1ST Biotherapeutics and twoXAR Form Co-Development Collaboration to Discover and Develop Treatments for Glioblastoma

On January 3, 2019 1ST Biotherapeutics, Inc., a preclinical-stage biotechnology company focused on neurodegenerative diseases, immuno-oncology, and orphan diseases, and twoXAR, Inc., an artificial intelligence (AI)-driven biopharmaceutical company, reported an agreement to jointly discover and develop novel, efficacious treatments to address unmet medical needs in glioblastoma multiforme ("glioblastoma") (Press release, 1ST Bio Therapeutics, JAN 3, 2019, View Source [SID1234532432]).

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Under the agreement, twoXAR will use its proprietary AI technology to identify a set of drug candidates with the potential to slow, stop, or reverse the progression of glioblastoma. twoXAR and 1ST Biotherapeutics will select candidates from this set to test in preclinical efficacy models of glioblastoma. Following identification of one or more candidates based on those evaluated, 1ST Biotherapeutics will use its team’s expertise in drug development to optimize candidates and finalize the creation of novel, efficacious treatments. Further details of the agreement were not disclosed.

Glioblastoma is the most common and lethal primary malignant brain tumor with a median survival of approximately 15 months1. It is characterized by an aggressive tumor that infiltrates various portions of the brain and is associated with rapid onset of neurological symptoms, including nausea, vomiting, severe headaches, cognitive issues, and seizures2. Current treatment options consist of surgical resection, radiation, chemotherapy, and angiogenesis inhibitors2. However, most glioblastoma patients experience disease relapse despite these aggressive therapies. It is estimated that 25,000 people will be diagnosed with glioblastoma in 20181.

"1ST Biotherapeutics is focused on efficiently building a pipeline of first-in-class therapeutic candidates with high likelihood of clinical success," said Jamie Jae Eun Kim, CEO of 1ST Biotherapeutics. "The twoXAR team has a track record of rapidly identifying testable novel treatments that can lead to first-in-class therapeutics. This collaboration is an opportunity to combine twoXAR’s AI-driven drug discovery approach and the 1ST Biotherapeutics team’s expertise in chemistry and pharmacology to discover and develop effective molecular therapeutics for glioblastoma patients."

"We are pleased to collaborate with 1ST Biotherapeutics, because we share common goals of efficiently discovering and developing novel therapeutics for diseases with high unmet medical need, such as glioblastoma," said Andrew A. Radin, Co-Founder and CEO of twoXAR. "The 1ST Biotherapeutics team’s deep medicinal chemistry and drug development experience in CNS and oncology diseases provides a strong complement to twoXAR’s data-driven discovery approach."

Onconova Submits Special Protocol Assessment (SPA) To FDA For Phase 3 Trial Of Oral Rigosertib In Combination With Azacitidine (Vidaza®) For First-Line Myelodysplastic Syndromes (MDS)

On January 2, 2019 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, with a primary focus on MDS, reported that it has submitted a Special Protocol Assessment request to the U.S. Food and Drug Administration (FDA) for a Phase 3 study of oral rigosertib combination therapy with azacitidine (Vidaza) for the treatment of adult patients with treatment-naïve higher-risk MDS (Press release, Onconova, JAN 2, 2019, Onconova Submits Special Protocol Assessment (SPA) To FDA For Phase 3 Trial Of Oral Rigosertib In Combination With Azacitidine (Vidaza) For First-Line Myelodysplastic Syndromes (MDS) [SID1234532341]). The request is part of the Company’s ongoing interaction with the FDA, following an End-of-Phase 2 Meeting with FDA guidance for the proposed Phase 3 study and Scientific Advice from the European Health Authorities, consistent with the Company’s strategy to study rigosertib in an earlier higher-risk MDS patient population with a more convenient mode of oral rigosertib administration. The End-of-Phase 2 Meeting also outlined that the primary endpoint of the proposed pivotal trial will be overall response rate (ORR), a composite of complete remission (CR), and partial remission (PR) based on the IWG Response Criteria.

