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Geron Corporation Reports Fourth Quarter and Full Year 2019 Financial Results and 2020 Milestones

On March 12, 2020 Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company developing a first-in-class telomerase inhibitor, imetelstat, to treat hematologic myeloid malignancies, reported financial results for the fourth quarter and year ended December 31, 2019 as well as 2020 milestones (Press release, Geron, MAR 12, 2020, View Source [SID1234555492]). The Company ended fiscal year 2019 with $159.2 million in cash and marketable securities.

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"2019 was a pivotal year for Geron as we completed the imetelstat program transition, assembled an impressive in-house team with a proven track record in drug development, and advanced imetelstat into late-stage development with the opening of our IMerge Phase 3 clinical trial in lower risk myelodysplastic syndromes," said John A. Scarlett, M.D., Chairman and Chief Executive Officer. "In 2020, we plan to complete enrollment in IMerge, announce our decision regarding any potential late-stage development plans for myelofibrosis by mid-year, and commence a proof of concept study in additional hematologic myeloid malignancies. With a strong team in place to execute these plans, we look forward to further advancing the development of imetelstat."

Planned 2020 Milestones

Geron is planning for the following milestones in 2020:

Complete enrollment for the Phase 3 IMerge clinical trial in lower risk myelodysplastic syndromes (MDS) by the end of 2020

Recently reported Phase 2 data continued to indicate meaningful and durable transfusion independence potentially achievable with imetelstat treatment in high transfusion burdened patients (> 4 units per 8 weeks). The Phase 3 IMerge clinical trial was opened for enrollment in August 2019, and the first patient was dosed in October 2019. As of the end of February 2020, 63% of planned clinical sites were opened for enrollment. Topline results are expected by mid-year 2022.

Determine a potential registration strategy for imetelstat in myelofibrosis (MF)

As a follow up to an End of Phase 2 meeting with the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2019, Geron plans to submit Phase 3 trial design proposals to the FDA and, in the second quarter, discuss with the FDA a potential regulatory approval path for imetelstat in MF. Geron expects to announce its decision regarding any potential late-stage development plans for MF by mid-year 2020.

Expect to present updated data and new analyses from the Phase 2 IMerge and IMbark clinical trials at future medical conferences

Geron expects to present more mature data from the Phase 2 IMerge clinical trial in lower risk MDS for the continued treatment and follow-up of remaining patients, including durability of transfusion independence.

Geron also expects to present new analyses from the IMbark Phase 2 clinical trial that correlate the median overall survival observed with other clinical endpoints from the trial. In addition, the new analyses are expected to provide further support for the potential improvement in overall survival as an indication of disease-modifying activity of imetelstat treatment in myelofibrosis.

Commence a proof of concept study of imetelstat in additional hematologic myeloid malignancies

Geron plans to expand imetelstat’s clinical development program with a proof of concept study in Intermediate-2 or High-risk, or higher risk, MDS and acute myeloid leukemia (AML) and expects to commence such a study by the end of the fourth quarter 2020.

2019 Accomplishments

Clinical – Advanced MDS development and presented data supporting potential late-stage development in MDS and MF

○Presented updated data from the Phase 2 IMerge clinical trial at the European Hematology Association (EHA) (Free EHA Whitepaper) meeting in June 2019 that reported continued meaningful and durable transfusion independence.
○Commenced screening and enrollment for the Phase 3 IMerge clinical trial in August 2019 and dosed the first patient in October 2019.
○Presented Phase 2 IMbark data at EHA (Free EHA Whitepaper) corroborating potential survival benefit of imetelstat in relapsed/refractory MF patients when compared to closely matched patients from real-world data treated with best available therapy.

Regulatory – Initiated FDA interactions to determine potential for late-stage development in MF

○The FDA granted Fast Track designation to imetelstat for the treatment of adult patients with Intermediate-2 or High-risk relapsed/refractory MF in September 2019.
○Conducted an End of Phase 2 meeting with the FDA in the fourth quarter of 2019.

