On January 9, 2020 Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company applying synthetic biology to beneficial microbes to develop novel, living medicines, reported that key objectives and anticipated milestones for 2020 and provided an overview of recent progress (Press release, Synlogic, JAN 9, 2020, View Source [SID1234552906]).

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"In 2020 we look forward to advancing our platform and our growing development stage pipeline of Synthetic BioticTM medicines – advancing our phenylketonuria program and additional oral metabolic programs that build on the critical capabilities we have established in development and manufacturing, as well as advancing our first clinical program in oncology," said Aoife Brennan, M.B. Ch.B., Synlogic’s president and chief executive officer. "We believe that Synthetic Biotic medicines have potential across multiple diseases and conditions. Our expanded collaboration with Ginkgo Bioworks, strong balance-sheet and solid momentum out of our discovery and development efforts position us well to execute on our near-term clinical plans."

2020 Goals and Milestones
Clinical Programs

SYNB1618: SYNB1618 is an orally delivered, Synthetic Biotic medicine designed to consume phenylalanine (Phe) in the gastrointestinal (GI) tract for the treatment of phenylketonuria (PKU) in patients regardless of age or disease type.
In the first half of 2020, Synlogic expects to initiate a Phase 2 clinical trial to evaluate the Phe-lowering potential of its solid formulation of SYNB1618 in patients with PKU. In addition, the study is expected to provide valuable information to validate predictive mathematical and preclinical modeling.
Strain development and optimization work is being carried out under Synlogic’s 2019 research and development agreement with Ginkgo Bioworks.
SYNB1891: Synlogic’s first clinical immuno-oncology (IO) program, SYNB1891, is an intratumorally delivered Synthetic Biotic medicine designed to produce a STING agonist and act as a dual innate immune activator for the treatment of refractory solid tumors and lymphoma.
SYNB1891 is being evaluated as a monotherapy in a Phase 1 open-label, multicenter, dose escalation clinical trial (NCT04167137) in patients with refractory solid tumors and lymphoma. Three U.S. sites have been activated to enroll and the first subject has been dosed. Synlogic expects to have data from the monotherapy arm of this study in 2020.
After establishing a maximum tolerated dose (MTD) for SYNB1891, Synlogic will initiate a second arm of the trial in which subjects will receive escalating dose levels of SYNB1891 in combination with a fixed dose of the checkpoint inhibitor, atezolizumab (Tecentriq), to establish a recommended dose for the combination regimen.
Pre-clinical data and early pipeline programs:
Synlogic has advanced two new preclinical programs onto its development pipeline, for the treatment of secondary hyperoxaluria and MSUD, respectively. Strain development and optimization work is being carried out under Synlogic’s 2019 research and development agreement with Ginkgo Bioworks.
Synlogic expects to publish and present data at major scientific and medical meetings throughout the year demonstrating the breadth and potential of its Synthetic Biotic platform.
Corporate

Synlogic ended the third quarter of 2019 with $138.7 million in cash, cash equivalents and short- and long-term investments and expects that this will fund Company operations through 2021 under its current plan.
The Company will continue to explore additional strategic opportunities to maximize the potential of its Synthetic Biotic platform.
2019 Accomplishments and Highlights
Clinical Pipeline

SYNB1618
Presentation of positive data from Phase 1/2a study of SYNB1618 in patients with phenylketonuria (PKU) using the liquid formulation as well as modeling work to estimate target dosing of SYNB1618. SYNB1618 was well tolerated in healthy volunteers and patients with PKU and the data demonstrated equivalent SYNB1618-dependent Phe-consumption from the GI tract in both populations.
Development of a solid oral formulation of SYNB1618 that is stable at room temperature. Synlogic executed a bridging study in healthy volunteers of a new solid oral formulation to identify an MTD that could be used to evaluate lowering of blood Phe levels in PKU patients based on Synlogic’s modeling data. Data from this study will be presented at an appropriate medical meeting in 2020. Synlogic expects to advance SYNB1618 into a Phase 2 study in patients in the first half of 2020.
SYNB1891:
Initiation of a Phase 1 open-label, multicenter, dose escalation trial of its STING-agonist producing bacterial strain, SYNB1891, for the treatment of refractory solid tumors and lymphoma.
Corporate

