On March 16, 2021 Pliant Therapeutics, Inc. (Nasdaq: PLRX) (the Company), a clinical stage biotechnology company focused on discovering and developing novel therapeutics for the treatment of fibrosis, reported fourth quarter and full year 2020 financial results (Press release, Pliant Therapeutics, MAR 16, 2021, View Source [SID1234576731]).

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"We entered 2021 in a strong position resulting from our team’s execution throughout the fourth quarter despite the challenges of the evolving backdrop of the COVID-19 pandemic," said Bernard Coulie, M.D., Ph.D., President and Chief Executive Officer of Pliant Therapeutics. "Our focus remains on continuing to actively drive our lead Phase 2a programs in IPF and PSC towards near term data readouts, to advance our earlier-stage oncology and muscular dystrophy portfolio towards the clinic while also leveraging our robust discovery engine to create a broad and differentiated product portfolio focused on delivering novel treatments to areas of unmet medical need."

Fourth Quarter and Recent Highlights

PLN-74809 Phase 2a positron emission tomography (PET) imaging trial resumed, with preliminary data expected in the first half 2021. With the reopening of the trial site in November, we were able to resume the Phase 2a PET trial. This open-label dose ranging trial will evaluate target engagement of PLN-74809 in IPF patients utilizing a PET tracer of the integrin αvβ6. We will assess receptor occupancy levels achieved by PLN-74809, a dual selective inhibitor of αvβ6/αvβ1, across multiple single-dose cohorts.

PLN-74809 Phase 2a INTEGRIS trials in idiopathic pulmonary fibrosis (IPF) and primary sclerosing cholangitis (PSC) gained momentum in the fourth quarter and are currently on track to complete enrollment by the end of 2021 and the first half of 2022, respectively. These 12-week randomized, dose-ranging, double-blind, placebo-controlled trials will evaluate safety, tolerability, and pharmacokinetics, as well as exploratory efficacy endpoints in patients with IPF and PSC.

IND open for development of a PET tracer of the protein integrin αvβ1. Following a December 2020 Investigational New Drug (IND) filing and the recently issued a "Safe to Proceed Letter", Pliant expects to rapidly advance into clinical trials a study of a wholly owned αvβ1 PET tracer to evaluate expression levels of αvβ1 in various fibrotic tissues. This marks the Company’s fifth IND.
Successful completion of PLN-1474 Phase 1 trial and transfer of PLN-1474 to Novartis. The Phase 1 trial of PLN-1474 was a safety, tolerability, and pharmacokinetics dose-escalating first-in-human trial that enrolled 84 healthy volunteers. PLN-1474 was rapidly absorbed and well tolerated with no dose-or treatment-limiting toxicities or severe/ serious adverse events observed. In preclinical studies, PLN-1474 was observed to selectively block the αvβ1 integrin-mediated activation of TGF-β, reducing liver fibrosis in animal models. Following the successful completion of this study, PLN-1474 has been transferred to Novartis.
Leadership Team

The Company appointed Gregory P. Cosgrove, M.D., FCCP as Vice President of Clinical Development to lead the execution of the IPF clinical development program. Dr. Cosgrove brings to Pliant over 20 years of pulmonary and critical care expertise in academic clinical research. Dr. Cosgrove most recently served as Associate Professor at the National Jewish Health in Denver, Colorado, the leading respiratory hospital in the United States and as the Chief Medical Officer of the Pulmonary Fibrosis Foundation, a leading pulmonary fibrosis focused nonprofit organization.
The Company appointed Dr. David Pyott to the Company’s Board of Directors. Dr. Pyott brings over 30 years of leadership and management experience with expertise in organizational scaling to the Company. Most recently, Dr. Pyott served as Chairman and Chief Executive Officer of Allergan Inc., a role he held for over 17 years.
COVID-19 Preparedness

The Company continues to develop and maintain policies and procedures to enable us to operate safely and productively during the COVID-19 pandemic. The Company has experienced delays in clinical trial operations which have impacted and may further impact the expected timing of data readouts. The Company is working closely with clinical sites to continue site initiation and operation activities in compliance with study protocols while observing government and institutional guidelines. The Company intends to provide more specific guidance regarding clinical trial progress and the timing of data readouts as the long-term impacts of the pandemic become better understood.