Operational – Completed transition of imetelstat development program and enhanced development capabilities

○Transitioned the imetelstat program back to Geron in the third quarter of 2019, including transfer of imetelstat investigational new drug (IND) sponsorship in May 2019.
○Throughout 2019, recruited hematology-oncology research and development expertise, including many team members with prior experience with imetelstat, as well as both early- and late-stage development experience, to establish a multi-functional development team to support current and future development plans.
○Re-established manufacturing supply chain to manufacture imetelstat.
Fourth Quarter and Full Year 2019 Results

For the fourth quarter of 2019, the Company reported a net loss of $29.1 million, or $0.15 per share, compared to $7.3 million, or $0.04 per share, for the fourth quarter of 2018. Net loss for the full year of 2019 was $68.5 million, or $0.36 per share, compared to $27.0 million, or $0.15 per share, for the full year of 2018.

Revenues for the three and twelve months ended December 31, 2019 were $171,000 and $460,000, respectively, compared to $375,000 and $1.1 million for the same periods in 2018. Revenues for the three and twelve months ended December 31, 2019 and 2018 included royalty and license fee revenues under various non-imetelstat license agreements. The decline in revenues reflects a reduction in the number of active research license agreements in 2019 related to the Company’s human telomerase reverse transcriptase, or hTERT, technology as a result of patent expirations on the underlying technology.

Total operating expenses for the three and twelve months ended December 31, 2019 were $30.2 million and $73.0 million, respectively, compared to $10.0 million and $32.1 million for the same periods in 2018. Research and development expenses for the three and twelve months ended December 31, 2019 were $24.9 million and $52.1 million, respectively, compared to $5.1 million and $13.4 million for the same periods in 2018. The increase in research and development expenses, compared to the same periods in 2018, primarily reflects costs for the transition of the imetelstat program, including resuming sponsorship of the ongoing imetelstat clinical trials; expenses for start-up activities for the IMerge Phase 3 clinical trial; purchase of inventories of drug product, drug substance and raw materials from Janssen; and higher personnel-related costs for the expanding development team. General and administrative expenses for the three and twelve months ended December 31, 2019 were $5.3 million and $20.9 million, respectively, compared to $4.9 million and $18.7 million for the same periods in 2018. The increase in general and administrative expenses, compared to the same periods in 2018, primarily reflects higher corporate and patent legal costs and increased personnel-related expenses for additional general and administrative headcount to support the development organization.

Interest and other income for the three and twelve months ended December 31, 2019 was $925,000 and $4.2 million, respectively, compared to $1.1 million and $3.3 million for the same periods in 2018. The overall increase in interest and other income in 2019 when compared to 2018 primarily reflects higher yields on the Company’s marketable securities portfolio.

The Company ended the 2019 fiscal year with $159.2 million in cash and marketable securities. The Company expects these funds to be sufficient to continue the IMerge clinical trial in 2020 and to commence a proof of concept study in 2020.

Projected 2020 Financial Guidance

For fiscal year 2020, the Company expects its operating expense burn to range from $70 to $75 million, which includes costs related to the global Phase 3 IMerge clinical trial in MDS; validation of supply chain vendors for the manufacturing of imetelstat; further interactions with the FDA in connection with the planned submission of Phase 3 trial design proposals in MF and discussion regarding a potential regulatory approval path in MF; and commencement of a proof of concept study of imetelstat.

As of December 31, 2019, the Company had 46 employees. The Company plans to grow to a total of approximately 55 to 60 employees by year-end 2020, of which the majority will be research and development personnel.

Conference Call

Geron will host a conference call to discuss fourth quarter and full year 2019 financial results and 2020 milestones at 4:30 p.m. ET on Thursday, March 12, 2020.

Participants may access the conference call live via telephone by dialing domestically +1 (866) 393-4306 or internationally +1 (734) 385-2616. The conference ID is 5528886. A live, listen-only webcast will also be available on the Company’s website at www.geron.com/investors/events. If you are unable to listen to the live call, an archived webcast will be available on the Company’s website for 30 days.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Early clinical data suggest imetelstat may have disease-modifying activity through the suppression of malignant progenitor cell clone proliferation, which allows potential recovery of normal hematopoiesis. Clinical studies of imetelstat sponsored by Geron include IMerge, a Phase 2/3 trial in lower risk myelodysplastic syndromes (MDS), and IMbark, a Phase 2 trial in Intermediate-2 or High-risk myelofibrosis (MF). Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis-stimulating agent and for patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus kinase (JAK) inhibitor treatment.

