On March 17, 2022 Synlogic, Inc. (Nasdaq: SYBX), a clinical-stage biotechnology company developing medicines for metabolic and immunological diseases through its proprietary approach to synthetic biology, reported financial results for the fourth quarter and full year ending December 31, 2021 and provided an update on pipeline programs (Press release, Synlogic, MAR 17, 2022, View Source [SID1234610274]).

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"2021 was a transformational year for Synlogic highlighted by proof of concept for our lead program in phenylketonuria (PKU)," said Aoife Brennan, M.B. Ch.B., Synlogic President and Chief Executive Officer. "Despite the devastating burden of this disease, a large majority of people living with PKU remain in need of an efficacious, safe, oral medication. We look forward to sharing Phase 2 data in the first half of this year as we continue plans to initiate Phase 3 later this year. We are also excited for clinical data readouts from our homocystinuria (HCU) and enteric hyperoxaluria programs, as well as research progress including our ongoing collaborations with Ginkgo Bioworks and Roche."

Recent Portfolio Highlights

SYNB1618 and SYNB1934: In Development for the Treatment of PKU

PKU is a rare metabolic disease that can result in irreversible cognitive and neurological damage. Approximately 75% of people with PKU remain untreated, reflecting limitations of current therapies. SYNB1618 and SYNB1934 are non-systemically absorbed, oral drug candidates being studied for both adjunctive and monotherapy treatment of PKU.
In September 2021, Synlogic reported positive interim results from the Phase 2 SynPheny-1 study for SYNB1618, with clinically meaningful and statistically significant reduction of plasma Phe levels in patients with PKU. Based on this achieved proof of concept, the Company began preparations for Phase 3 development.
In parallel, Synlogic added an arm to the Phase 2 Synpheny-1 study for SYNB1934, a next-generation drug candidate for PKU designed for greater potency in Phe reduction. This additional arm in Synpheny-1 followed positive results with SYNB1934 in healthy volunteer studies.
In H1 2022, Synlogic expects to announce additional data from the Synpheny-1 study and, based on those findings, which of the two candidates will proceed to Phase 3 and potential commercialization.
SYNB1353: In Development for the Treatment of HCU

In November 2021, Synlogic and Ginkgo Bioworks announced the nomination of SYNB1353, a drug candidate designed to consume methionine for the treatment of HCU. Like PKU, HCU is an inherited rare metabolic disease caused by an inborn error of metabolism that results in significant disease burden, including intellectual disability and thromboembolism.
During 2022, the Company plans to submit an investigational new drug application (IND), initiate clinical trials and, in H2 2022, report Phase 1 healthy volunteer data for SYNB1353.
SYNB8802: In Development for the Treatment of Enteric Hyperoxaluria

Enteric hyperoxaluria, a leading cause of recurrent kidney stones, is a chronic, progressive disease that can lead to chronic kidney disease (CKD) and end-stage renal disease (ESRD), and for which there is currently no FDA-approved treatment.
Synlogic demonstrated proof of mechanism for SYNB8802 in enteric hyperoxaluria in 2021, and it is currently being evaluated in patients who have undergone Roux-en-Y gastric bypass surgery, with proof-of-concept data from this study expected in 2022.
Preclinical Pipeline

Synlogic plans to advance research programs to address metabolic and immunologic diseases, including wholly owned programs targeting inflammatory bowel disease and hyperuricemia (gout), diseases for which the Synthetic Biotic clinical profile of orally-administered, non-systemically absorbed biotherapeutics is particularly compelling.
The Company’s research collaboration with Roche to develop a Synthetic Biotic for the treatment of inflammatory bowel disease continues to progress.
Synlogic and Ginkgo continue to advance their long-term strategic platform collaboration with multiple undisclosed metabolic and immunology programs now in preclinical development.

Corporate Updates

Earlier this month, the company announced the appointment of Michael Jensen as Chief Financial Officer. Mr. Jensen brings extensive and diversified experience within global pharmaceutical and medical device companies to Synlogic, spanning analytics, financial management, information systems, and operations. Mr. Jensen was formerly the CFO of Intrinsic Therapeutics.

Fourth Quarter 2021 Financial Results

As of December 31, 2021, Synlogic had cash, cash equivalents, and marketable securities of $136.6 million.

Revenue was $0.6 million for the three months ended December 31, 2021. Revenue in 2021 was associated with the ongoing research collaboration with Roche for the discovery of a novel Synthetic Biotic medicine for the treatment of IBD. There was no revenue for the three months ended December 31, 2020.