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Dr. Steve Fruchtman, President of Onconova, commented: "We remain focused in 2019 on the completion of the pivotal INSPIRE trial studying intravenous rigosertib in higher-risk MDS after patients fail to respond to or progress on hypomethylation therapy, the standard of care. The timely achievement of this regulatory milestone of this SPA submission is an important step in advancing the development of rigosertib for patients with earlier stage higher-risk MDS. We believe that the promising data in hand, including the data from the Phase 2 expansion trial of oral rigosertib and azacitidine presented at this year’s 2018 ASH (Free ASH Whitepaper) Annual Meeting, provides a strong scientific rationale for the proposed Phase 3 program. As the INSPIRE trial continues to mature, we look forward to a constructive engagement with the FDA on future studies. We also aim to help fund these additional studies through expanding our partnerships."

The FDA’s SPA process fosters dialogue between the FDA and clinical trial sponsors before studies commence, in an attempt to reach potential agreement with the agency on the design and size of clinical trials, to determine if they adequately address the scientific and regulatory requirements for a study to ultimately support marketing approval. The Company expects its dialogue with the FDA on this SPA submission to conclude in H1 2019.

Harpoon Therapeutics Announces Preliminary Safety and Pharmacology Data from its HPN424 Phase 1 Trial in Prostate Cancer

On January 2, 2019 Harpoon Therapeutics, Inc. ("Harpoon"), a clinical-stage immunotherapy company developing a novel class of T cell engagers that harness the power of the body’s immune system to treat patients with cancer and other diseases, reported preliminary safety and mechanism of action findings from an ongoing Phase 1 clinical trial evaluating its lead compound, HPN424, in patients with progressive metastatic castration resistant prostate cancer (mCRPC) (Press release, Harpoon Therapeutics, JAN 2, 2019, View Source [SID1234532362]). The Phase 1 trial is a multicenter, open-label dose escalation and dose expansion study to evaluate the safety, tolerability, pharmacokinetics and clinical activity of HPN424, a TriTAC (Tri-specific T Cell Activating Construct) that targets prostate-specific membrane antigen (PSMA), a clinically validated tumor antigen target in prostate cancer.

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Eligible patients must have mCRPC, have received at least two prior treatment regimens for mCRPC, and have evidence of disease progression on the most recent systemic treatment regimen. Clinical observations from the first seven prostate cancer patients treated with HPN424 include:

Measurement of drug in the sera from patients indicates sufficient drug exposure during the treatment course to support once weekly dosing.
Measurable reduction in circulating tumor cells (CTC) in three of four patients evaluated as a biomarker for PSMA target engagement.
Dose-dependent, transient increase in peripheral cytokine and chemokine levels, consistent with the expected T cell activation mechanism of action.
No dose limiting toxicities have been observed to date, with four dose levels tested.
Adverse events were consistent with the expected mechanism of action, with three patients reporting grade 2 rigors or fevers that were manageable. A grade 3 cytokine release syndrome (CRS) event was reported in one patient from the fourth cohort but resolved within eight hours. All patients experiencing adverse events were successfully re-treated after one week without further complications.
All seven patients remain on study with weekly treatment of HPN424. Three patients have been analyzed for disease burden using computed tomography scans, bone scans and measurement of prostate serum antigen levels. To date, two patients have shown stable disease and one patient exhibited unconfirmed progressive disease and continues on study. The other four patients are early in their treatment and have not yet been evaluated for disease burden.
"These initial results in advanced metastatic castration resistant prostate cancer patients suggest that HPN424 is activating T cells in a manner that is consistent with tumor target engagement," said Natalie Sacks, M.D., Chief Medical Officer of Harpoon. Dr. Sacks added, "We have observed early signals confirming the mechanism of action of HPN424, and have obtained encouraging safety data that supports continued dose escalation to define a Phase 2 therapeutic dose and regimen."

"These results provide the first clinical evidence suggesting Harpoon’s TriTAC platform is performing as designed, by activating T cells in a target-dependent manner and by showing evidence of sufficient drug exposure during the treatment course to support once weekly dosing," said Holger Wesche, Ph.D., Chief Scientific Officer of Harpoon. "HPN424 is a novel T cell biologic that has been designed to deliver potent T cell killing to PSMA-expressing tumor cells. We are encouraged by the early human safety and pharmacology data emerging from our Phase 1 trial."

"We are excited by this early data for HPN424 and the broader application of our technology. Our novel TriTAC drug candidates are designed to enable a patient’s own T cells to fight tumors in a wide range of human malignancies," said Jerry McMahon, Ph.D., President and Chief Executive Officer of Harpoon. "We are looking forward to reporting additional clinical data from our ongoing HPN424 Phase 1 clinical trial in 2019 as we continue to enroll and analyze data from existing and new patients."