Establishment of a collaboration with Ginkgo Bioworks that provides expanded synthetic biology capabilities and strengthened Synlogic’s balance sheet. In June 2019, Synlogic and Ginkgo entered into a long-term strategic platform collaboration. Under the agreement Ginkgo invested $80.0 million in Synlogic at a premium to market. Synlogic is using Ginkgo’s cell programming platform for building and testing microbial strains to accelerate progression of early preclinical leads to drug candidates optimized for further clinical development. Under the terms of the agreement, Synlogic paid $30.0 million to Ginkgo for synthetic biology services and has exclusive rights to any Synthetic Biotic medicines that it develops as part of the collaboration and to intellectual property covering such products.
Strengthened Synlogic’s executive leadership with the following appointments:
Richard Riese, M.D. Ph.D., joined Synlogic in September 2019 as chief medical officer assuming responsibilities for all clinical and regulatory functions from current president and CEO, Aoife Brennan. Prior to joining Synlogic, he served as Vice President, Clinical Development at Alnylam Pharmaceuticals where he led clinical development projects in several areas across Alnylam’s rare disease portfolio.
Gregg Beloff was appointed as Synlogic’s interim chief financial officer, in October 2019. Mr. Beloff has more than 20 years of experience in the life sciences industry and brings significant expertise in operational management, strategic planning, corporate and business development, fundraising and mergers and acquisitions.
Patricia Hurter, Ph.D., chief executive officer of Lyndra Therapeutics, was appointed to the Synlogic board of directors in January 2019. Dr. Hurter previously served as Senior Vice President at Vertex. Prior to joining Vertex, Dr. Hurter was Director, Formulation Design and Characterization for Merck.
Established a clinical collaboration with Roche that enables evaluation of SYNB1891, in combination with atezolizumab in the second arm of Synlogic’s ongoing Phase 1 study in patients with advanced solid tumors and lymphoma.
Established GMP manufacturing capabilities and manufactured solid and liquid formulations of clinical trial material for its oral and intra-tumoral programs, respectively.
Advanced collaboration with AbbVie to develop Synthetic Biotic-based treatments for inflammatory bowel disease resulting in payment to Synlogic of a $2.5 million milestone in March 2019.
Published first in human clinical data of a Synthetic Biotic medicine (SYNB1020) as well as preclinical data from the program in Science Translational Medicine.

On January 9, 2020 ProMIS Neurosciences, Inc. (TSX: PMN) (OTCQB: ARFXF), a biotechnology company focused on the discovery and development of antibody therapeutics targeting toxic oligomers implicated in the development of neurodegenerative diseases, reported its participation in the Sachs 3rd Annual Neuroscience Innovation Forum being held on January 12, 2020 at the Marine’s Memorial Club, San Francisco, CA (Press release, ProMIS Neurosciences, JAN 9, 2020, View Source [SID1234552903]).

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ProMIS’ President and CEO, Elliot Goldstein, MD will provide overviews of its novel drug discovery and development programs for Alzheimer’s disease, Parkinson’s disease and ALS (amyotrophic lateral sclerosis). The audio webcast and slides of Dr. Goldstein’s presentation will be available approximately one week following the conference presentation on ProMIS’ web site View Source

On January 9, 2020 Compugen Ltd. (Nasdaq: CGEN), a clinical-stage cancer immunotherapy company and a leader in predictive target discovery, reported an update on upcoming milestones for its COM701 and COM902 clinical programs and highlighted 2019 accomplishments (Press release, Compugen, JAN 9, 2020, View Source [SID1234552901]).

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"2019 has been a transformative year for Compugen, including our first presentation of preliminary clinical data from our internally discovered and developed lead asset, COM701," said Anat Cohen-Dayag, Ph.D., President and CEO of Compugen. "The report of encouraging initial signs of anti-tumor activity of COM701 monotherapy in a challenging, heavily pretreated population marked an important milestone for the Company. In 2020, we plan to continue our clinical advancement with additional expected milestones, including initial data from the Phase 1 dose escalation study of COM701 with Opdivo, as well as the initiation of a Phase 1 clinical study for COM902, our second internally discovered and developed asset. We are proud of our strong execution and look forward to continued progress as we advance multiple clinical studies to potentially expand the reach of cancer immunotherapy treatments."