Fourth Quarter 2020 Financial Results

Research and development expenses were $17.9 million, as compared to $11.7 million for the prior-year quarter. The increase was due primarily to higher costs related to the advancement of several programs and ongoing Phase 1/2 clinical trials.
General and administrative expenses were $5.6 million, as compared to $3.1 million for the same period in 2019. The increase was due to higher personnel-related and professional services expenses.
Net loss of $19.0 million as compared to a net income of $42.2 million for the prior-year quarter due to a decrease in related party revenue.
As of December 31, 2020, the Company had cash, cash equivalents and short-term investments of $276.9 million. Pliant believes it has sufficient funds to meet its operating and capital requirements into 2023.

On March 16, 2021 Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology, reported its financial results for 2020 highlighting the increase in revenues to PLN 37.3 million, including PLN 15.4 million from partnering agreements and recapped on the most significant clinical and corporate milestones of the past year (Press release, Ryvu Therapeutics, MAR 16, 2021, View Source [SID1234576748]).

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"The year 2020 was very eventful for us. We announced an updated development strategy, made significant progress in clinical projects and, with the move to our new headquarters, we have completed the largest investment in Ryvu 13-year history. We are working at full speed to bring new treatments to oncology patients and 2021 is shaping up to be an important year for the company" – said Pawel Przewiezlikowski, CEO of Ryvu Therapeutics.

Przewiezlikowski also draws attention to the most important achievements of the Company in the passing year:

Strengthening our resources

"We raised over USD 36 million from the share issue in July 2020, which combined with the grant financing received, gives us financial resources required to fulfill all the goals set until 2023. We have also completed an investment into our fully-owned R&D Center for Innovative Drugs in Krakow. We moved to our new laboratories, finalizing the corporate division between Ryvu and Selvita, now in distinct locations. What’s more, additional research space allows us to scale up our discovery and development activities."

We are bringing oncology treatments closer to patients

"SEL24/MEN1703 has successfully completed Phase I Clinical Study in Acute Myeloid Leukemia (AML). A couple of months later, the first patient was dosed with SEL24/MEN1703 in Europe within the Expansion Cohort of Phase I/II Study also in AML.

RVU120 (formerly SEL120), our flagship, fully-owned first in class CDK8/CDK9 inhibitor, has progressed through its first Phase I study in AML and myeloid dysplastic syndrome. The Phase Ib study is on-going in six clinical sites in the U.S. In order to de-risk the study and provide a platform for future development, we decided to activate additional sites in Europe, including Poland. We have already received appropriate official approvals to start research in our country and one of the European countries.

We are also investigating the development potential of RVU120 in multiple solid tumors, which can potentially significantly increase the market potential for our molecule. We submitted appropriate applications to start clinical trials in 2021. In March this year, we signed a contract with Covance Inc. for the execution of Phase I studies for RVU120 in solid tumors.

In 2020, We have initiated and are developing a promising research collaboration with Galapagos NV in the area of inflammatory diseases.

Throughout the year, we actively presented our projects at several scientific and investor conferences.

We assured business continuity and safety of our employees during COVID-19 pandemic introducing the highest sanitary standards and preventive measures."

Major Achievements

February 2020: Ryvu signed a grant agreement for the development of targeted oncology therapies based on the synthetic lethality concept. This grant provides Ryvu with almost USD 8.3 million of non-dilutive financing to discover, develop and select a clinical candidate targeting cancers which had been considered in the past as largely undruggable using rational approaches. The total net value of the project amounts to USD 14 million, and the anticipated project duration is until December 2023.

