% convertible promissory notes due November 2021.
Conference Call and Webcast Information

The Company will host a conference call on Monday, May 6, 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 6169584.

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.

Onxeo Announces Treatment of First Patient in DRIIV-1b, a Phase 1b Clinical Trial of AsiDNA™ in Combination with Chemotherapy

On May 6, 2019 Onxeo S.A. (Euronext Paris, NASDAQ Copenhagen: ONXEO), ("Onxeo" or "the Company"), a clinical-stage biotechnology company specializing in the development of innovative drugs targeting tumor DNA Damage Response (DDR) in oncology, in particular against rare or resistant cancers, reported a new milestone in the clinical development of AsiDNA with the treatment of the first patient in DRIIV-1b, a phase 1b clinical study of AsiDNA, a first-in-class tumor DNA repair inhibitor, in combination with carboplatin and with carboplatin plus paclitaxel, in patients with solid tumors eligible to such treatments (Press release, Onxeo, MAY 6, 2019, View Source [SID1234535758]).

DRIIV-1b is an extension of the DRIIV-1 (DNA Repair Inhibitor administered IntraVenously) phase 1 study currently being completed, in which AsiDNA, administered intravenously (IV) demonstrated its intratumoral activity by inducing a significant increase in its activity biomarkers in the tumor cells of patients, with a favorable safety profile at various active doses.

At the active dose of 600 mg, among the three patients included in the cohort, two patients with relapsed, multi-treated metastatic colorectal cancer were controlled with medical imaging, which showed no further disease progression after the second treatment cycle, and continued their treatment with AsiDNA for three months. The 600 mg active dose was considered to be optimal for further development of AsiDNA in combination with chemotherapy.

DRIIV-1b aims at showing the safety and efficacy of a 600 mg dose of AsiDNA in combination with carboplatin, and carboplatin plus paclitaxel, in up to 18 patients with solid tumors eligible for such treatments (lung, breast, ovarian or head and neck cancers, …). The efficacy of the combinations will be evaluated every six to eight weeks by medical imaging in accordance with RECIST criteria (Response evaluation criteria in solid tumors). The study will take place in Belgium, and initial results are expected in the second half of 2019.

Dr Nuria Kotecki of the Institute Jules Bordet in Brussels commented: "The «DDR» (DNA Damage Response) approach represents a particularly interesting alternative in cancer treatment. Indeed, combining AsiDNA, a tumor DNA repair inhibitor, with agents such as carboplatin, that causes breaks in that same DNA, is a very promising approach in terms of synergistic efficacy. On the basis of the safety profile of AsiDNA observed in monotherapy, this combination can be considered as we are looking for greater efficacy without aggravating the toxicity observed with chemotherapy. We are thrilled to start this DRIIV-1b study, which should enable us to confirm the preclinical and clinical results already obtained."

This first combination trial represents a major milestone in the clinical development of AsiDNA. Thanks to its highly differentiated mechanism of action, confirmed by exhaustive preclinical studies, the combination of AsiDNA with various anti-cancer treatments appears especially relevant to increase their efficacy and avoid the occurrence of resistance from tumors.

DRIIV-1b is the first combination study of AsiDNA by IV administration, aimed at confirming such synergistic efficacy on tumors for which the medical needs remain immense. Positive results from this study will represent a proof of the interest of AsiDNA combined with chemotherapy and will open the door to further clinical development of AsiDNA IV in a phase 2 program in one or several indications.

Olivier de Beaumont, Onxeo’s Chief Medical Officer, concluded: "This study marks the start of the clinical development of AsiDNA in combination with chemotherapy. The results, expected by the end of the year, will enable us to confirm the potential of our flagship product in indications with strong medical needs. Other combination studies are also being prepared to further support the growing interest in AsiDNA and its broad clinical potential. We are very pleased to be continuing our collaboration with Dr Nuria Kotecki, a clinical investigator already involved in the DRIIV-1 study, and we thank her for her help and support in this promising research program."

Onconova Therapeutics Announces Presentations at the 15th International Symposium on Myelodysplastic Syndromes (MDS 2019) in Copenhagen, Denmark

On May 6, 2019 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3 stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with a primary focus on Myelodysplastic Syndromes (MDS), reported that five posters related to the Company’s lead product candidate, rigosertib, were accepted for presentation at the 15th International Symposium on Myelodysplastic Syndromes taking place May 8-11, 2019 at the Tivoli Hotel & Congress Center in Copenhagen, Denmark (Press release, Onconova, MAY 6, 2019, View Source [SID1234535757]).

Dr. Ric Woodman, CMO of Onconova, will be attending the Symposium and available to meet with interested parties.

DETAILS OF THE POSTERS:

1) PHASE II STUDY OF ORAL RIGOSERTIB COMBINED WITH AZACITIDINE IN PATIENTS WITH HIGHER-RISK MYELODYSPLASTIC SYNDROMES (MDS)
Abstract: MDS19-0216
May 8 – May 11, Room Vandsalen
Presenter: Lewis R. Silverman, MD
Tisch Cancer Institute, Division of Hematology/Oncology, Icahn School of Medicine at Mount Sinai, New York, NY

2) RIGOSERTIB /AZACITIDINE MODULATES INNATE IMMUNE SIGNALING AND HEMATOPOIETIC GENE PATHWAYS WHEN SEQUENCED IN VITRO, IN THE MYELODYSPLASTIC SYNDROME (MDS)
Abstract: MDS19-0201
May 8 – May 11, Room Vandsalen
Presenter: Lewis R. Silverman, MD
Tisch Cancer Institute, Division of Hematology/Oncology, Icahn School of Medicine at Mount Sinai, New York, NY

3) EMERGENCE OF PTPN11 MUTATIONS IS ASSOCIATED WITH CLINICAL RESISTANCE TO RIGOSERTIB AND AZACITIDINE IN PATIENTS WITH MYELODYSPLASTIC SYNDROME (MDS)
Abstract: MDS19-0195
May 8 – May 11, Room Vandsalen
Presenter: Lewis R. Silverman, MD
Tisch Cancer Institute, Division of Hematology/Oncology, Icahn School of Medicine at Mount Sinai, New York, NY

4) UTILITY OF ADAPTIVE TRIAL DESIGN IN HIGHER-RISK MYELODYSPLASTIC (HR-MDS) SYNDROMES IN A PHASE 3 TRIAL OF INTRAVENOUS RIGOSERTIB: INSPIRE TRIAL
Abstract: MDS19-0211
May 8 – May 11, Room Vandsalen
Presenter: Aref Al-Kali, MD
Mayo Clinic, Hematology, Rochester, MN

5) EX VIVO RESPONSE PROFILING OF RIGOSERTIB IDENTIFIES DISTINCT CLASSES OF RESPONDERS IN ELODYSPLASTIC SYNDROME
Abstract: MDS19-0200
Presenter: Marianne Santaguida, PhD, Director of Scientific Partnerships
Notable Labs, Foster City, CA

About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) are conditions that can occur when the blood-forming cells in the bone marrow become dysfunctional and thus produce an inadequate number of circulating blood cells. It is frequently associated with the presence of blasts or leukemic cells in the marrow. This leads to low numbers of one or more types of circulating blood cells, and to the need for blood transfusions. In MDS, some of the cells in the bone marrow are abnormal (dysplastic) and may have genetic abnormalities associated with them. Different cell types can be affected, although the most common finding in MDS is a shortage of red blood cells (anemia). Patients with higher-risk MDS may progress to the development of acute leukemia.