Intrexon Announces Presentations at Upcoming Investor Conferences

On January 2, 2019 Intrexon Corporation (NASDAQ: XON), a leader in the engineering and industrialization of biology to improve the quality of life and health of the planet, reported that the company and its subsidiaries will present at upcoming investor conferences in San Francisco (Press release, Intrexon, JAN 2, 2019, View Source [SID1234532340]).

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Pieter Rottiers, PhD, Chief Executive Officer of ActoBio Therapeutics, will present at the Biotech Showcase on January 8th, 2019 at 9:00 AM PT at the Hilton San Francisco Union Square.

Thomas D. Reed, PhD, Chief Science Officer of Intrexon and Amit Patel, MD, Founder of Xogenex, will present at the Biotech Showcase on January 9th, 2019 at 8:45 AM PT at the Hilton San Francisco Union Square.

Randal J. Kirk, Chairman and Chief Executive Officer of Intrexon, will present at the 37th Annual J.P. Morgan Healthcare Conference on Wednesday, January 9th, 2019 at 11:30 AM PT at the Westin St. Francis Hotel. A live webcast of the presentation will be available on the Investors section of Intrexon’s website at View Source, and a replay of the webcast will be available for 30 days following the event.

Additionally, as previously announced, Helen Sabzevari, PhD, President of Precigen, will be presenting a first-look at Precigen’s preclinical and clinical portfolio at an investor event Monday, January 7, 2019 at 3:00pm PT. The presentation will be available via live audio webcast through Intrexon’s website at View Source. Spaces are available for individuals who would like to participate in-person at the onsite event. For in-person participation, RSVP is required, and all RSVPs must be received at [email protected] by January 6, 2019 at 5:00 pm PT.

FLX Bio to Present at 37th Annual J.P. Morgan Healthcare Conference

On January 2, 2018 FLX Bio, Inc., a clinical-stage, biopharmaceutical company focused on the development of oral small-molecule drugs that target immune drivers of cancer and other immune-related disorders, reported that Brian Wong, M.D., Ph.D., CEO will present at the 37th Annual J.P. Morgan Healthcare Conference on Wednesday, January 9, 2019 at 7:30 a.m. Pacific Standard Time in San Francisco, CA (Press release, FLX Bio, JAN 2, 2019, View Source [SID1234532361]). Dr. Wong will provide a corporate update and company overview.

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Vertex to Present at the J.P. Morgan Healthcare Conference on January 7

On January 2, 2019 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported that management will present at the J.P. Morgan Healthcare Conference onMonday, January 7, 2019 at 2:30 p.m. ET (11:30 a.m. PT) (Press release, Vertex Pharmaceuticals, JAN 2, 2019, View Source [SID1234532399]).

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The audio portion of management’s remarks can be accessed live through Vertex’s website, www.vrtx.com, in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company’s website.

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.