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.

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 Calithera Biosciences, Inc. (Nasdaq: CALA), a clinical stage biotechnology company focused on discovering and developing novel small molecule drugs for the treatment of cancer and other life-threatening diseases, reported its financial results for the fourth quarter ended December 31, 2020. As of December 31, 2020, cash, cash equivalents and investments totaled $115.2 million (Press release, Calithera Biosciences, MAR 16, 2021, View Source [SID1234576729]).

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"In the fourth quarter, we maintained a strong cash position and continued to advance our key clinical development programs, including the KEAPSAKE clinical trial evaluating telaglenastat in non-small cell lung cancer in patients with NRF2/KEAP1 genetic mutations, and the Ph1b trial of our arginase inhibitor CB-280 in cystic fibrosis patients," said Susan Molineaux, PhD, president and chief executive officer of Calithera. "We look forward to sharing interim data from the KEAPSAKE trial and the results of the CB-280 trial in cystic fibrosis patients, each in the second half of 2021."

Fourth Quarter 2020 and Recent Highlights

Announced top-line results of randomized CANTATA trial of telaglenastat with cabozantinib in advanced renal cell carcinoma (RCC). The Phase 2 CANTATA trial is a global, randomized, double-blind clinical trial of telaglenastat combined with cabozantinib, in patients with advanced or metastatic RCC who have received one or two prior treatments. On January 4, 2021, Calithera announced topline results from the CANTATA clinical study and reported the trial did not meet the primary endpoint of improving progression free survival (PFS) in the study population.
Continued enrollment of the Phase 2 randomized KEAPSAKE trial in non-small cell lung cancer (NSCLC) patients with genetic mutation NRF2/KEAP1. The double-blind KEAPSAKE trial will enroll approximately 120 patients with stage IV non-squamous NSCLC with tumors that have the KEAP1 or NRF2 mutation. Patients will be randomized to receive telaglenastat or placebo, in combination with pembrolizumab, carboplatin and pemetrexed. The study will evaluate the safety and investigator-assessed progression-free survival (PFS) of telaglenastat plus this standard-of-care chemoimmunotherapy regimen. Calithera anticipates sharing interim data from the KEAPSAKE trial in the second half of 2021.
Initiated a Phase 1b clinical trial of CB-280 in patients with cystic fibrosis. In October, Calithera presented a trial in progress poster at the North American Cystic Fibrosis 2020 Virtual Conference. The presentation included preclinical study results which suggest CB-280 significantly improved lung function and reduced Pseudomonas aeruginosa colony-forming units in pre-clinical models. Arginase inhibition with CB-280 resulted in improved central airway resistance in CFTR knockout mice, and decreased lung infection in wild type and DeltaF508-CFTR-expressing mice infected with Pseudomonas aeruginosa. Enrollment in the Ph1b study is ongoing and Calithera expects to share data in the second half of 2021. In November 2020, Calithera was awarded up to $2.4M from the Cystic Fibrosis Foundation to support clinical development of CB-280.

Presented preclinical data for CB-668 IL4I1 program at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in November. CB-668 is a potent, selective, small-molecule, oral inhibitor of IL4I1, an amino acid oxidase that inhibits anti-tumor immunity and promotes tumor growth. IL4I1 regulates several aspects of adaptive immunity, including inhibition of cytotoxic T cells through its production of both hydrogen peroxide and activators of the aryl hydrocarbon receptor. CB-668 increases pro-inflammatory gene expression in tumors leading to an anti-tumor effect in mouse tumor models.
Selected Fourth Quarter and Full Year 2020 Financial Results

Cash, cash equivalents and investments totaled $115.2 million at December 31, 2020, which management believes will be sufficient to meet its current operating plan through 2022.

Research and development expenses for the full year 2020 were $71.0 million, compared to $76.3 million in the prior year. The decrease of $5.3 million was due to a $6.2 million decrease in the INCB001158 program and a $3.8 million decrease in early-stage research programs, partially offset by an increase of $2.7 million in the telaglenastat program and an increase of $2.0 million in the CB-280 program. Research and development expenses for the fourth quarter of 2020 were $17.1 million, compared to $17.9 million for the same period last year.