Research and development expenses were $11.9 million for the three months ended December 31, 2021, compared to $11.4 million for the corresponding period in 2020.

General and administrative expenses for the three months ended December 31, 2021 were $3.9 million compared to $3.3 million for the corresponding period in 2020.

For the three months ended December 31, 2021, Synlogic reported a consolidated net loss of $15.1 million, or $0.21 per share, compared to a consolidated net loss of $14.6 million, or $0.39 per share, for the corresponding period in 2020.

Full Year 2021 Financial Results

Revenues were $1.8 million for the year ended December 31, 2021, compared to $0.5 million for the same period in 2020. Revenue in 2021 was associated with the ongoing research collaboration with Roche for the discovery of a novel Synthetic Biotic medicine for the treatment of IBD. Revenue in 2020 was due to the prior collaboration with AbbVie, which was terminated in May 2020. Operating expenses were $62.5 million for the year ended December 31, 2021, compared to $61.0 million for the same period in 2020. For the year ended December 31, 2021, consolidated net loss was $60.6 million, or $1.09 per share, compared to a consolidated net loss of $59.2 million, or $1.65 per share, for the year ended December 31, 2020.

Financial Outlook

Based upon its current operating plan and balance sheet as of December 31, 2021, Synlogic expects to have sufficient cash to be able to fund operations into 2024.

Investor Conference Presentation

Today, Synlogic will participate in Oppenheimer’s 32nd Annual Healthcare Conference. Dr. Brennan will present virtually at 10:40 am ET.

A live webcast of the presentation will be accessible under the "Event Calendar" in the Investors & Media section of the Company’s website. An archived version will also be available after the presentation on the Synlogic website.

Conference Call & Webcast Information

Synlogic will host a conference call and live webcast at 8:30 a.m. ET today, March 17, 2022. To access the live webcast, please visit the "Event Calendar" page within the Investors and Media section of the Synlogic website. Investors may listen to the call by dialing +1 (844) 815-2882 from locations in the United States or +1 (213) 660-0926 from outside the United States. The conference ID number is 1719849. A replay will be available for 30 days on the Investors and Media section of the Synlogic website.

On March 17, 2022 ORIC Pharmaceuticals, Inc. (Nasdaq: ORIC), a clinical stage oncology company focused on developing treatments that address mechanisms of therapeutic resistance, reported that management will host a conference call and webcast to discuss its fourth quarter and full year 2021 financial results and provide an operational update on Monday, March 21, 2022, at 5:00 p.m. ET (Press release, ORIC Pharmaceuticals, MAR 17, 2022, View Source [SID1234610270]).

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Webcast and Conference Call

To participate in the conference call, please dial (833) 651-0991 (domestic) or (918) 922-6080 (international) and refer to conference ID 8637367. A live webcast and audio archive of the conference call will be available through the investor section of the company’s website at www.oricpharma.com. The webcast will be available for replay for 90 days following the presentation.

On March 17, 2022 Onconova Therapeutics, Inc. (NASDAQ: ONTX), ("Onconova"), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, reported financial results for the twelve months ended December 31, 2021, and provided a business update (Press release, Onconova, MAR 17, 2022, View Source [SID1234610269]).

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Highlights for the fourth quarter of 2021 and recent weeks include:

The Phase 1 solid tumor trials of narazaciclib in the United States and China are ongoing with no dose-limiting toxicities observed to date. The trial in the United States is currently enrolling into its fourth dose cohort, which is evaluating a 160 mg daily dose administered orally on each day of a 28-day treatment cycle. The trial in China is enrolling into its fifth dose cohort, which is evaluating a 200 mg dose administered orally once a day on days 1-21 of a 28-day treatment cycle. A protocol amendment is being prepared to enable further dose escalation in the trial in China.
Rigosertib’s investigator-sponsored program has seen progress across multiple trials. This is highlighted by continued enrollment in the dose expansion portion of the Phase 1/2a study of oral rigosertib plus nivolumab in patients with KRAS+ non-small cell lung cancer (NSCLC). Additionally, recent single-patient data from a heavily pre-treated participant in the Phase 2 trial of rigosertib monotherapy in advanced squamous cell carcinoma associated with recessive dystrophic epidermolysis bullosa (RDEB-associated SCC) showed a durable complete response without signs of metastatic disease.
The Company strengthened its team with the appointment of Adar Makovski Silverstein, Ph.D., as Director, Corporate Development.
Management Commentary