2020 Anticipated Milestones:

Initiate and complete enrollment in the COM701 Phase 1 monotherapy expansion cohorts in biomarker-driven indications, including non-small cell lung, ovarian, breast and endometrial cancers

Present initial data from Phase 1 combination dose escalation study evaluating COM701 with Opdivo in 2H 2020

Initiate Phase 1 combination expansion cohorts for COM701 with Opdivo

Initiate COM902 Phase 1 study in patients with advanced malignancies in early 2020

2021 Anticipated Milestones:

Present initial data from COM701 Phase 1 monotherapy expansion cohort study in 1H 2021

Present initial data from COM902 Phase 1 monotherapy dose escalation study

Recent Accomplishments:

Presented encouraging preliminary data from ongoing Phase 1 monotherapy dose escalation study of COM701 demonstrating that COM701 is well-tolerated with initial signs of anti-tumor activity in a heavily pretreated, refractory patient population at SITC (Free SITC Whitepaper) 2019

Completed enrollment of COM701 Phase 1 monotherapy dose escalation at Q3 weekly dosing schedule; enrollment in COM701 Phase 1 monotherapy dose escalation cohort at Q4 weekly dosing schedule is on-going

Completed enrollment of Q3 weekly dosing schedule of Phase 1 combination dose escalation of COM701 with fixed-dose Opdivo. Enrollment status of Q4 weekly dosing schedule will be presented at a trial-in-progress poster at the ASCO (Free ASCO Whitepaper)-SITC Clinical Immuno-Oncology Symposium on February 6, 2020

Received FDA clearance for COM902 Investigational New Drug Application

Strengthened COM701 and COM902 intellectual property portfolio with new composition of matter and method of use patents

Added computational discovery and business development capabilities and expertise to senior management team

About COM701
COM701 is a humanized antibody that binds with high affinity to PVRIG, a novel immune checkpoint target candidate discovered by Compugen, blocking the interaction with its ligand, PVRL2. Blockade of PVRIG by COM701 has demonstrated potent, reproducible enhancement of T cell activation, consistent with the desired mechanism of action of activating T cells in the tumor microenvironment to generate anti-tumor immune responses. In addition, COM701 combined with antagonist anti-PD-1 antibodies has demonstrated synergistic effects in enhancing human T cell stimulation and inhibiting tumor growth in murine models, indicating an intersection of the PVRIG and PD-1 inhibitory pathways and the potential of these combinations to further enhance immune response against tumors.

PVRIG and TIGIT constitute parallel immune checkpoint pathways that counteract DNAM, a costimulatory molecule on T cells and NK cells. Preclinical data for COM701 suggest that PVRIG may be a dominant checkpoint pathway in diverse patient populations with tumors that express elevated PVRL2, the ligand of PVRIG, as compared to expression of PVR, the ligand of TIGIT. This includes patients with breast, endometrial, and ovarian cancers. In addition, expression studies show that PVRIG, TIGIT, and their respective ligands, are expressed in a broad variety of tumor types, such as those noted above, as well as lung, kidney, and head & neck cancers. In these tumors the blockade of both TIGIT and PVRIG may be required to sufficiently stimulate an anti-tumor immune response, with or without additional PD-1 pathway blockade.

COM701 is in a Phase 1 clinical trial in patients with advanced solid tumors, to evaluate monotherapy and combination therapy with a PD-1 inhibitor. The Phase 1 open-label trial is designed to assess the safety and tolerability of COM701 monotherapy as well as COM701 with Bristol-Myers Squibb’s Opdivo (nivolumab) in patients with advanced solid tumors. Secondary endpoints include preliminary anti-tumor activity, pharmacokinetics and pharmacodynamics of COM701 monotherapy as well as COM701 in combination with Opdivo in patients with selected tumor types, including non-small cell lung cancer, ovarian cancer, breast cancer and endometrial cancer. The Phase 1 study, which is expected to enroll approximately 140 patients, is currently recruiting in the United States. The Phase 1 dose escalation study showed that COM701 is well-tolerated through 10 mg/kg with no dose-limiting toxicities observed. Furthermore, data showed preliminary signs of anti-tumor activity in heavily pretreated patient population (with a median of seven prior anticancer therapies (range of 2-15)), with best timepoint response of stable disease (SD)/disease control rate reported in 9 of 13 patients (69%). Additional information is available at www.clinicaltrials.gov (NCT03667716).