Bellicum Reports Fourth Quarter and Full Year 2019 Financial Results and Provides Operational Update

On March 12, 2020 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported financial results for the fourth quarter and full year 2019 and provided an operational update (Press release, Bellicum Pharmaceuticals, MAR 12, 2020, View Source [SID1234555491]).

"Bellicum is at an exciting inflection point as we continue to validate the GoCAR platform and explore its utility more broadly," said Rick Fair, President and Chief Executive Officer of Bellicum. "We recently presented promising new translational data from the BPX-601 Phase 1 study, and we intend to present an update later this year on safety and preliminary activity in pancreatic cancer using repeated BPX-601 GoCAR-T activation with rimiducid. We have also expanded utilization of our GoCAR platform by initiating our first off-the-shelf GoCAR-NK program. In 2020, we look forward to the progress we expect to make applying our platform to solid tumor and off-the-shelf cell therapies."

PROGRAM HIGHLIGHTS AND CURRENT UPDATES
BPX-601 GoCAR-T

Bellicum presented new Phase 1 translational data for BPX-601 at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO GI) in San Francisco in January 2020. Primary observations included tumor infiltration, GoCAR-T mediated immunomodulation, survival and persistence of cells for up to nine months, and changes in the tumor microenvironment gene expression consistent with a productive CAR-T cell immune response.

Bellicum is currently enrolling cohort 5C of this trial to collect data to evaluate the safety of repeat rimiducid dosing to re-activate GoCAR-T cells over time, the first-in-human experience using the GoCAR platform as intended. Initial results from Cohort 5C are expected to be presented at a medical meeting by the end of 2020.

BPX-603 GoCAR-T

In response to Bellicum’s IND application for BPX-603, the FDA requested additional nonclinical data to further characterize this product candidate. Non-clinical experiments to generate the data are underway. Management expects to provide an update on its progress for this program in the third quarter of 2020.

BCMA GoCAR-NK Program

Bellicum recently initiated formal preclinical development activities for its GoCAR-NK program targeting B-cell maturation antigen, or BCMA, for the treatment of multiple myeloma. Bellicum presented a poster at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in November 2019 that included preclinical data that suggest the GoCAR platform may enhance NK cell proliferation, persistence, and cytotoxicity, potentially improving their utility as an off-the-shelf cancer therapy. Specifically, Bellicum believes that GoCAR-NK may improve the durability of clinical responses while offering the anticipated advantages that an allogeneic, off-the-shelf product may provide, including faster and more certain time to treatment, greater scalability and convenience, and potentially lower cost.

Management expects to present additional preclinical data for this program by the end of 2020.

Corporate Highlights

In January 2020, Bellicum entered into an asset purchase agreement under which The University of Texas MD Anderson Cancer Center will acquire Bellicum’s approximately 60,000-square-foot Houston facility, including manufacturing, office and laboratory space, for $15.0 million. As part of the transaction, Bellicum will also enter into a master services agreement with MD Anderson. Following completion of the transaction, MD Anderson will operate the Houston facility for its own internal programs as well as to manufacture Bellicum’s GoCAR and other cellular therapy programs for clinical trials and potentially early commercial supply.

In December 2019, Bellicum licensed its CaspaCIDe safety switch to MD Anderson for use in its CD19 CAR-NK program. Under terms of the original license agreement, MD Anderson exercised its option to non-exclusively license the technology for this construct and subsequently sublicensed it to a third party for future development. These actions entitled Bellicum to receive an upfront payment of $5 million and undisclosed future milestone payments and royalties on sales.

Fourth Quarter and Full Year 2019 Financial Results and Outlook
Revenue: Bellicum reported revenue of $5.1 million and $7.1 million for the fourth quarter and year ended December 31, 2019, respectively compared to $0.3 million and $1.1 million during the comparable periods in 2018. The increase in revenues in the fourth quarter and full year 2019 compared to the respective periods in 2018 were primarily due to a $5.0 million license fee received from MD Anderson for the non-exclusive license to the CaspaCIDe safety switch.
R&D Expenses: Research and development expenses were $13.3 million and $64.5 million for the fourth quarter and year ended December 31, 2019, respectively, compared to $19.9 million and $71.6 million during the comparable periods in 2018. The reduction in expenses in the fourth quarter and full year 2019 compared to respective periods in 2018 were primarily due to reduced expenses related to rivo-cel, reductions in general R&D expenses, and reduced employee salary-related charges from the reduction in force that was implemented during the second half of 2019, partially offset by the impairment of the intangible asset previously recorded from the Miltenyi Supply Agreement, increased expenses related to our GoCAR-T program, and employee severance costs arising from the reduction in force.
G&A Expenses: General and administrative expenses were $5.7 million and $30.0 million for the fourth quarter and year ended December 31, 2019, respectively, compared to $7.0 million and $25.0 million during the comparable periods in 2018. The reduction in expenses in the fourth quarter of 2019 relative to the comparable period in 2018 was primarily due to a decrease in personnel costs and share-based compensation from the reduction in force that was implemented during the second half of 2019. The increase in G&A expenses for the year ended December 31, 2019, compared to the year ended December 31, 2018, was primarily due to an increase in personnel costs and commercialization activities during the first half of 2019, partially offset by a reduction in rivo-cel related commercialization activities as well as the effects of the reduction in force that reduced employee salary-related charges.