General and administrative expenses for the full year 2020 were $20.4 million, compared to $16.6 million in the prior year. The increase of $3.8 million was primarily related to a $2.5 million increase in personnel-related and facility costs and a $1.3 million increase in professional services costs. General and administrative expenses for the fourth quarter of 2020 were $5.6 million, compared to $4.6 million for the same period last year.

Interest and other income, net for the full year 2020 was $1.3 million, compared to $3.0 million in the prior year, mainly as a result of lower interest rates. Interest and other income, net for the fourth quarter of 2020 was $0.1 million, compared to $0.7 million for the fourth quarter of 2019.

Net loss for the three months and year ended December 31, 2020, was $22.6 million and $90.1 million, respectively.

Conference Call Information

Calithera will host an update conference call today, Tuesday, March 16, at 5:00 p.m. Eastern Time/2:00 p.m. Pacific Time. The call may be accessed by dialing (855) 783-2599 (domestic) or (631) 485-4877 and referring to conference ID 6250035. To access the live audio webcast or the subsequent archived recording, visit the Investors section of the Calithera website at www.calithera.com. The webcast will be recorded and available for replay on Calithera’s website for 30 days.

On March 16, 2021 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) ("Infinity" or the "Company"), a clinical-stage biotechnology company developing eganelisib (IPI-549), a potentially first-in-class, oral, immuno-oncology macrophage reprogramming therapeutic, reported its full year 2020 financial results and provided an update on the Company, including recent progress with eganelisib (Press release, Infinity Pharmaceuticals, MAR 16, 2021, View Source [SID1234576728]).

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"Over the past months, we have presented transformative data across multiple indications, I/O combinations, and lines of therapy which together demonstrate the broad potential of eganelisib to improve upon standard of care therapy across a broad range of treatment settings addressing some of the most challenging unmet needs in oncology," said Adelene Perkins, Chief Executive Officer and Chair of Infinity Pharmaceuticals. "Our data from MARIO-275, a randomized, double-blind, placebo controlled study, clearly demonstrate the benefit of adding eganelisib to nivolumab monotherapy, a standard of care in advanced 2L urothelial cancer, with improved ORR, DCR and PFS as compared to nivolumab monotherapy. Striking benefits were observed in the PD-L1 low patient population, who are underserved by checkpoint inhibitor monotherapy and represent the majority of this patient population. Based on these results, we are planning a registration enabling study in this population.

"Compelling safety and efficacy results presented at SITC (Free SITC Whitepaper), SABCS, and GU ASCO (Free ASCO Whitepaper) suggest that eganelisib may have broad potential to improve outcomes in both PD-L1 low and high patients across tumor types, driven by the immune modulatory mechanism of eganelisib targeting macrophages and other myeloid-derived immune cells," said Brian Schwartz, M.D., consulting Chief Physician of Infinity. "Our initial results from MARIO-3 show that adding eganelisib to an approved front-line regimen in TNBC provide clear patient benefit above standard-of-care atezolizumab and nab-paclitaxel. We observed tumor reductions in 100% of evaluable patients, irrespective of PD-L1 status, and are excited by the broad potential of this novel triplet regimen in both PD-L1 low and high patients given the significant unmet need in this aggressive and difficult-to-treat disease. We look forward to presenting additional data in the first and second half of this year, which will include increased patient numbers, response rate, disease control rate and initial response durability data, with preliminary PFS data by the end of the year. In parallel, the RCC cohort of MARIO-3 continues to advance with data from this proof-of-concept study in a novel triplet regimen expected in the first half of 2022. With our recently strengthened balance sheet, we will continue to advance our development of eganelisib across multiple indications, leveraging data from MARIO-275 in UC, MARIO-3 in TNBC, as well as the ARC-2 data presented by Arcus Biosciences at SABCS in second line TNBC and are evaluating opportunities to further unlock the potential of eganelisib in melanoma and SCCHN from MARIO-1 and in ovarian cancer from the ARC-2 study as we seek to improve outcomes in multiple treatment settings and patient populations including those least likely to respond to immunotherapies."