"The advancements made across our pipeline over the past months have us on track for an exciting 2022," said Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova. "We continue to be pleased with the safety findings from narazaciclib’s ongoing Phase 1 program and remain on track to identify a recommended Phase 2 dose in the second half of the year. Narazaciclib shows low nanomolar activity against kinases associated with tumor growth, metastasis, and potentially drug resistance, which differentiates it from currently approved CDK 4/6 inhibitors. We thus believe it may have broad therapeutic potential in monotherapy and combination settings, both in patients showing resistance to approved CDK 4/6 inhibitors and in indications where these agents are not currently approved. Narazaciclib’s clinical development plan is designed to test this hypothesis, and we look forward to its continued progress."

Dr. Fruchtman continued, "Beyond our lead program in narazaciclib, we also reported compelling preliminary clinical data from an investigator-sponsored study of rigosertib in RDEB-associated SCC. These data showed a sustained complete response in a patient that was unresponsive to several prior treatments. Though from a single patient, we believe these data represent a potentially powerful observation in this ultra-rare indication given its invariably fatal nature and the lack of effective therapies. Rigosertib’s additional investigator-sponsored trials have also shown strong progress, as we are on track to report updated data from the Phase 1/2a study evaluating rigosertib-nivolumab combination therapy in KRAS+ NSCLC this year, and to open a Phase 2 metastatic melanoma trial of oral rigosertib plus pembrolizumab in the first half. Looking ahead, we will continue to leverage the collaborations that form the basis of these trials to pursue rigosertib’s clinical development across multiple indications while remaining primarily focused on our lead narazaciclib program."

Full Year Financial Results

Cash and cash equivalents as of December 31, 2021, were $55.1 million, compared with $19.0 million as of December 31, 2020. The Company believes that its cash and cash equivalents will be sufficient to fund ongoing clinical trials and business operations for at least two years.

Research and development expenses were $7.3 million for 2021, compared with $16.9 million for 2020. The decrease was primarily related to the Company’s focus on its Phase 1 program with narazaciclib, following the completion of the Phase 3 INSPIRE study in 2020.

General and administrative expenses were $9.4 million for 2021, compared with $8.3 million for 2020. The increase was primarily related to costs related to special and annual general meeting expenses in the 2021 period.

Net loss for 2021 was $16.2 million, or $0.96 per share on 16.8 million weighted average shares outstanding, compared with a net loss of $25.2 million, or $2.17 per share for 2020 on 11.6 million weighted average shares outstanding.

Conference Call and Webcast

Onconova will host an investment community conference call today beginning at 4:30 p.m. Eastern Time, during which management will discuss financial results for 2021, provide a business update, and answer questions. Interested parties can participate by dialing (855) 428-5741 (domestic callers) or (210) 229-8823 (international callers) and using conference ID 8097917.

A live webcast of the conference call will be available in the Investors & Media section of the Company’s website at www.onconova.com. A replay of the webcast will be available on the Onconova website for 90 days following the call.

On March 17, 2022 Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC) reported the publication of preclinical and patient data on pelareorep in the peer-reviewed journal Immunology (Press release, Oncolytics Biotech, MAR 17, 2022, View Source [SID1234610268]). The paper, entitled "Oncolytic virus treatment differentially affects the CD56dim and CD56bright NK cell subsets in vivo and regulates a spectrum of human NK cell activity," was published in collaboration with researchers at several prestigious institutions, including the University of Leeds School of Medicine and the Institute of Cancer Research, London. A link to the paper can be found by clicking here.

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Described in the paper are in vitro studies evaluating pelareorep’s effects on Natural Killer (NK) cells as well as analyses of blood samples from patients with colorectal liver metastases taken prior to and after treatment with pelareorep. Results showed pelareorep treatment led to the activation of NK cells, which are known to directly kill cancer cells while stimulating adaptive anti-tumor immunity. The beneficial effects of pelareorep were observed both in patient samples and in vitro and were mediated by type 1 interferon (IFN-1) signaling, a key pathway involved in immunoregulation and tumor cell recognition.

"These important results further demonstrate both pelareorep’s innate and adaptive immunologic mechanism of action, and we are pleased to have them published in such a prestigious, peer-reviewed journal," said Dr. Matt Coffey, President and Chief Executive Officer of Oncolytics Biotech and co-author of the paper. "When viewing these findings, alongside prior clinical data demonstrating pelareorep’s ability to activate T cells while promoting their infiltration into tumors, we see pelareorep delivering a robust, coordinated anti-cancer immune response driven by both the innate and adaptive immune systems. Looking forward, we will continue to leverage the immunotherapeutic effects of pelareorep as we further its development as an enabling technology for a wide cross-section of immunotherapeutic agents in breast cancer and other oncologic indications with high unmet needs."