Loss from Operations: Bellicum reported a loss from operations of $13.9 million and $87.4 million for the fourth quarter and year ended December 31, 2019, respectively, compared to a loss from operations of $26.6 million and $95.5 million for the comparable periods in 2018.
Cash used in operating activities was $12.7 million and $77.6 million for the fourth quarter and year ended December 31, 2019, respectively, compared to cash used in operating activities of $20.4 million and $74.8 million for the comparable periods in 2018.
Net Loss: Bellicum reported a net loss of $29.0 million and $112.5 million for the fourth quarter and year ended December 31, 2019, respectively, compared to a net loss of $27.2 million and $98.0 million for the comparable periods in 2018. The results included non-cash expense of $14.3 million and $19.2 million related to the change in fair value of warrant liability in the fourth quarter and year ended December 31, 2019, respectively.
Shares Outstanding: In February, Bellicum effected a reverse stock split of its issued and outstanding common stock, at a ratio of 1-for-10. As of February 28, 2020, Bellicum had 5,047,892 shares of common stock outstanding and 534,200 shares of preferred stock outstanding. Each preferred share can be converted into 10 shares of common stock.
Cash Position and Guidance: Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements into the second half of 2021. Management expects cash utilization of $55 to $65 million in 2020. Bellicum reported cash and cash equivalents, restricted cash and investments totaling $93.8 million as of December 31, 2019, compared to $106.9 million as of September 30, 2019.

Conference Call and Webcast
Bellicum’s management will host a webcast and conference call today at 5 p.m. ET / 2 p.m. PT, March 12, 2020, to discuss the financial results for the fourth quarter 2019 and provide a corporate update. The live call may be accessed by dialing (877) 407-3103 for domestic callers and (201) 493-6791 for international callers. A live webcast of the call will be available from the Investors and Media section of the company’s website at www.bellicum.com and a replay will be available shortly after the live event.

Synlogic Reports Fourth Quarter and Full Year 2019 Financial Results and Provides Business Update

On March 12, 2020 Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company applying synthetic biology to beneficial microbes to develop novel, living medicines, reported its financial results for the fourth quarter and full year ended December 31, 2019 (Press release, Synlogic, MAR 12, 2020, View Source [SID1234555490]).

"We have built key Synthetic Biotic platform capabilities in synthetic biology, manufacturing and development to enable the efficient generation of therapeutics that have the potential to address unmet medical need in a range of indications from rare metabolic diseases to cancer," said Aoife Brennan, M.B., Ch.B., Synlogic’s president and chief executive officer. "Building on our experience in our PKU program we have made steady progress on new programs in enteric hyperoxaluria and maple syrup urine disease and we look forward to providing more detail on these initiatives as well as the underlying engine that powers our pipeline as year progresses."

