On January 11, 2021 Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, reported that significant clinical milestones for 2021 and provided an overview of recent progress (Press release, Synlogic, JAN 11, 2021, View Source [SID1234573785]).

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"With three programs in clinical trials, multiple proof of concept opportunities, and a preclinical portfolio advancing rapidly towards the clinic, Synlogic is poised for success with a number of data readouts coming in 2021," said Aoife Brennan, M.B. Ch.B., Synlogic’s President and Chief Executive Officer. "2020 was a year we will not forget. Despite the external challenges, the Synlogic team moved our programs forward with grit and resilience. We enter 2021 with momentum and the opportunity to truly see the potential of novel Synthetic Biotic medicines to make a meaningful difference in patients’ lives."

Synlogic anticipates clinical proof of concept data in 2021 across two metabolic programs, SYNB1618 for the treatment of Phenylketonuria (PKU) and SYNB8802 for the treatment of Enteric Hyperoxaluria, as well as continued advancement of SYNB1891 for the treatment of solid tumors and lymphomas.

Execution Across Clinical Pipeline: Metabolic Programs

Progression of a proof of concept Phase 2 clinical trial of SYNB1618 for the treatment of Phenylketonuria (PKU)
SYNB1618 is an investigational drug composed of a Synthetic Biotic medicine designed to consume phenylalanine (Phe) in the gastrointestinal (GI) tract for the treatment of PKU in patients regardless of age or disease type.
A solid oral formulation of SYNB1618 has been shown to metabolize Phe in the GI tract in a healthy volunteer study.
The SynPheny-1 study evaluates plasma Phe lowering of SYNB1618 in adult PKU patients who do not benefit from, or do not tolerate, existing therapies such as Kuvan or Palynziq.
Synlogic anticipates data from SynPheny-1 will be available mid- 2021.

Progression of a Phase 1 clinical study of SYNB8802 for the treatment of Enteric Hyperoxaluria
SYNB8802 is an investigational drug composed of a Synthetic Biotic medicine designed to consume oxalate in the GI tract and lower urinary oxalate levels, potentially reducing kidney damage due to Enteric Hyperoxaluria.
In data presented at the American Society of Nephrology’s (ASN) 2020 Kidney Week, SYNB8802 was shown to reduce urinary oxalate in two animal models of Hyperoxaluria.
The Phase 1 clinical study evaluates the safety, tolerability, and potential for urinary oxalate lowering in healthy volunteers and patients.
The study has two parts: Part A is a multiple ascending dose study in healthy volunteers; Part B is a placebo controlled, cross-over design study in patients with Enteric Hyperoxaluria following Roux-n-Y gastric bypass surgery which provides an opportunity to demonstrate proof of concept.
Synlogic anticipates data from Part B of the study will be available mid-2021.
Execution Across Clinical Pipeline: Immunomodulation Programs

Advancement of SYNB1891 into combination arm dosing with PDL1 checkpoint inhibitor in ongoing Phase 1 study
SYNB1891 is an investigational drug composed of an intratumorally delivered Synthetic Biotic medicine designed to produce a STING agonist and act as a dual innate immune activator for the treatment of advanced solid tumors and lymphoma.
SYNB1891 is currently being evaluated in a Phase 1 study that has two parts:
Part A is a monotherapy arm that has enrolled four dose cohorts to date.
A maximum tolerated dose has not been reached and dose escalation continues.
Part A of the study has demonstrated target engagement and activation of the STING pathway.
Part B of the study will combine escalating dose levels of SYNB1891 with a fixed dose of the PD-L1 checkpoint inhibitor atezolizumab, to establish a recommended Phase 2 dose for the combination regimen.
Synlogic anticipates additional data from cohorts in both arms will be available in mid to late 2021.
Preclinical Roadmap

Synlogic continues to advance preclinical programs including additional effectors for immune-oncology; immune regulation targets for treatment of inflammatory bowel disease and other inflammatory disorders; and additional undisclosed rare metabolic diseases.
Further updates on these programs will be shared as they advance towards the clinic.
2020 Corporate Milestones