Patient samples evaluated in the publication were from a completed window-of-opportunity clinical study evaluating pelareorep in colorectal cancer patients with liver metastases. Patients received between one and five doses of pelareorep prior to planned surgery to resect metastases. Blood samples were taken both prior to and at multiple time points after pelareorep treatment. Additional details of the trial, as well as previously reported results, are available in prior peer-reviewed publications1,2.

References:
1.Adair RA, Roulstone V, Scott KJ, et al. Sci Transl Med. 2012;4(138):138ra77. doi:10.1126/scitranslmed.3003578

2.El-Sherbiny YM, Holmes TD, Wetherill LF, et al. Clin Exp Immunol. 2015;180(1):98-107. doi:10.1111/cei.12562

On March 17, 2022 Nkarta, Inc. (Nasdaq: NKTX), a clinical-stage biopharmaceutical company developing engineered natural killer (NK) cell therapies to treat cancer, reported financial results for the fourth quarter and year ended December 31, 2021 (Press release, Nkarta, MAR 17, 2022, View Source [SID1234610267]).

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"2021 was a year of solid execution for Nkarta across our two clinical development programs, unique NK cell engineering platform and efficient manufacturing processes," said Paul J. Hastings, President and CEO of Nkarta. "2022 is set to be a catalyst rich year with clinical data milestones that include initial results from our single-agent NKX101 Phase 1 clinical trial in the first half of 2022 as well as initial results from our single-agent NKX019 Phase 1 clinical trial in 2022. Nkarta continues to make excellent progress in advancing our pipeline of groundbreaking therapies for cancer patients with limited treatment options."

Anticipated Clinical Milestones

NKX101 – As previously announced, Nkarta plans to present initial clinical data from its ongoing clinical trial of NKX101 as monotherapy in patients with relapsed/refractory AML and higher-risk MDS in the first half of 2022.

NKX019 – As previously announced, Nkarta plans to present initial clinical data from its ongoing clinical trial of NKX019 as monotherapy in patients with advanced B cell malignancies in 2022.
2021 and Recent Operational Highlights
NKX101

In February 2022, Nkarta filed a protocol amendment with the U.S. Food and Drug Administration (FDA) for the ongoing Phase 1 clinical trial of NKX101 to optimize the study design for maximum benefit and flexibility as the company prepares for potential dose expansion cohorts. The amended protocol allows for a higher dose of cyclophosphamide for lymphodepletion, enrollment of patients who have received as few as 1 to 2 prior lines of therapy, and increased dosing of NKX101.

In December 2021, NKX101 received orphan drug designation (ODD) for the treatment of acute AML from the FDA. The FDA grants ODD to drugs defined as those intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. ODD may qualify the company developing the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions, and seven-year marketing exclusivity upon FDA approval.
NKX019

In January 2022, Nkarta filed a protocol amendment with the FDA for the ongoing Phase 1 clinical trial of NKX019 to optimize the study design for maximum benefit and flexibility as the company prepares for potential dose expansion cohorts. The amended protocol allows for administration of a consolidation cycle of NKX019 to patients following a complete response to NKX019, and increased dosing of NKX019.

In October 2021, Nkarta announced the dosing of the first patients in the international Phase 1 clinical trial evaluating NKX019 in CD19+ advanced B cell malignancies.
Pipeline and Platform

In November 2021, Nkarta presented preclinical data from its engineered NK cell platform in four posters at the annual meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper). The posters included data on CRISPR/Cas9 genome engineering of NK cells (jointly presented with CRISPR Therapeutics), engineered NK cells containing CISH gene knock-out and CD70 chimeric antigen receptor (CAR) targeting (jointly presented with CRISPR Therapeutics), donor selection in next generation NK cell development programs and novel methods for scaling the expansion of engineered NK cells to potentially supply a life cycle’s worth of commercial off-the-shelf product from a single donor.