Recent Updates and Program Guidance:

MARIO-275 and Advanced Urothelial Cancer

Presented positive data from the MARIO-275 randomized, placebo-controlled Phase 2 study evaluating eganelisib in combination with Opdivo in platinum-refractory, I/O naïve patients with advanced urothelial cancer (aUC), in collaboration with Bristol Myers Squibb (BMS) at the 2021 ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium
Combination of eganelisib with nivolumab demonstrated improved ORR, DCR, and PFS versus 2L standard of care nivolumab monotherapy
Greatest benefit of eganelisib with nivolumab combination therapy over nivolumab monotherapy was observed in the PD-L1 low patient population (n=23) with improvement over nivolumab monotherapy (n=7) for overall response rate (ORR) (26% vs. 14%); disease control rate (DCR) (57% vs. 14%); and best responses of complete response (CR) (9% vs. 0%), and stable disease (SD) (30% vs. 0%)
PD-L1 low patients demonstrated an extended progression free survival (PFS) with a hazard ratio of 0.54 reflecting a 46% reduction in probability of progression
The combination of eganelisib and nivolumab was well tolerated at the 30mg once daily dose
Translational data support immune modulatory mechanism of action of eganelisib with increased immune activation and decreased immune suppression observed in both the PD-L1 high and low patient populations
Infinity is planning a registration enabling study leveraging findings from MARIO-275. After initial feedback from the U.S. Food and Drug Administration (FDA), additional trial details are expected by the end of Q2 2021, and will likely focus on PD-L1 low aUC patients with a 30 mg QD dose of eganelisib.
MARIO-3 Triple Negative Breast Cancer Cohort

Presented positive data from the ongoing Phase 2 study in collaboration with Roche/Genentech to evaluate eganelisib in a novel triple combination in the front-line setting, adding to the approved doublet of Tecentriq and Abraxane in triple negative breast cancer (TNBC) at the 2020 San Antonio Breast Cancer Symposium
100% of evaluable patients (n=13) demonstrated tumor reduction with a clinical benefit (disease control rate) in 92% of patients (12/13)
69.2% (9/13) overall response rate (ORR) with best responses of complete response (CR) or partial response (PR)
100% (5/5) ORR (CR + PR) with 1 CR and 4 PRs observed in PD-L1 high patients
50% (4/8) ORR (CR + PR) with 4 PRs observed in PD-L1 low patients
The novel triple combination treatment with eganelisib, atezolizumab (atezo) and nab-paclitaxel (nab-pac) demonstrated safety in line with expectations of the component drugs with no additive or new safety signals
Translational data support immune modulatory mechanism of action of eganelisib with increased immune activation and decreased immune suppression observed in both the PD-L1 high and low patient populations
Additional data presentations from the TNBC cohort of MARIO-3 are expected in the first and second half of 2021 with increased patient numbers and early durability data which will include progression free survival in the second half of 2021.
Completion of enrollment is expected in the 2H 2021.
MARIO-3 Renal Cell Carcinoma Cohort

Data from the ongoing, proof-of-concept novel triple combination of eganelisib with Tecentriq and Avastin in the front line setting in renal cell cancer (RCC) are expected in the first half of 2022.
MARIO-1

MARIO-1 melanoma and squamous cell carcinoma of the head and neck (SCCHN) cohorts were designed to isolate the clinical benefit of eganelisib by examining clinical activity in patients not expected to respond to checkpoint inhibitors due to progression following immediately prior checkpoint inhibitor therapy which were presented at the 35th Anniversary Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper). The data presented demonstrated that eganelisib had a manageable safety and tolerability profile with ORR ≥ 20% and DCR ≥ 40% in melanoma and SCCHN patients who had progressed on a CPI as their immediate prior therapy after having ≤ 2 prior lines of therapy and support the ongoing strategic prioritization of earlier lines of treatment with eganelisib.
Arcus Collaboration

Data presented at the 2020 San Antonio Breast Cancer Symposium from the Phase 1b collaboration study conducted by Arcus Biosciences, evaluating a checkpoint-inhibitor free, novel triple-combination regimen of eganelisib + etrumadenant (AB928, a dual adenosine receptor antagonist) + Doxil in advanced TNBC patients demonstrated improved activity on top of the doublet therapy of etrumadenant +Doxil. The triplet including eganelisib demonstrated numerical ORR benefit over the doublet in the overall cohort population (42% vs 11%) as well as in the ovarian cancer patient subset (75% vs 14%) and the TNBC patient subset (25% and 9%).
Corporate Update