Phase 1 Trial Observations (As of December 31, 2018)

Patient Treatment and Tolerability: Seven patients have been treated in the dose escalation portion of the trial, at doses ranging from 1.3 to 24 ng/kg. All seven have been treated previously with a second-generation anti-androgen therapy. All seven patients remain on study treatment, with the time on treatment ranging from one to more than 20 weeks, and the number of doses received ranging from one to 22 weekly treatments. No dose limiting toxicities have been observed. One patient experienced a grade 3 CRS event which resolved within eight hours of dosing. This patient was re-administered HPN424 consistent with protocol guidelines. The patient experienced no further reactions and continues to be enrolled in the study. Based on this event, the dose escalation is proceeding using three to six patients per cohort consistent with the original Phase 1 plan.

Serum Drug Exposure: Preliminary pharmacokinetic analysis supports at least once weekly dosing. Maximum serum concentration of HPN424 was found to be proportional to the dose. The volume of distribution and clearance rates appear to be similar among different dose levels suggesting linear pharmacokinetic properties.

T Cell Engagement: Transient and dose-dependent increases in peripheral cytokines (interleukin-6, interleukin-8, interleukin-10) and chemokines (macrophage inflammatory protein-1-alpha, macrophage inflammatory protein-1-beta, monocyte chemoattractant protein-1) were observed, consistent with the expected mechanism of action related to T cell activation.

Tumor Assessments: Radiographic assessment of disease burden is performed at nine-week intervals. For the first three patients in the trial, radiographic and PSA data have shown stable disease for two patients and one patient exhibited unconfirmed progressive disease. The other four patients are early in their treatment and not yet evaluable. Whole blood samples from three of four patients with measurable baseline CTC levels showed a reduction in CTC following treatment with HPN424.

About HPN424-1001 Clinical Trial

The HPN424-1001 Phase 1 trial is a multicenter, open-label dose escalation and dose expansion study to evaluate the safety, tolerability, pharmacokinetics and activity of HPN424, a novel T cell engaging therapeutic that targets PSMA (clinicaltrials.gov ID#: NCT03577028). Eligible patients must have mCRPC, have received at least two prior regimens for mCRPC, and have evidence of disease progression on the most recent systemic regimen. In the first part of the trial, HPN424 is administered once weekly via intravenous infusion in ascending dose cohorts until a therapeutic dose is achieved. Following dose escalation, the study will expand and enroll approximately 20 patients at the recommended Phase 2 dose established in the first part of the trial.

Novavax to Present at the 37th Annual J.P. Morgan Healthcare Conference

On January 2, 2019 Novavax, Inc. (Nasdaq:NVAX) reported that it will present at the 37th Annual J.P. Morgan Healthcare Conference (Press release, Novavax, JAN 2, 2019, View Source [SID1234532400]).

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Presentation details are as follows:

Date and time:Wednesday, January 9, 3:00 p.m. – 3:25 p.m. P.T.
Location:Colonial Room, Westin St. Francis Hotel, San Francisco
Live webcast:www.novavax.com, "Investors"/"Events"

A replay of the presentation will also be accessible under the "Investors/Events" section of the website at www.novavax.com.

Stemline Therapeutics to Present at the 37th Annual J.P. Morgan Healthcare Conference

On January 2, 2019 Stemline Therapeutics, Inc. (NASDAQ:STML), a biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported that Ivan Bergstein, M.D., Stemline’s CEO, will present at the 37th Annual J.P. Morgan Healthcare Conference on Wednesday, January 9, 2019 at 5:00 PM PT at the Westin St. Francis Hotel in San Francisco, CA (Press release, Stemline Therapeutics, JAN 2, 2019, View Source [SID1234532342]). A live webcast of the presentation can be viewed on the company’s website at www.stemline.com.

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About ELZONRIS
ELZONRIS (tagraxofusp), a CD123-directed cytotoxin, was approved by the Food and Drug Administration (FDA) on December 21, 2018 for the treatment of adult and pediatric patients, two years and older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). In November 2018, the European Medicines Agency (EMA) granted ELZONRIS accelerated assessment to the upcoming marketing authorization application (MAA), which is expected to be submitted in the first quarter of 2019. ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF) and other CD123 positive diseases.

About BPDCN
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.