March 2020:

On March 5, Menarini Group announced the successful completion of Phase I clinical study of SEL24/MEN1703 in Acute Myeloid Leukemia, which entitled Ryvu to receive a USD 1.96 million milestone payment. The full data from the study was presented as a poster "Results of the dose escalation part of DIAMOND trial (CLI24-001): First-in-human study of SEL24/MEN1703, a dual PIM/FLT3 kinase inhibitor, in patients with acute myeloid leukemia", during the Virtual 25th EHA (Free EHA Whitepaper) Congress taking place June 11-21.
On March 27, the U.S. Food and Drug Administration (FDA) granted an orphan drug designation (ODD) to Ryvu’s SEL120 for the treatment of patients with acute myeloid leukemia (AML). The FDA’s orphan drug designation allows the drug for the designated indication to be eligible for requesting a seven-year period of U.S. marketing exclusivity upon approval of the drug, as well as potential of other development assistance and financial incentives.
April 2020: Galapagos NV (Euronext &NASDAQ: GLPG) and Ryvu Therapeutics announced a collaboration focused on the discovery and development of novel small molecule drugs in inflammation. Ryvu will contribute its biology and chemistry platform, as well as related intellectual property, to the program. During the joint research collaboration, Ryvu is responsible for early drug discovery, and Galapagos will be responsible for the further development of this program.

June 2020:

On June 2, Ryvu obtained the occupancy permits for its newly built R&D Center for Innovative Drugs, meaning it has completed the construction of the facility. Subsequently, Ryvu has initiated its move to the new headquarters. The laboratory-office complex is made up of 6 floors with a total area of ca. 108,000 sq. ft. At the heart of the new Ryvu facility is an array of laboratories incl. medicinal chemistry, biochemistry, cell & molecular biology and analytical chemistry, capable of accommodating up to 300 employees. The newly built facilities are located at Sternbach St., named after Dr. Leo Sternbach, a graduate of Jagiellonian University who went on to discover Vallium, the most prescribed drug in the history of the pharmaceutical industry. New building offers Ryvu additional research space and comfort, which turned out to be a great advantage for Ryvu during COVID-19 spatial restrictions. Cost of the investment, including the purchase of a plot of land and laboratory equipment, incurred until February, 2021, amounted to approximately USD 21.2 million, of which ca. USD 6.7 million was covered by the grant financing.
On June 3, NodThera, Ryvu spin-off company, secured GBP 44.5 million (USD 54.5 million) Series B financing. NodThera was founded by Epidarex Capital and Ryvu in 2016, based on world class research on NLRP3 inflammasome conducted at Ryvu (at that time Selvita) in 2012-2016. The Company focused on the development of inflammasome inhibitors has already raised over GBP 80.8 million (over USD 100 million) in three funding series. After the full completion of Series B capital increase, Ryvu owns 4.8% share in NodThera.
On June 4, Ryvu signed a grant agreement for the development of targeted immuno-oncology therapy, which provides Ryvu with almost USD 5.6 million of non-dilutive financing to discover, develop and select a clinical candidate targeting cancers which had been considered in the past as largely undruggable using rational approaches.
On June 15, Ryvu announced its updated development strategy for 2020-2022, which assumes, i.a.: completing Phase I clinical development of SEL120 in AML/MDS by the end of 2022, expanding therapeutic potential for SEL120 in solid tumors and launching a new Phase I study in selected indications in parallel to the ongoing hemato-oncology studies, advancing at least one program into the Phase I of clinical trials from preclinical pipeline.
On June 22-24, Ryvu presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II the most recent data from its oncology projects, including results from the small-molecule STING agonists, dual A2A/A2B adenosine receptors antagonist program, HPK1 inhibitors and SMARCA2 (BRM) degraders program.