Synlogic strengthened leadership with the following appointments:
Synlogic appointed Dr. David Hava, Ph.D., as Chief Scientific Officer. Dr. Hava brings over a decade of senior experience in research and development to Synlogic, including deep academic expertise in pillars of synthetic biology.
Synlogic promoted Antoine ‘Tony’ Awad to Chief Operating Officer. Mr. Awad brings over 15 years of experience in the biotechnology and pharmaceutical industry with substantial experience in the development and manufacturing of novel therapeutics from pre-IND studies through global commercialization.
Synlogic appointed Michael Heffernan, seasoned entrepreneur and biopharmaceutical leader, and Dr. Michael Burgess, physician scientist and expert in translational development, to its board of directors.
Synlogic and Ginkgo Bioworks advanced their long-term strategic platform collaboration that provides expanded synthetic biology capabilities to Synlogic.
Ginkgo and Synlogic are collaborating on multiple efforts including metabolic and immunomodulation programs, and assessment of the potential application of Synthetic Biotics for vaccine development.
Synlogic ended the third quarter of 2020 with $102.0 million in cash, cash equivalents and short- and long-term investments and expects this will fund company operations through 2022 under its current plan.

On January 11, 2021 Surface Oncology (Nasdaq: SURF), a clinical-stage immuno-oncology company developing next-generation immunotherapies that target the tumor microenvironment, reported that Jeff Goater, chief executive officer, will participate in the upcoming H.C. Wainwright Bioconnect 2021 Conference, which will take place January 11–14 (Press release, Surface Oncology, JAN 11, 2021, View Source [SID1234573784]).

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The discussion will focus on Surface Oncology’s lead programs, SRF617 (targeting CD39) and SRF388 (targeting IL-27), as well as Surface’s emerging pre-clinical program, SRF114 (targeting CCR8). The discussion will be available on demand to conference attendees for the duration of the event beginning at 6:00 a.m. ET on Monday, January 11.

On January 11, 2021 ORIC Pharmaceuticals, Inc. (Nasdaq: ORIC), a clinical stage oncology company focused on developing treatments that address mechanisms of therapeutic resistance, reported program updates and announced key milestones for 2021, which are expected to substantially expand and advance the company’s clinical pipeline (Press release, ORIC Pharmaceuticals, JAN 11, 2021, View Source [SID1234573783]).

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"2020 was a transformational year for ORIC during which we significantly broadened the pipeline via internal discovery and business development efforts, expanded the team, and strengthened the balance sheet with the completion of an IPO and follow-on financing," said Jacob Chacko, M.D., president and chief executive officer. "These efforts have positioned us for a dynamic 2021, with our first data from two ongoing trials of our lead program ORIC-101 and three INDs/CTAs for our other product candidates, which represents a tremendous amount of development activity for a company at our stage."

Program Updates and 2021 Milestones

ORIC-101: Glucocorticoid Receptor (GR) Antagonist
ORIC-101 is a potent and selective GR antagonist, with two distinct mechanisms of action being evaluated in two Phase 1b trials in combination with: (1) Xtandi (enzalutamide) in metastatic prostate cancer and (2) Abraxane (nab-paclitaxel) in advanced or metastatic solid tumors.