Significantly strengthened balance sheet with the successful completion in February 2021 of a $92 million public offering with approximately $86 million net proceeds to support execution on the next phase of eganelisib development.
Full Year 2020 Financial Results:

At December 31, 2020, Infinity had total cash, cash equivalents and available-for-sale securities of $34.1 million, compared to $42.4 million at December 31, 2019.
Revenue during 2020 was $1.7 million, which relates to royalties on net sales of Copiktra from Verastem, Inc. and Secura Bio, Inc., who purchased the rights to Copiktra in 2020. Revenue during 2019 was $3.0 million which primarily relates to the achievement of a $2.0 million milestone from PellePharm.
Research and development expense for 2020 was $26.8 million, compared to $27.1 million in 2019.
General and administrative expense was $12.4 million for 2020, compared to $14.3 million for 2019. The decrease in G&A expense in 2020 compared to 2019 was primarily due to a decrease of $0.8 million in compensation, primarily related to a reduction in stock compensation, and a decrease of $0.5 million in professional services.
Net loss for 2020 was $40.5 million, or a basic and diluted loss per common share of $0.68, compared to a net loss of $47.1 million, or a basic and diluted loss per common share of $0.83 for 2019. The decrease in net loss was mostly driven by a decrease in royalty expense in 2020. In 2019, Takeda consented to the sale of the royalties to HealthCare Royalty Partners III, L.P. (HCR) and agreed to forego its rights to an equal share of the royalties due from Secura Bio. In exchange, we paid Takeda $6.7 million representing 25% of the net proceeds from the royalty monetization which we recognized as royalty expense in 2019.
Financial Outlook: Infinity’s 2021 financial guidance, following the closing in February 2021 of a $92 million public offering of Infinity’s common stock is as follows:

Net Loss: Infinity expects net loss for 2021 to range from $40 million to $50 million.
Cash and Investments: Infinity expects to end 2021 with a year-end-cash, cash equivalents and available for sale securities balance ranging from $70 million to $80 million.
Infinity’s financial guidance does not include
additional funding or business development activities, or
a potential $5 million milestone payment from BVF for positive patidegib Phase 3 data and any milestones from, or the sale of the company’s equity interest in, PellePharm
Conference Call Information

Infinity will host a conference call today, March 16, 2021, at 4:30 p.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors/Media" section of Infinity’s website at www.infi.com. To participate in the conference call, please dial (877) 316-5293 (domestic) and (631) 291-4526 (international) five minutes prior to start time. The conference ID number is 7174736. An archived version of the webcast will be available on Infinity’s website for 30 days.

On March 16, 2021 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported its financial results for the fourth quarter and year ended December 31, 2020 (Press release, Curis, MAR 16, 2021, View Source [SID1234576727]).

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"2020 was a transformative year for Curis, as we made significant progress in our mission to develop the next generation of targeted cancer therapies that meaningfully improve and extend the lives of patients. Despite the difficulties and uncertainty brought about by the ongoing coronavirus pandemic, we significantly advanced and expanded each program in our clinical pipeline, headlined by the very encouraging data from our Phase 1 trials of lead asset, CA-4948, presented in December in conjunction with ASH (Free ASH Whitepaper)," said James Dentzer, President and Chief Executive Officer of Curis. "We look forward to providing additional updates on our IRAK4 program throughout the year, with clinical data readouts from our ongoing studies, including the CA-4948 monotherapy study in AML/MDS and the CA-4948/ibrutinib combination study for patients with R/R hematologic malignancies that we initiated in early 2021. We also continue to enroll patients and bring additional trial sites online in our Phase 1a/1b trial of CI-8993, our first-in-class monoclonal anti-VISTA antibody for the treatment of patients with R/R solid tumors, and look forward to providing preliminary data from this exciting study later this year."