July 2020: Ryvu successfully closed its series I follow-on share offering, with 2,384,245 shares offered at a price of PLN 60 per share. The Company raised over USD 36 million (PLN 143 million) of new capital. Proceeds from the share issue will be allocated primarily to Company’s R&D programs.

September 2020: The first patient has been treated in Europe for the cohort expansion part of Phase II DIAMOND-01 clinical trial (CLI24-001; NCT03008187) investigating SEL24/MEN1703, a first-in-class, oral dual PIM/FLT3 inhibitor, as a single agent in Acute Myeloid Leukemia (AML).

October 2020: The Management Board of the Company made a strategic decision to revise its preclinical projects’ pipeline. As a consequence, the Company stopped the development of two projects: a dual adenosine receptor antagonist (A2A/A2B) and the project in the area of synthetic lethality (SMARCA2). The above decision was made after consultation with the Supervisory Board of the Company. Ryvu now intends to concentrate its resources on the SEL120 project, currently in Phase I clinical trials, as well as the remaining preclinical projects, and assign financing to newly initiated discovery and development projects in the area of synthetic lethality.

November 2020: Ryvu presented its poster regarding the STING agonists program – "Development of improved small molecule STING agonists suitable for systemic administration", at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 35th Anniversary Annual Meeting (SITC 2020).

December 2020: Announcement of the positive results of the pharmacodynamic assay demonstrating target engagement in the dose escalation part of the DIAMOND-01 trial (CLI24-001; clinicaltrials.gov identifier NCT03008187), a study investigating SEL24/MEN1703 – first-in-class, orally available, dual PIM/FLT3 inhibitor as single agent in acute myeloid leukemia (AML). The poster entitled "SEL24/MEN1703 provides PIM/FLT3 Downstream Pathway Inhibition in Acute Myeloid Leukemia (AML) Blast Cells: Results of the Pharmacodynamic (PD) Assay in the Dose Escalation Part of First-in-Human DIAMOND Trial" was presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, 2020.

Throughout 2020, Ryvu Therapeutics participated and presented at several scientific and investor conferences, including:

AACR 2020 Virtual Annual Meeting,
Virtual 25th Annual European Hematology Association (EHA) (Free EHA Whitepaper) Congress,
Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 35th Anniversary Annual Meeting (SITC 2020),
Jefferies 2020 Virtual Healthcare Conference,
BIO Digital 2020,
Solebury Trout Investor Access during JP Morgan 2020,
Solebury Trout Virtual Investor Conference,
BIO-Europe Spring 2020,
32nd Annual ROTH Conference,
H.C. Wainwright & Co. 22nd Annual Global Investment Conference,
Erste Group Innovation Conference 2020,
Trigon Investor Week,
The Finest CEElection Investor Conference by Erste Group,
BIO-Europe 2020 – one of the global biotechnology flagship events.
Important events in Q1 2021, until the report publication date

January 2021:

Ryvu has submitted a new Clinical Trial Application (CTA), seeking approval to commence a Phase I/II trial, investigating the safety and efficacy of RVU120 in patients with relapsed / refractory metastatic or advanced solid tumors. The study will start in selected investigational sites in Europe, and later on at start of phase II, will expand also to other regions.
Ryvu Therapeutics’ CTA to commence the First In Human (FIH), Phase I trial investigating RVU120, a selective CDK8/CDK19 inhibitor, in patients with Acute Myeloid Leukemia (AML) or High-Risk Myelodysplastic Syndrome (HRMDS), who have failed the prior standard treatment, has been fully approved by the Polish Office for Registration of Medicinal Products, Medical Devices and Biocidal Products, and the respective Central Ethics Committee. Following these approvals, Ryvu Therapeutics can expand the clinical trial already ongoing in the United States also in Poland, aiming to assess the safety and tolerability of RVU120, as well as to determine the Recommended Phase II Dose (RP2D) of the study drug, in participants with AML or HRMDS.
March 2020: Ryvu Therapeutics has concluded a service agreement with Covance Inc. to conduct a Phase I (dose escalation) part of a Phase I/II clinical study – aimed at determining the safety and efficacy profile of RVU120 (SEL120) in patients with relapsed/refractory metastatic or advanced solid tumors.