2020 Priorities

Pipeline

Initiation of a Phase 2 clinical trial to evaluate a solid formulation of SYNB1618 in patients with phenylketonuria (PKU) expected in the first half of 2020. The trial is designed to evaluate safety and tolerability of a solid formulation of SYNB1618 as well as its potential to lower blood phenylalanine (Phe) levels in PKU patients. In addition, the study is expected to provide valuable information to validate predictive pharmacodynamic and preclinical modeling.
Evaluation of data expected in 2020 from the monotherapy arm of the Phase 1 clinical study of SYNB1891 in patients with advanced solid tumors or lymphoma. SYNB1891 is an intra-tumorally administered Synthetic Biotic medicine engineered to produce cyclic di-AMP, an agonist of the STING pathway, that is designed to serve as a dual innate activator of the immune system as a potential treatment for solid tumors or lymphoma. SYNB1891 is being evaluated as a monotherapy in an ongoing Phase 1 open-label, multicenter, dose escalation clinical trial (NCT04167137) in patients with advanced solid tumors or lymphoma. Synlogic expects to have data from the monotherapy arm of this study in 2020. After establishing a maximum tolerated dose for SYNB1891 as monotherapy, Synlogic expects to 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.
Continued development of patient and commercialization-appropriate presentations of SYNB1618. Synlogic has developed and manufactured a solid formulation of its Synthetic Biotic SYNB1618 suitable for future clinical trials and continues to evaluate and develop presentations such as enteric-coated capsules and pressed tablets for eventual commercialization.
Advancement of new Synthetic Biotic programs in metabolic diseases with high unmet medical need. Synlogic is conducting preclinical studies of Synthetic Biotic medicines to treat enteric hyperoxaluria (HOX), an acquired metabolic disorder in which patients develop recurrent kidney stones due to elevated urinary oxalate levels and are at an increased risk of kidney failure. In addition, Synlogic is also developing Synthetic Biotic medicines for the treatment of maple syrup urine disease (MSUD), a rare inherited metabolic disease caused by defective enzymes that metabolize branched chain amino acids (BCAAs) which are components of protein. Elevated blood levels of BCAAs can lead to can lead to seizures, coma, and death. There are currently no approved therapies to treat these disorders.
Presentation and publication of data at major scientific and medical meetings. Synlogic is committed to publishing and presenting data that demonstrate the breadth of Synlogic’s Synthetic Biotic platform.
Corporate

Continued strengthening of Synlogic’s leadership. In January 2020, Synlogic announced the appointment of Michael Burgess, M.B., Ch.B., Ph.D., President, Research & Development, at Turnstone Biologics, to its board of directors. Dr. Burgess is a physician scientist who brings extensive experience in translational drug development from leadership roles at several large Pharma companies including Roche, Bristol-Myers Squibb and Lilly.
Continued exploration of additional strategic collaborations. Synlogic expects to continue to develop strategic collaborations to expand the breadth of its Synthetic Biotic pipeline in therapeutic areas that have high biology risk.
Fourth Quarter 2019 Financial Results

As of December 31, 2019, Synlogic had cash, cash equivalents, and short- and long-term investments of $127.1 million.

For the three months ended December 31, 2019, Synlogic reported a consolidated net loss of $12.8 million, or $0.37 per share, compared to a net loss of $11.9 million, or $0.47 per share, for the corresponding period in 2018.

Research and development expenses were $11.3 million for the three months ended December 31, 2019 compared to $8.9 million for the corresponding period in 2018. The increase in expenses was primarily due to use of synthetic biology services provided under Synlogic’s collaboration with Ginkgo and increased clinical activities, including the SYNB1618 bridging study and initiation of the SYNB1891 Phase 1 clinical study.

General and administrative expenses for the three months ended December 31, 2019 were $3.5 million compared to $4.0 million for the corresponding period in 2018.

Revenue was $1.2 million for the three months ended December 31, 2019 compared to $0.1 million for the three months ended December 31, 2018. Revenue is associated with services performed under the Synlogic’s collaboration with AbbVie to develop a Synthetic Biotic medicine for the treatment of inflammatory bowel disease (IBD). The increase in revenue for the fourth quarter of 2019 compared to the same period in 2018 was a result of revised estimates of time and effort required to reach certain milestones in the collaboration.

Full Year 2019 Financial Results

For the year ended December 31, 2019, consolidated net loss was $51.4 million, or $1.70 per share, compared to a consolidated net loss of $48.4 million, or $2.03 per share, for the year ended December 31, 2018. Revenues were $2.2 million for the year ended December 31, 2019, compared to $2.5 million for the same period in 2018. Total operating expenses were $56.6 million for the year ended December 31, 2019, compared to $53.8 million for the same period in 2018.

Conference Call & Webcast Information

Synlogic will host a conference call and live webcast today at 5:00 p.m. ET today, Thursday, March 12, 2020. To access the live webcast, please visit the "Event Calendar" page within the Investors and Media section of the Synlogic website. Alternatively, 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 4089293. For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors and Media section of the Synlogic website.