Mr. Dentzer continued, "2020 was also a pivotal year on the corporate side for Curis. Through the execution of several key financings and partnerships, we have the resources needed to advance our programs through their next data catalysts, while also providing us the ability to invest efficiently in our pipeline of first-in-class cancer therapeutics. We are excited about the opportunities stemming from our Q4 signing of the CRADA with the NCI in addition to the recently announced Phase 2 IST of CA-4948 for the treatment of anemia in patients with lower-risk MDS led by Dr. Uwe Platzbecker at Universität Leipzig. These new partnerships provide powerful validation of our IRAK4 platform and allow us to leverage the resources of premier research organizations to significantly expand the reach of our clinical and preclinical programs."

Fourth Quarter 2020 and Recent Operational Highlights

Precision oncology, CA-4948 (IRAK4 Inhibitor; Aurigene collaboration):

In December 2020, Curis announced positive preliminary data from its ongoing Phase 1 study of CA-4948 monotherapy in patients with R/R AML and high-risk MDS, including marrow blast reductions observed in all evaluable patients and 2 of 6 evaluable patients experiencing a marrow complete response. Curis continues to enroll patients, is currently enrolling in the 500mg BID dose cohort of the study and expects to report additional data in mid-year 2021.
In December 2020, Curis provided updated preliminary data from its ongoing Phase 1 study of CA-4948 showing durable and dose-dependent reductions in tumor burden in patients with R/R NHL and announced the recommended Phase 2 dose, in addition to the identification of two potentially predictive biomarkers demonstrating target engagement and potential for patient enrichment, in an oral presentation at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.
In February 2021, Curis announced the dosing of the first patient in its Phase 1 dose-escalation and expansion study of CA-4948, an IRAK4 kinase inhibitor, and the BTK inhibitor, ibrutinib, for the treatment of patients with relapsed or refractory hematologic malignancies. In preclinical models, CA-4948 demonstrated synergistic anti-cancer activity when combined with a potent BTK inhibitor such as ibrutinib.
Approximately 18 patients will be enrolled in the dose-escalation portion and will receive starting dose and escalation doses that have been observed to be safe and effective, combined with ibrutinib doses appropriate for their respective NHL subtype.
The primary endpoints of Part 1 will be determination of maximum tolerated dose (MTD), and the recommended Phase 2 dose (RP2D).
Part 2 of the study will enroll patients across a basket of four cohorts:
Marginal zone lymphoma (MZL)
Activated B-cell subtype of Diffuse Large B-cell Lymphoma (ABC-DLBCL)
Primary central nervous system lymphoma (PCNSL)
NHL with adaptive ibrutinib resistance.
An interim futility analysis will be conducted after approximately 15-20 patients are enrolled in each cohort.
Primary endpoints of Part 2 will be complete response or objective response rate and duration of response.
Curis expects to report initial data from the study in the fourth quarter of 2021.
In February 2021, Curis announced the initiation of the investigator-sponsored Phase 2 LUCAS trial of CA-4948 for the treatment of anemia in patients with very low, low, or intermediate-risk MDS. The trial is expected to start recruitment in the second quarter of 2021 and is expected to enroll 84 patients across two cohorts:
Cohort A: Erythropoiesis stimulating agent (ESA) refractory/intolerant patients
Cohort B: ESA naïve patients with transfusion dependence (min. 20 patients) or transfusion independence (min. 20 patients)
Patients in both cohorts will receive 300mg CA-4948 twice-daily (BID) for 21 days in at least four repeating cycles lasting 28 days each.

The primary endpoint of the study is to evaluate the proportion of patients that develop an erythroid response (HI-E) according to IWG 2018 criteria.

Immuno-oncology, CI-8993 (anti-VISTA antibody; ImmuNext collaboration):

In November 2020, Curis published trial design details from its ongoing Phase 1a/1b dose-escalation study of its first-in-class monoclonal anti-VISTA antibody for the treatment of R/R solid tumors.
Curis continues to enroll patients in the study and expects to report initial safety and efficacy data in the second half of 2021.
Corporate:

In December 2020, Curis closed an underwritten public offering of 29,500,000 shares of its common stock, including the exercise in full by the underwriters of their option to purchase up to an additional 3,847,826 shares, raising gross proceeds of approximately $169.6 million before deducting underwriting discounts and commissions and offering expenses.
In November 2020, Curis entered into a CRADA with the NCI. Under the CRADA, Curis will collaborate with the NCI Experimental Therapeutics Program (NExT) and the NCI Cancer Therapy Evaluation Program to conduct non-clinical and clinical studies of Curis’ proprietary compound, CA-4948, an IRAK4 kinase inhibitor that acts as a Toll-like Receptor (TLR) suppressor, as an anti-cancer agent.
Upcoming 2021 Planned Milestones

Report additional clinical data from the Phase 1 study of CA-4948 in patients with AML and high-risk MDS, including patients with spliceosome mutations that encode oncogenic IRAK4-L in mid-year 2021.
Report additional clinical biomarker data gathered in the Phase 1 study of CA-4948 in patients with R/R NHL in mid-year 2021.
Announce initial safety and efficacy data from the ongoing Phase 1 study of CA-4948 in combination with ibrutinib in patients with R/R NHL in the second half of 2021.
Report initial safety and efficacy data from the ongoing Phase 1a/1b dose-escalation study of CI-8993 for the treatment of R/R solid tumors in the second half of 2021.
Full Year and Fourth Quarter 2020 Financial Results

For the year ended December 31, 2020, Curis reported a net loss of $29.9 million, or $0.61 per share on both a basic and diluted basis, as compared to a net loss of $32.1 million, or $0.97 per share on both a basic and diluted basis in 2019. For the fourth quarter of 2020, Curis reported a net loss of $7.5 million or $0.11 per share on both a basic and diluted basis, as compared to a net loss of $8.6 million, or $0.26 per share on both a basic and diluted basis for the same period in 2019.

Revenues for the year ended December 31, 2020, were $10.8 million as compared to $10.0 million for the same period in 2019. Revenues for both periods comprise primarily royalty revenues recorded on Genentech and Roche’s net sales of Erivedge. Revenues for the fourth quarters of 2020 and 2019 were $3.0 million and $3.3 million, respectively.

Operating expenses for the year ended December 31, 2020 were $35.7 million as compared to $34.4 million for the same period in 2019. Operating expenses for the fourth quarter of 2020 were $9.3 million, as compared to $10.6 million for the same period in 2019, and comprised the following:

Costs of Royalty Revenues. Costs of royalty revenues, primarily amounts due to third-party university patent licensors in connection with Genentech and Roche’s Erivedge net sales, were $0.5 million for the years ended December 31, 2020 and 2019. Cost of royalty revenues were $0.2 million for the fourth quarter of 2020 and 2019.

Research and Development Expenses. Research and development expenses were $23.1 million for the year ended December 31, 2020, as compared to $22.3 million for the same period in 2019. Research and development expenses were $5.6 million for the fourth quarter of 2020 as compared to $7.5 million for the same period in 2019. The decrease was primarily due to a decrease in clinical and manufacturing costs related to CA-170 and fimepinostat.

General and Administrative Expenses. General and administrative expenses were $12.1 million for the year ended December 31, 2020, as compared to $11.6 million for the same period in 2019. General and administrative expenses were $3.5 million for the fourth quarter of 2020, as compared to $3.0 million for the same period in 2019. The increase was primarily due to an increase in personnel related costs.

Other Expense, Net. Net other expense was $5.0 million for the year ended December 31, 2020, as compared to $7.8 million for the same period in 2019. For the fourth quarter of 2020 and 2019, net other expense was $1.2 million and $1.3 million, respectively. Net other expense primarily consisted of imputed interest expense related to future royalty payments.

As of December 31, 2020, Curis’s cash, cash equivalents and investments totaled $183.1 million, and there were approximately 91.5 million shares of common stock outstanding. Curis expects that its existing cash, cash equivalents and investments should enable it to maintain its planned operations into 2024.

Conference Call Information

Curis management will host a conference call today, March 16, 2021, at 4:30 p.m. ET, to discuss these financial results, as well as provide a corporate update.

To access the live conference call, please dial 1-888-346-6389 from the United States or 1-412-317-5252 from other locations, shortly before 4:30 p.m. ET. The conference call can also be accessed on the Curis website at www.curis.com in the Investors section.