About COM902
COM902, a high affinity, fully human antibody targeting TIGIT, is being developed for combination treatment with COM701. COM902 was shown to have superior binding affinity to T cells with similar and or greater in vitro function compared to several clinical anti-TIGIT antibodies. COM902 is a mouse-cross reactive Ab which inhibited tumor growth and increased survival when combined with anti-PVRIG or anti-PD-L1 antibodies in in vivo studies. Preclinical data demonstrate that TIGIT inhibition, either alone or in combination with other checkpoint inhibitors, can enhance T cell activation and increase anti-tumor immune responses. Parallel inhibition of TIGIT and PVRIG, the two coinhibitory arms of the DNAM axis, results in synergistic effects on effector T cell function and tumor growth inhibition in various model systems that can be further increased with the addition of PD-1 blockade. Based on preclinical data these combinations may be clinically important for enhancing anti-tumor immune response and expanding the patient population responsive to checkpoint inhibition.

Compugen discovered TIGIT in 2009 with its immune checkpoint computational discovery platform through which PVRIG was also discovered. The TIGIT discovery was published by Compugen in October 2009 in the Proceedings of the National Academy of Sciences (PNAS).

Compugen plans to initiate a Phase 1 study in patients with advanced malignancies in early 2020. The study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary anti-tumor activity of COM902. The study is planned to be conducted at multiple centers in the United States.

On January 9, 2020 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for the treatment of cancer, reported its participation at the 38th Annual J.P. Morgan Healthcare Conference (Press release, Kura Oncology, JAN 9, 2020, View Source [SID1234552899]). Troy Wilson, Ph.D., J.D., President and Chief Executive Officer, is scheduled to present an overview of the company on Thursday, January 16, 2020 at 10:00 a.m. PT / 1:00 p.m. ET, followed by a Q&A session at 10:30 a.m. PT / 1:30 p.m. ET. The conference will be held from January 13-16, 2020 in San Francisco.

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Live audio webcasts of the presentation and Q&A session will be available in the Investors section of Kura’s website at www.kuraoncology.com, with an archived replay available for 30 days following the event.

On January 9, 2020 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a biotechnology company developing novel immunotherapy treatments for cancer and autoimmune diseases, provides an update on its TACTI-002 and AIPAC studies for its lead product candidate, eftilagimod alpha ("efti" or "IMP321") (Press release, Immutep, JAN 9, 2020, View Source [SID1234552898]).

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TACTI-002 – Phase II study

The requisite number of predefined patient responses was observed in stage 1 of Part C. The decision by the DMC to recommend opening stage 2 recruitment follows its review of preliminary safety and efficacy data and is based on a predefined efficacy threshold.

This allows the Company to now proceed with the recruitment of an additional 19 patients, forming stage 2 of Part C of the study, having now also completed recruitment of the 18 HNSCC patients for stage 1. The staged approach to patient enrolment is based on the study’s Simon’s two-stage clinical trial design.

Stage 1 of Part A (first line Non-Small Cell Lung Cancer, NSCLC) was expanded in September 2019. Recruitment is ongoing for Part B (second line NSCLC) with 10 out of 23 patients now participating, and for stage 2 of Part A (first line NSCLC), where 4 out of 19 patients are now participating. The Company expects to report more mature data from TACTI-002 in Q1 CY20.

TACTI-002 is being conducted in collaboration with Merck & Co., Inc., Kenilworth, NJ, USA (known as "MSD" outside the United States and Canada).

Immutep CSO and CMO, Dr Frederic Triebel said: "The study is progressing well as the expansion of Part C to include 19 additional patients with second line HNSCC marks the second out of three parts of our TACTI-002 study to be expanded, having already observed the pre-determined number of partial responses in Parts A and C patients."

AIPAC – Phase IIb study in metastatic breast cancer

The Company continues to progress its AIPAC trial which evaluates efti in combination with chemotherapy in 227 metastatic breast cancer patients in a randomized, double blinded, placebo-controlled phase IIb clinical trial. The first progression-free survival read-out remains on track for Q1 CY20 and is expected to be reported in March 2020.

Immutep Limited, Level 12, 95 Pitt Street, Sydney NSW 2000

ABN: 90 009 237 889

About TACTI-002

TACTI-002 (Two ACTive Immunotherapies) is being conducted in collaboration with Merck & Co., Inc., Kenilworth, NJ, USA (known as "MSD" outside the United States and Canada). The study is evaluating the combination of efti with MSD’s KEYTRUDA (or pembrolizumab, an anti-PD-1 therapy) in up to 109 patients with second line head and neck squamous cell carcinoma or non-small cell lung cancer in first and second line. The trial is a Phase II, Simon’s two-stage, non-comparative, open-label, single-arm, multicentre clinical study that is taking place in up to 13 study centres across the U.S., Europe and Australia.