Regulus Reports Fourth Quarter and Year-End 2019 Financial Results and Recent Updates

On March 12, 2020 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported financial results for the fourth quarter and year ended December 31, 2019 and provided a corporate update (Press release, Regulus, MAR 12, 2020, View Source [SID1234555489]).

"We have made significant progress over the past few months, including receiving notification from FDA of their decision to lift the partial clinical hold on our Phase 1 multiple ascending dose clinical study for RGLS4326, enabling the initiation of dosing of the second cohort of that study for the treatment of autosomal dominant polycystic kidney disease this past February," said Jay Hagan, CEO of Regulus. "This important milestone, coupled with the closing of the second tranche of financing, enables us to advance the program toward key data read-outs."

Corporate Highlights

Closed $26 Million Second Tranche of Private Financing: In December 2019, following the announcement of the Company’s plan to recommence the Phase 1 Multiple Ascending Dose ("MAD") clinical study of RGLS4326 for the treatment of autosomal dominant polycystic kidney disease ("ADPKD") in the first quarter of 2020, the Company completed a second and final closing under the May 2019 securities purchase agreement, pursuant to which the Company sold and issued 3,288,390 shares of non-voting Class A-2 convertible preferred stock, in lieu of shares of common stock, at a price of $6.66 per share, and accompanying warrants to purchase an aggregate of 32,883,900 shares of common stock at a price of $0.125 for each share of common stock underlying such warrants. Each share of the non-voting Class A-2 convertible preferred stock is convertible into 10 shares of common stock, subject to certain beneficial ownership conversion limitations. The warrants are exercisable for a period of five years following the date of issuance and have an exercise price of $0.666 per share, pursuant to proportional adjustments in the event of stock splits or combinations or similar events. Together with the first tranche, which closed in May 2019, the Company raised a total of $42.7 million from the private financing, which the Company expects will provide cash resources to fund planned activities into mid-2021.

Program Highlights

Initiated Dosing of the Second Cohort in RGLS4326 Phase 1 for ADPKD: In February 2020, the Company initiated dosing of the second cohort of the MAD clinical study of RGLS4326, a novel oligonucleotide designed to inhibit miR-17 for the treatment of ADPKD. The Company expects to complete this study in mid-2020 with topline results available thereafter.

The Company is also planning to initiate a Phase 1b short-term dosing study in patients with ADPKD in the second half of 2020 to evaluate RGLS4326 for safety, pharmacokinetics, and biomarkers of pharmacodynamic activity. The re-initiation of the MAD study was allowed following a satisfactory complete response provided to FDA in November 2019 to address the partial clinical hold placed on the clinical program in July 2019. A partial clinical hold on studies of RGLS4326 dosed for extended durations remains in effect until the second set of requirements outlined by FDA have been satisfactorily addressed. Information from the Phase 1 clinical studies, together with information from additional nonclinical studies, will be used to address the second set of requirements to support studies of extended duration.

Financial Results

Cash Position: As of December 31, 2019, Regulus had $34.1 million in cash and cash equivalents.

Research and Development (R&D) Expenses: R&D expenses were $2.1 million and $12.3 million for the quarter and year ended December 31, 2019, respectively, compared to $5.3 million and $34.0 million for the same periods in 2018. The decreases were largely driven by decreases in external development expenses, primarily attributable to the pause of the RGLS4326 MAD study in the third quarter of 2018 and transfer of the RG-012 program to Sanofi beginning in the fourth quarter of 2018. Additionally, the decreases were driven by reductions in personnel and internal expenses, primarily attributable to a reduction in costs subsequent to our corporate restructuring in the third quarter of 2018.

General and Administrative (G&A) Expenses: G&A expenses were $2.4 million and $11.3 million for the quarter and year ended December 31, 2019, respectively, compared to $2.7 million and $12.9 million for the same periods in 2018. The decreases were mostly driven by a reduction in costs subsequent to our corporate restructuring in the third quarter of 2018.

Revenue: Revenue was less than $0.1 million and $6.8 million for the quarter and year ended December 31, 2019, respectively, compared to less than $0.1 million and $0.1 million for the same periods in 2018. The increase for the year ended December 31, 2019 was attributable to revenue recognition of the upfront payments received under the 2018 Sanofi Amendment related to the transfer of the RG-012 program to Sanofi.