Ryvu Financial Results 2020 acc. to IFRS

In 2020 Ryvu Therapeutics recognized total operating revenue of USD 9.6 million, which constitutes the increase of 9%* compared to the corresponding period in 2019. Revenues from partnering contracts have increased to USD 4.0 million from USD 1.0 million comparing to the corresponding period in 2019. Revenue from subsidies decreased to USD 5.5 million from USD 7.8 million in the previous period year.

Operational costs related in majority to the research and development expenditures amounted in 2020 to USD 18.7 million, as compared to USD 20.7 million in the same period last year. Operational loss has decreased and amounted to USD 9.2 million, compared to USD 11.8 million in 2019. Net loss amounted to USD 8.1 million compared to USD 11.5 million from the previous year. The realized financial result is in line with Ryvu’s strategy, since the company focuses on the increase of projects value, taking into account the plan of commercialization on later stages of development.

As of December 31, 2020, the value of the Company’s liquid financial resources amounted to USD 42.9 million. On March 10, 2021, all liquid financial resources amounted to USD 40.3 million – decrease is caused mainly by operating costs and capital expenditure.

*Percentage changes in the press release are calculated based on the functional currency [PLN].

On March 16, 2021 Sorrento Therapeutics, Inc. (Nasdaq: SRNE), reported that Dr. Henry Ji, Chairman and CEO, will participate in the following upcoming conference (Press release, Sorrento Therapeutics, MAR 16, 2021, View Source [SID1234576802]):

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CEO Roundtable during WuXi Healthcare Forum 2021

Dr. Henry Ji will participate in the CEO forum this evening and provide an update on Sorrento’s programs. Sorrento’s Management will be available during the CEO roundtable to answer questions.

Interested investors and industry partners can register through the following link:

View Source

On March 16, 2021 Elevation Oncology, a clinical stage biopharmaceutical company focused on the development of precision medicines for patients with genomically defined cancers, and NeoGenomics, Inc. (NASDAQ:NEO), a leading provider of cancer-focused genetic testing services and global oncology contract research services, reported a collaboration to enhance identification of patients with any solid tumor harboring an NRG1 fusion who may be eligible for enrollment in the Phase 2 CRESTONE study (Press release, Elevation Oncology, MAR 16, 2021, View Source;utm_medium=rss&utm_campaign=elevation-oncology-and-neogenomics-announce-collaboration-to-expand-genomic-testing-for-nrg1-fusions-across-solid-tumors-in-support-of-the-phase-2-crestone-study [SID1234576708]). NeoGenomics is a leading provider of next-generation sequencing (NGS) performing more than 50,000 NGS tests per year.

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NRG1 gene fusions are oncogenic driver alterations that have recently been identified in over 10 solid tumor types, with an overall estimated incidence of 0.2% in all solid tumors.1 Though rare, gene fusions are a proven target for precision oncology therapeutics, with over a dozen FDA-approved therapies now available for both hematologic and solid tumors driven by various gene fusions.2

"Our collaboration with Elevation Oncology reflects our shared pursuit of matching cancer patients to clinical trials and therapies that are precisely targeted to their unique tumor types and genomic biomarkers," said Douglas M. VanOort, Chairman and Chief Executive Officer of NeoGenomics. "Through cross-industry collaborations, we enable greater efficiency in the development and delivery of the latest advancements in both diagnostics and targeted therapies. Together, we work towards a future where every patient living with cancer has the opportunity to benefit from precision medicine."

