On March 10, 2020 Sunesis Pharmaceuticals, Inc. (Nasdaq: SNSS) reported financial results for the fourth quarter and year ended December 31, 2019 (Press release, Sunesis, MAR 10, 2020, View Source [SID1234555372]). Loss from operations for the three months and year ended December 31, 2019 was $5.4 million and $23.3 million. As of December 31, 2019, cash and cash equivalents, restricted cash, and marketable securities totaled $34.6 million.

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"We concluded 2019 having made solid progress across our portfolio. Vecabrutinib, our non-covalent BTK inhibitor, demonstrated a very favorable safety profile combined with evidence of clinical activity in patients with and without BTK C481-mutations. We continue to advance and characterize our proprietary PDK1 inhibitor, SNS-510, with findings supporting development in both hematologic and solid tumors. We are also building value in our product pipeline through partnerships. In December, we partnered vosaroxin with Denovo Biopharma and TAK-580 with DOT Therapeutics-1 to advance these programs to the market," said Dayton Misfeldt, Interim Chief Executive Officer of Sunesis. "Looking ahead, we remain on track to complete the Phase 1b dose escalation component of our Phase 1b/2 vecabrutinib trial in the second quarter and to advance SNS-510 to an IND by the end of year."

Vecabrutinib Phase 1b/2 Clinical Update. Since the presentation of clinical data through cohort 5 at ASH (Free ASH Whitepaper) in December 2019, Sunesis has enrolled patients in cohorts 6 and 7 of the ongoing Phase 1b/2 trial of vecabrutinib.

Cohort 5 (300mg): Sunesis announced at ASH (Free ASH Whitepaper) 2019 that stable disease was observed in three of five patients from cohort 5 (300mg BID). As of today, one chronic lymphocytic leukemia (CLL) patient remains on study in cycle 8 at 300mg BID with a 47% reduction in tumor burden at their second scan, improving from their initial 41% reduction, with normalized hematology parameters.

Cohort 6 (400mg BID): Four patients, three CLL and one diffuse large B cell lymphoma (DLBCL), were enrolled in the cohort. The DLBCL patient was nonevaluable due to disease progression during cycle one. The three CLL patients

completed the safety evaluation period, remain on treatment, and results of their first response assessments will be available later this month.

Cohort 7 (500mg BID): Six patients, four with CLL and two with mantle cell lymphoma (MCL), cleared the safety evaluation period and four of the patients remain on treatment. We expect first response assessments for these patients in the second quarter. Additional patients are being evaluated for the cohort.

Vecabrutinib has been very well tolerated, with no Grade 3 or higher drug-related adverse events reported to date across cohorts 3 – 7.

SNS-510, first-in-class PDK1 inhibitor. In October, at the 2019 AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper), Sunesis presented data profiling the oral PDK1 inhibitor SNS-510 showing potent activity in hematologic and solid tumor cancer models. New results of in vitro combination studies indicate that SNS-510 can combine synergistically with several drugs including inhibitors of CDK4/6, KRAS G12C, and BCL2. The IND-enabling program is progressing as planned and an IND filing is targeted for the end of 2020.

Partnering TAK-580 and vosaroxin. In December, Sunesis consented to Takeda Oncology’s assignment of our agreement relating to the pan-Raf inhibitor TAK-580 to DOT Therapeutics-1, Inc. ("DOT-1"). Coincident with the transaction, Sunesis and DOT-1 entered into a new agreement covering TAK-580 and DOT-1 paid Sunesis an upfront fee of $2.0 million. Under the new TAK-580 agreement, DOT-1 agreed to pay Sunesis up to $57.0 million in pre-commercialization milestone payments, plus royalties on future sales of TAK-580. Also in December, Sunesis licensed vosaroxin to Denovo Biopharma LLC ("Denovo"). Sunesis received a $0.2 million upfront payment and is eligible to receive up to $57.0 million in regulatory and commercial milestones, plus double-digit royalties on future sales of vosaroxin.

Financial Highlights

Cash and cash equivalents, restricted cash and marketable securities totaled $34.6 million as of December 31, 2019, compared to $13.7 million as of December 31, 2018. The increase of $20.9 million was primarily due to $45.1 million of net proceeds from the issuance of common and preferred stock, and $5.5 million of proceeds from the SVB loan, partially offset by $22.2 million net cash used in operating activities and a $7.5 million principal repayment of the prior loan from Western Alliance Bank and Solar Capital Ltd.

Revenue was $2.1 million in 2019 compared to $0.2 million in 2018. Revenue in both periods was derived from license agreements. The increase of $2.0 million in 2019 was primarily due to revenue recognized from the upfront payments received under the license agreements with DOT-1 and Denovo.