The company announced today the completion of the Part I dose escalation portion of the Phase 1b trial of ORIC-101 in combination with enzalutamide in metastatic prostate cancer, by identifying the provisional recommended Phase 2 dose (RP2D) that will be used in the Part II expansion portion of the trial. The selection of the provisional RP2D was based upon the totality of safety, pharmacokinetic, and pharmacodynamic data demonstrating a well-tolerated regimen that achieved ORIC-101 exposures leading to demonstrable target engagement and GR inhibition. In the Part I dose escalation portion of the trial, patients were enrolled to evaluate daily dosing of ORIC-101 with doses ranging from 80 to 240 mg, in combination with daily dosing of 160 mg of enzalutamide. In the Part II dose expansion portion of the trial, up to 48 patients are expected to be enrolled and treated at the RP2D of 240 mg of ORIC-101 and 160 mg of enzalutamide on a continuous daily dosing schedule. Patients will be enrolled independent of GR status, with retrospective analysis of GR expression and other potentially predictive biomarkers. The company expects to report interim safety, efficacy, and translational data from this trial in the second half of 2021.
The Phase 1b trial of ORIC-101 in combination with nab-paclitaxel is now enrolling patients in the Part II dose expansion portion. In December 2020, the company announced the completion of the Part I dose escalation portion of ORIC-101 in combination with nab-paclitaxel in solid tumors, the selection of the RP2D, and the initiation of the dose expansion portion of the trial. For the Part II dose expansion portion of the trial, up to 132 patients are expected to be enrolled across four cohorts, including pancreatic ductal adenocarcinoma, ovarian cancer, triple negative breast cancer, and other advanced solid tumors. Patients in Part II of the trial will be treated at the RP2D of 160 mg of ORIC-101 continuous once daily dosing and 75 mg/m2 of nab-paclitaxel on days 1, 8, and 15 of a 28-day cycle, without requirement for prophylactic granulocyte colony-stimulating factor. Eligible patients must have previously progressed on a taxane-containing regimen and will be enrolled independent of baseline GR status, with retrospective analysis of GR expression and other potentially predictive biomarkers. The company expects to report interim safety, efficacy, and translational data from this trial in the first half of 2021.
ORIC-533: CD73 Inhibitor
ORIC-533 was designed to be a highly potent, orally bioavailable CD73 inhibitor and has demonstrated more potent adenosine inhibition in preclinical studies compared to an antibody approach and other small molecule CD73 inhibitors. ORIC-533 continues to progress in Investigational New Drug (IND) enabling studies and the company expects to file an IND with the Food and Drug Administration (FDA) in the first half of 2021. Having conducted a preclinical collaboration with an academic key opinion leader that generated compelling single agent activity in patient derived model systems in an undisclosed tumor type, the company plans to pursue a single agent clinical development path in this indication.

ORIC-944: PRC2 Inhibitor
ORIC-944, in-licensed in August 2020, is a potent and selective allosteric inhibitor of polycomb repressive complex 2 (PRC2), that targets its regulatory embryonic ectoderm development (EED) subunit and has demonstrated single agent efficacy in multiple enzalutamide-resistant prostate cancer models in preclinical studies. The company plans to conduct IND enabling studies and then file an IND with the FDA in the second half of 2021, with initial clinical development as a single agent in treatment-resistant prostate cancer.

ORIC-114: EGFR/HER2 Inhibitor
ORIC-114, in-licensed in October 2020, is a brain penetrant, orally bioavailable, irreversible inhibitor designed to selectively target EGFR and HER2 with high potency against exon 20 insertion mutations. ORIC-114 has demonstrated greater brain exposure in preclinical studies compared to other compounds being developed against exon 20 mutations and has shown strong antitumor activity in an EGFR-driven intracranial lung cancer model. The company plans to conduct IND enabling studies and then file a Clinical Trial Application (CTA) in South Korea in the second half of 2021.

Discovery Pipeline
In addition to the four product candidates, the company is leveraging its resistance platform in pursuit of multiple discovery research programs that focus on its expertise in precision oncology, hormone-dependent cancers, and key tumor dependencies. These programs highlight the company’s medicinal chemistry and structure-based drug design proficiency to target drivers of resistance in solid tumors like prostate, breast, and lung cancer that relapse with innate, acquired or bypass mechanisms of resistance. The company recently advanced one of these programs into lead optimization.

Anticipated 2021 Milestones

ORIC anticipates the following milestones in 2021:

ORIC-101: Report interim safety, efficacy, and translational data from ongoing combination trial with nab-paclitaxel in the first half of 2021
ORIC-101: Report interim safety, efficacy, and translational data from ongoing combination trial with enzalutamide in the second half of 2021
ORIC-533: File an IND in the first half of 2021
ORIC-944: File an IND in the second half of 2021
ORIC-114: File a CTA in the second half of 2021
Present additional preclinical and translational research data on ORIC-101, ORIC-533, ORIC-944, and ORIC-114 at scientific conferences in 2021
Financial Guidance

Cash, cash equivalents and marketable securities totaled $293.6 million as of December 31, 2020, which included gross proceeds of $133.3 million from the follow-on financing completed in November 2020. The company expects its cash, cash equivalents and marketable securities will be sufficient to fund its current operating plan into the second half of 2023.