Net Loss: Net loss was $4.9 million and $18.6 million for the quarter and year ended December 31, 2019, respectively, compared to a net loss of $8.6 million and $48.7 million for the same periods in 2018. Basic and diluted net loss per share was $0.23 and $1.08 for the quarter and year ended December 31, 2019, respectively, compared to $0.98 and $5.59 for the same periods in 2018.

About ADPKD

ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

About RGLS4326

RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and designed to preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of PKD1 and PKD2 in human ADPKD cyst cells, a reduction in kidney cyst formation, improved kidney weight/body weight ratio, decreased cyst cell proliferation, and preserved kidney function in mouse models of ADPKD. The RGLS4326 IND is currently on a Partial Clinical Hold for treatment of extended duration by the U.S. Food and Drug Administration.

CohBar Reports Fourth Quarter 2019 Financial Results and Provides Business Update

On March 12, 2020 CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company developing mitochondria based therapeutics (MBTs) to treat chronic diseases and extend healthy lifespan, reported its financial results for the fourth quarter ended December 31, 2019 (Press release, CohBar, MAR 12, 2020, View Source [SID1234555488]).

"We made substantial progress in this past quarter," said Steven Engle, CohBar’s Chief Executive Officer. "We completed the Phase 1a stage of our ongoing Phase 1a/1b clinical trial of CB4211, initiated the Phase 1b stage, and announced promising preclinical data in two new programs, a novel family of CXCR4 antagonists for cancer and other indications, and anti-fibrotic peptides for the potential treatment of fibrotic diseases such as idiopathic pulmonary fibrosis. We now have five mitochondrial derived peptide programs, up from three last summer. These new data provide additional evidence supporting the breadth of biological effects and therapeutic potential of mitochondrial peptides and strengthen our belief that our platform will continue to identify even more mitochondria based therapeutics going forward."

Fourth Quarter 2019 and R&D and Business Highlights

●CB4211 Clinical Study: In November, the company announced the completion of the Phase 1a stage of the company’s ongoing Phase 1a/1b study evaluating the safety, tolerability, and activity of CB4211, and the initiation of recruitment for the Phase 1b stage. The Phase 1b stage is designed to assess the safety, tolerability, and activity of CB4211 in obese subjects with non-alcoholic fatty liver disease (NAFLD). The double-blind, placebo-controlled evaluation will be conducted over four weeks in twenty patients randomized 1:1 to receive either CB4211 or placebo. Assessments will include changes in liver fat as determined by MRI-PDFF, body weight, and biomarkers relevant to NASH and obesity. CohBar has previously presented evidence that the novel mechanism of action of CB4211 enhances insulin effects on fat cells (adipocytes) leading to reduction of liver fat in preclinical models. Clinical data from both stages of the study are expected in the third quarter of 2020.

●New CXCR4 Inhibitor Program for Cancer and Other Indications: Recently, the company announced its discovery of MBT5 analogs, a novel family of potent and selective peptide inhibitors of the chemokine receptor, CXCR4. Analogs of MBT5 have demonstrated high potency at nanomolar levels in in vitro studies of CXCR4 inhibition in cultured cells. In a difficult to treat in vivo animal model of melanoma, the B16F10 syngeneic tumor model, an analog of MBT5 showed enhanced antitumor activity in combination with the chemotherapeutic agent temozolomide. CXCR4 is overexpressed in more than 75% of cancers and high levels of the receptor are associated with poor survival prognosis.

●MBT2 Analogs for Fibrotic Diseases: In December, CohBar announced the further demonstration of the therapeutic potential of a novel peptide in a preclinical model of idiopathic pulmonary fibrosis (IPF). In a therapeutic animal model of IPF, CohBar’s peptide analog, MBT2, demonstrated reductions in all parameters of the disease including lung fibrosis, inflammation, and collagen levels. These data further support earlier positive findings in a prophylactic model of IPF. IPF is a chronic, progressive, debilitating and usually fatal interstitial lung disease that affects approximately 100,000 people in the U.S.

CohBar is continuing to refine and evaluate these novel analogs in preclinical models of multiple indications. In addition, the company continues to develop its programs in cancer immunotherapy and type 2 diabetes.