As part of its comprehensive oncology test menu, NeoGenomics offers a variety of gene fusion panels that leverage RNA-based NGS to detect translocations and fusions with known and novel fusion partners across all solid tumors, including actionable gene fusions such as in ALK, BRAF, FGFR, NRG1, NTRK, PDGF, RET, and ROS1. In addition to all solid tumor panels, targeted panels are available in tumor types where gene fusions are commonly enriched:

Lung NGS Fusion Panel (Complete or Limited)
Cholangio/Pancreatic Carcinoma NGS Fusion Panel
Prostate NGS Fusion Panel
Thyroid NGS Fusion Panel
Targeted Solid Tumor NGS Fusion Panel
Universal Solid Tumor NGS Fusion Panel
RNA-based sequencing is the most sensitive detection method for structural variants that span large intronic regions, such as those leading to NRG1 gene fusions, which may not be detectable by most DNA-based sequencing techniques reliant on short-read methods. In addition, the use of RNA-based NGS furthers the scientific understanding of gene fusions by enabling full characterization of gene fusion partners and nucleotide-level resolution of fusion junctions.

"Accurate and widespread identification of gene fusions is critical to ensure patients can be matched to the growing number of approved and investigational precision therapies for these key oncogenic drivers," said Shawn Leland, PharmD, RPh, Founder and CEO of Elevation Oncology. "We are pleased to partner with NeoGenomics to ensure that across any solid tumor, each positive NRG1 fusion test result has the potential to be met with an actionable therapeutic opportunity in our Phase 2 CRESTONE study."

Patients and physicians can learn more about the CRESTONE study at www.nrg1fusion.com or on www.ClinicalTrials.gov under the NCT number NCT04383210.

On March 16, 2021 Aprea Therapeutics, Inc. (Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate the mutant tumor suppressor protein, p53, reported financial results for the three months and year ended December 31, 2020 and provided a business update (Press release, Aprea, MAR 16, 2021, View Source [SID1234576732]).

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"Though disappointed the topline complete remission rate from the Phase 3 clinical trial narrowly missed its primary endpoint, we continue to analyze the totality of the data from the study to understand those differences from our prior Phase 2 experience in frontline MDS patients and expect to present these findings in the second quarter of 2021," said Christian S. Schade, Chairman and Chief Executive Officer of Aprea. "Our dedicated team remains committed to the clinical development of eprenetapopt and our next generation, oral p53 reactivator, APR-548, in hematological and solid tumor malignancies. In 2021, we look forward to sharing data from our current clinical studies as well as our plans to expand the clinical pipeline to include new indications."

Business Operations Update:

The Company is conducting, supporting, and planning multiple clinical trials of eprenetapopt (APR-246) and APR-548:

Pivotal Phase 3 MDS Trial—In December 2020, the Company announced its pivotal Phase 3 randomized, controlled trial evaluating eprenetapopt with azacitidine as frontline therapy in HMA-naïve TP53 mutant myelodysplastic syndromes (MDS) patients failed to meet its predefined primary endpoint of complete remission (CR) rate. Analysis of the primary endpoint at this data cut demonstrated a 53% higher number of patients achieving a CR in the experimental arm receiving eprenetapopt with azacitidine versus the control arm receiving azacitidine alone but did not reach statistical significance. The Company is completing analysis from this Phase 3 clinical trial and expects to present additional information in the second quarter of 2021.
Phase 2 MDS/AML Post-Transplant Trial – The Company has completed enrollment of 33 patients in a single-arm, open-label Phase 2 clinical trial evaluating eprenetapopt with azacitidine as post-transplant maintenance therapy in TP53 mutant MDS and AML patients who have received an allogeneic stem cell transplant. The Company anticipates initial results from the primary endpoint of relapse-free survival at 12 months in the second quarter of 2021.
Phase 1/2 AML Trial – The Company is currently enrolling a Phase 1/2 clinical trial evaluating the safety, tolerability, and preliminary efficacy of eprenetapopt therapy in TP53 mutant AML patients. The lead-in portion of the trial evaluated the tolerability of eprenetapopt with venetoclax, with or without azacitidine, and no dose-limiting toxicities were observed in 12 patients receiving either regimen. Based on these results, the Company has expanded the trial to treat 33 additional frontline TP53 mutant AML patients with the combination of eprenetapopt, venetoclax and azacitidine. In the 19 frontline AML patients who are evaluable for efficacy with the triplet regimen, the Company has observed a 63% CR + CRi composite response rate and a 31% CR rate. The Company anticipates completion of enrollment in the triplet regimen expansion cohort during the second quarter of 2021 with availability of preliminary response rate data from the cohort also in the second quarter of 2021.
Phase 1 NHL Trial – The Company is currently enrolling a Phase 1 clinical trial in relapsed/refractory TP53 mutant chronic lymphoid leukemia (CLL) assessing eprenetapopt with venetoclax and rituximab and eprenetapopt with ibrutinib in order to further assess eprenetapopt in hematological malignancies. The first patient was enrolled in the first quarter of 2021. The Company is also planning to evaluate the combination of eprenetapopt with venetoclax in relapsed/refractory mantle cell lymphoma.
Phase 1/2 Solid Tumor Trial – The Company is currently enrolling a Phase 1/2 clinical trial in relapsed/refractory gastric, bladder and non-small cell lung cancers assessing eprenetapopt with anti-PD-1 therapy. The dose-escalation phase of the trial enrolled 6 patients with advanced solid tumors and no dose-limiting toxicities were observed. Based on these results, the Company is enrolling expansion cohorts for patients with advanced gastric, bladder and non-small cell lung cancers and has currently enrolled 8 patients across these expansion arms.
APR-548 — The Company’s second product candidate, APR-548, is a next-generation p53 reactivator that is being developed in an oral dosage form. The Company has planned a Phase 1 dose-escalation clinical trial evaluating the safety, tolerability, and preliminary efficacy of APR-548 with azacitidine in frontline and relapsed/refractory MDS patients. The Company anticipates the first patient to be enrolled early in the second quarter of 2021.
Fourth Quarter Financial Results

Cash and cash equivalents: As of December 31, 2020, the Company had $89.0 million of cash and cash equivalents compared to $130.1 million of cash and cash equivalents as of December 31, 2019. The Company expects cash burn for the full year 2021 to be between $30.0 million $35.0 million. The Company believes its cash and cash equivalents as of December 31, 2020 will be sufficient to meet its current projected operating requirements into 2023.
Research and Development (R&D) expenses: R&D expenses were $9.3 million for the quarter ended December 31, 2020, compared to $8.0 million for the comparable period in 2019. The increase in R&D expenses was primarily related to the continued development of the Company’s lead product candidate, eprenetapopt, in the following ongoing clinical trials; its pivotal Phase 3 clinical trial of eprenetapopt with azacitidine for frontline treatment of TP53 mutant MDS, its Phase 1/2 clinical trial for the treatment of TP53 mutant AML with venetoclax and azacitidine, its Phase 1/2 clinical trial in relapsed/refractory gastric, bladder and non-small cell lung cancers assessing eprenetapopt with anti-PD-1 therapy, its Phase 1 clinical trial in relapsed/refractory TP53 mutant chronic lymphoid leukemia (CLL) assessing eprenetapopt with venetoclax and rituximab, and eprenetapopt with ibrutinib and its Phase 2 post-transplant MDS/AML clinical trial.
General and Administrative (G&A) expenses: G&A expenses were $4.9 million for the quarter ended December 31, 2020, compared to $3.9 million for the comparable period in 2019. The increase in G&A expenses was primarily due to increases in non-cash stock-based compensation, insurance expense and commercial development expense.
Net loss: Net loss was $15.4 million, or $0.73 per share for the quarter ended December 31, 2020, compared to a net loss of $13.1 million, or $0.64 per share for the quarter ended December 31, 2019. The Company had 21,186,827 shares of common stock outstanding as of December 31, 2020.