Presentation and Webcast

Jacob Chacko, M.D., president and chief executive officer, will present a corporate overview at the 39th J.P. Morgan Healthcare Conference beginning at 12:40 p.m. PT on Tuesday, January 12, 2021. A live webcast will be available through the investor section of the company’s website at View Source A replay of the webcast will be available for 90 days following the event.

On January 11, 2021 NeuBase Therapeutics, Inc. (NASDAQ: NBSE) ("NeuBase" or the "Company"), a biotechnology company accelerating the genetic revolution using a new class of synthetic medicines, reported that Dietrich A. Stephan, Ph.D., Chief Executive Officer of NeuBase, will present a corporate overview during the H.C. Wainwright Virtual BioConnect Conference, which is being held from January 11 – 14, 2021 (Press release, NeuBase Therapeutics, JAN 11, 2021, View Source [SID1234573782]). A webcast of the on-demand presentation will be available beginning today at 6:00 a.m. Eastern Time and archived on the NeuBase website, www.neubasetherapeutics.com.

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On January 11, 2021 Invitae Corporation (NYSE: NVTA), a leading medical genetics company, reported preliminary unaudited full-year 2020 results, reporting strong growth in volume and revenue, signaling continued momentum into 2021 (Press release, Invitae, JAN 11, 2021, View Source [SID1234573781]).

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Invitae’s (NVTA) mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. www.invitae.com (PRNewsFoto/Invitae Corporation)

"We are very pleased by the acceleration of our business in 2020, particularly in light of the impact of the COVID-19 pandemic on the healthcare system throughout the year," said Sean George, Ph.D., co-founder and chief executive officer of Invitae. "Our results underscore the strength of our customer relationships, our continued ability to execute and the benefits of the investments we’ve made in expanding our menu, services and platform. The growth in testing during this time of unrelenting stress on the healthcare system further demonstrates the expanding value genetic information is providing patients as they and their physicians face health challenges and decisions."

Preliminary, unaudited financial results and other metrics for 2020

Generated revenue of more than $278 million in 2020, an approximate 28% increase from $217 million in 2019
Reported billable volume of more than 655,000 in 2020, an approximate 40% increase from billable volume of 469,000 in 2019
Announced year-end 2020 cash, cash equivalents, restricted cash and marketable securities of more than $360 million as of December 31, 2020
Outlook for 2021
Looking ahead to 2021 and factoring in the continued impact associated with the COVID-19 pandemic, Invitae reiterates its stated outlook for revenue growth targets of 50% – 60% annual growth over the next few years consistent with its view at the time of the announcement of the ArcherDX acquisition. Invitae anticipates generating revenue in excess of $450 million in 2021.

"We are confident we will continue growing rapidly in 2021, even as we navigate the continued pressure the pandemic is placing on clinicians," said Dr. George. "Our momentum coming out of 2020 and the underlying strength and breadth of our platform enable us to recognize and overcome these challenges while continuing to drive the acceleration of the adoption of genetics in mainstream medicine as the new standard of care."

Invitae has not completed preparation of its financial statements for the fourth quarter or full year 2020. The preliminary, unaudited results presented in this press release for the year ended December 31, 2020 are based on current expectations and are subject to adjustment. Actual results may differ materially from those disclosed in this press release. Invitae will report its full financial results and other metrics during its fourth quarter and year-end 2020 conference call in February.

Invitae’s Presentation at 39th Annual J.P. Morgan Healthcare Conference
Invitae will present at the 39th Annual J.P. Morgan Healthcare Conference on Tuesday, January 12, 2021 at 10:50 a.m. Eastern/7:50 a.m. Pacific. Following the company presentation, management will participate in a breakout session at 11:10 a.m. Eastern/8:10 a.m. Pacific. The 2021 J.P. Morgan Healthcare Conference is being held virtually via webinar. A live webcast of both the presentation and the breakout session may be accessed at the following direct link or by visiting the investors section of the company’s website at ir.invitae.com. Public listeners can access an audio and slide recording of the session, which will be available shortly after the conclusion of the presentation and breakout session on the investors section of the company’s website at ir.invitae.com.