●Investment Community Outreach: In October 2019, CohBar presented an overview of the company and its clinical development program at the BIO Investor Forum. In January 2020, CohBar met with investors, bankers, and analysts at the JP Morgan Healthcare Conference. In February 2020, CohBar presented at the BIO CEO and Investor conference.

●Expanded the Company’s Board of Directors: In October 2019, Misha Petkevich, Chief Investment Officer of V2M Capital, joined the CohBar board of directors. Mr. Petkevich’s appointment adds extensive financial and investment expertise. His past experience includes roles at V2M Capital, Robertson Stephens & Co. and Hambrecht & Quist.

During the fourth quarter and subsequent period, Dr. Pinchas Cohen and Dr. Nir Barzilai continued to be recognized as international leaders in the study of mitochondrial science, aging and age-related diseases:

Dr. Cohen received the Irving S. Wright Award of Distinction from the American Federation for Aging Research in New York in November 2019. In addition, he delivered a keynote presentation on "Endocrine Gerontology: from IGFs to Mitochondrial Peptides" at the Annual Meeting of the Gerontological Society of America, in Austin, Texas. Dr. Cohen also published a paper entitled "The mitochondrial-derived peptide MOTS-c is a regulator of plasma metabolites and enhances insulin sensitivity" in the journal Physiological Reports.

Dr. Barzilai delivered keynotes at Harvard’s Boston Pepper Center OAIC Mini-Symposium on Geroscience in Boston in December 2019 and at The Science and Economy of Aging World Economic Forum in Davos, Switzerland in January 2020. In January 2020 he also participated in a panel on aging during the JP Morgan Healthcare Conference and at the Longevity Therapeutics conference. Dr. Barzilai authored two articles in the prestigious Nature Medicine Journal, titled "Undulating changes in human plasma proteome profiles across the lifespan" and "Chronic inflammation in the etiology of disease across the lifespan." He also authored two papers on the latest advances in aging research and drug discovery in the Journal of Aging, titled "Screening human embryos for polygenic traits has limited utility" and "Varying Effects of APOE Alleles on Extreme Longevity in European Ethnicities."

Fourth Quarter 2019 Financial Highlights

●Cash and Investments. CohBar had cash and cash equivalents of $12.6 million as of December 31, 2019, compared to $22.2 million as of December 31, 2018. The cash burn for the quarter ended December 31, 2019, was approximately $2.2 million.

●R&D Expenses. Research and development expenses were $1.9 million for the three months ended December 31, 2019, compared to $2.1 million in the prior year quarter. The decrease was primarily due to lower clinical and preclinical costs in the quarter, partially offset by an increase in costs associated with CohBar’s research programs focused on the continuing development of peptides.

●G&A Expenses. General and administrative expenses were $1.7 million for the three months ended December 31, 2019, compared to $2.0 million in the prior year quarter. The decrease in general and administrative expenses was primarily due to severance and non-cash stock-based compensation costs related to the termination of CohBar’s former CEO in the prior year period and a decrease in recruiting fees, partially offset by an increase in payroll related accruals, legal fees and D&O insurance premiums.

●Net Loss. For the three months ended December 31, 2019, net loss, which included $0.7 million of non-cash expenses, was $3.7 million, or $0.09 per basic and diluted share. For the three months ended December 31, 2018, net loss, which included $1.0 million of non-cash expenses, was $4.2 million, or $0.10 per basic and diluted share.

Fourth Quarter Investor Call and Slide Presentation:

Date: March 12, 2020
Time: 5:00 p.m. ET (2:00 p.m. PT)

Conference Audio

- Dial-in U.S. and Canada: (877) 451-6152
- Dial-in International: (201) 389-0879
- Conference ID No.: 13698823

Slide Presentation

-Go to www.webex.com, click on the ‘Join’ button and enter meeting number 925 797 732 and Password CWBR, or
-Go to www.cohbar.com and click on Q4 2019 Shareholder Presentation at top of homepage.

We kindly request that you please call into the conference audio and log into Webex approximately 10 minutes prior to the start time so that we can begin promptly.

An audio replay of the call will be available beginning at 8:00 p.m. Eastern Time on March 12, 2020, through 11:59 p.m. Eastern Time on April 2, 2020. To access the recording please dial (844) 512-2921 in the U.S. and Canada, or (412) 317-6671 internationally, and reference Conference ID# 13698823. The audio recording along with the slide presentation will also be available at www.cohbar.com during the same period.