In May 2021, Nkarta and CRISPR Therapeutics announced a research and development collaboration to co-develop and co-commercialize two genome engineered NK cell product candidates, one targeting CD70, and a product candidate combining NK cells and T cells (NK+T). The collaboration also gives Nkarta a license to CRISPR/Cas9 gene editing technology for use in its own engineered NK cell therapy products.
Manufacturing

In October 2021, Nkarta announced that it is producing clinical supply of NKX019 at its in-house cGMP clinical manufacturing facility in South San Francisco, California.

In July 2021, Nkarta entered a lease agreement to establish a new 88,000 square foot combined manufacturing facility and company headquarters. Once operational, the manufacturing facility will increase Nkarta’s manufacturing footprint with capacity to produce materials for potential pivotal trials and commercial launch of Nkarta’s engineered NK cell therapy products.
Fourth Quarter and Full Year 2021 Financial Highlights

Cash and Cash Equivalents: As of December 31, 2021, Nkarta had cash, cash equivalents, restricted cash and short-term investments of $240.2 million.

R&D Expenses: Research and development (R&D) expenses were $63.4 million for the full year 2021 and $17.3 million for the fourth quarter of 2021. Non-cash stock-based compensation expense included in R&D expense was $6.7 million for the full year 2021 and $1.7 million for the fourth quarter of 2021.

G&A Expenses: General and administrative (G&A) expenses were $23.0 million for the full year 2021 and $5.6 million for the fourth quarter of 2021. Non-cash stock-based compensation expense included in G&A expense was $7.7 million for the full year 2021 and $2.0 million for the fourth quarter of 2021.

Net Loss. Net loss was $86.1 million, or $2.62 per basic and diluted share, for the full year 2021. This net loss includes non-cash charges of $16.5 million that consisted primarily of share-based compensation of $14.5 million. Net loss was $22.8 million, or $0.69 per basic and diluted share, for the fourth quarter of 2021.
Financial Guidance

Nkarta expects its current cash and cash equivalents will be sufficient to fund its current operating plan into at least the second half of 2023.
About NKX101
NKX101 is an allogeneic and off-the-shelf natural killer (NK) cell immunotherapy candidate that builds on the innate anti-cancer biology of NK cells and their positive safety profile. Using NK cells selected from healthy donors, NKX101 is engineered to express a chimeric antigen receptor (CAR) targeting NKG2D ligands on tumor cells and a proprietary membrane-bound form of interleukin 15 (IL-15) to augment the activity of the NK cells. NKG2D, a key activating receptor found on naturally occurring NK cells, induces a cell-killing immune response through the detection of stress ligands that are widely expressed on cancer cells. To learn more about the NKX101 clinical trial in adults with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS), please visit ClinicalTrials.gov, identifier NCT04623944.

About the NKX101-101 Clinical Trial
The NKX101-101 clinical trial is a Phase 1, multi-center, open-label, sequential dose-finding and dose-expansion study to evaluate the safety and anti-tumor activity of NKX101 as a multi-dose, multi-cycle therapy. Patients with relapsed or refractory acute myeloid leukemia (AML) or higher-risk myelodysplastic syndromes (MDS) will be enrolled in the dose-finding portion of the study followed by disease specific expansion cohorts including a combination cohort. Additional information is available on ClinicalTrials.gov, identifier NCT04623944.

About NKX019
NKX019 is an allogeneic and off-the-shelf natural killer (NK) cell immunotherapy candidate that builds on the innate anti-cancer biology of NK cells and their positive safety profile. Using NK cells selected from healthy donors, NKX019 is engineered to express a chimeric antigen receptor (CAR) targeting the B-cell antigen CD19 and a proprietary membrane-bound form of interleukin 15 (IL-15) to augment the activity of the NK cells. CD19 is a biomarker for normal and malignant B cells, and it is a validated target for B cell cancer therapies. To learn more about the clinical trial of NKX019 in advanced B cell malignancies, please visit ClinicalTrials.gov, identifier NCT05020678.

About the NKX019-101 Clinical Trial
The NKX019-101 clinical trial is a Phase 1, multi-center, open-label, sequential dose-finding and dose-expansion study to evaluate the safety and anti-tumor activity of NKX019 as a multi-dose, multi-cycle therapy. Patients with CAR T naïve relapsed/refractory non-Hodgkin lymphoma (NHL), chronic lymphocytic leukemia (CLL) or B-cell acute lymphoblastic leukemia (B-ALL) will be enrolled in the dose-finding portion of the study, followed by disease specific expansion cohorts that also include CAR T exposed patients. To learn more about the clinical trial of NKX019 in advanced B cell malignancies, please visit ClinicalTrials.gov, identifier NCT05020678.