Research and development expense was $15.4 million in 2019 compared to $14.6 million in 2018, primarily relating to the vecabrutinib development program. The increase of $0.8 million in 2019 was primarily due to a $1.8 million increase in professional services and clinical expenses related to the preparation for the Phase 2 portion of our ongoing clinical trial for vecabrutinib, offset by a $1.0 million decrease in salary and personnel expenses.

General and administrative expense was $9.9 million in 2019 compared to $11.3 million in 2018. The decrease of $1.4 million in 2019 was primarily due to a $1.1 million decrease in salary and personnel expenses due in large part to lower stock-based compensation and a $0.8 million decrease in professional services expenses due to lower legal and vosaroxin patent expenses. The decreases in the comparable periods were partially offset by a $0.3 million increase in insurance premiums.

Interest expense was $0.5 million in 2019 compared to $1.2 million in 2018. The decrease in 2019 was primarily due to lower interest paid under the SVB Loan Agreement compared to the prior loan.

Cash used in operating activities was $22.2 million in 2019, compared to $24.4 million in 2018. Cash used in the 2019 period resulted primarily from the net loss of $23.3 million and changes in operating assets and liabilities of $0.7 million, offset by net adjustments for non-cash items of $1.8 million.

Loss from operations was $5.4 million and $23.3 for the three months and year ended December 31, 2019, compared to $5.8 million and $25.7 million for the same periods in 2018. Net loss was $5.3 million and $23.3 million for the three months and year ended December 31, 2019, compared to $6.0 million and $26.6 million for the same periods in 2018.

Conference Call Information

Sunesis will host a conference call today at 4:30 p.m. Eastern Time. The call can be accessed by dialing (844) 296-7720 (U.S. and Canada) or (574) 990-1148 (international) and entering passcode 2388763. To access the live audio webcast, or the subsequent archived recording, visit the "Investors and Media – Calendar of Events" section of the Sunesis website at www.sunesis.com. The webcast will be recorded and available for replay on the company’s website for two weeks

On March 10, 2020 Protagonist Therapeutics, Inc. (Nasdaq:PTGX) reported its financial results for the fourth quarter and full year ended December 31, 2019, and provided an update on its clinical development programs (Press release, Protagonist, MAR 10, 2020, View Source [SID1234555371]).

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"We are pleased to have made great progress in 2019, creating multiple opportunities to execute on our clinical development plans with three platform-generated therapeutic candidates," commented Dinesh V. Patel, Ph.D., Protagonist President and Chief Executive Officer. "Our priorities for 2020 include evaluating PTG-300 in multiple blood disorders with the intent of selecting the first clinical indication for a pivotal study, continuing Phase 2 development of PTG-200 with our partner Janssen Biotech, and advancing PN-943 into Phase 2 development in ulcerative colitis. Our financial position provides us with sufficient resources through the end of 2021 which should enable us to reach definitive conclusions for all of the ongoing clinical proof of concept studies."

Product Development Update

PTG-300: Injectable Hepcidin Mimetic for Blood Disorders

·The Company is conducting Phase 2 proof of concept studies with PTG-300 in patients with beta-thalassemia, polycythemia vera and hereditary hemochromatosis.
·In December 2019, the Company reported observations of dose-related reductions from high baseline serum iron and transferrin saturation (TSAT) levels in the ongoing open-label TRANSCEND Phase 2 study of PTG-300, supporting continued evaluation with additional dose regimens and longer follow-up time periods.
·An investigator-sponsored study of PTG-300 in patients with myelodysplastic syndromes, a fourth potential indication for PTG-300, is expected to begin in the first half of 2020.

PTG-200 (JNJ-67864238): Oral IL-23 Receptor Antagonist for Inflammatory Bowel Disease

Protagonist Therapeutics and Janssen Biotech are jointly conducting the development of PTG-200 (or JNJ-67864238) through completion of a Phase 2a study in patients with moderate-to-severe Crohn’s disease, with the anticipation of completion in the first half of 2021.
Protagonist achieved milestones leading to payments from Janssen Biotech of $25 million received in 2019 triggered by the decision to advance PTG-200 in a Phase 2a study and expansion of the existing collaboration agreement, and $5 million received in early 2020 on the nomination of a second-generation oral IL-23 receptor antagonist.

PN-943: Oral Alpha-4-Beta-7 Integrin Antagonist for Inflammatory Bowel Disease

·Results from a Phase 1 study of PN-943 demonstrated a sustained and superior target engagement as compared with the first-generation oral alpha-4-beta-7 integrin antagonist PTG-100. These results were highlighted in an oral presentation at the 2019 Digestive Disease Week conference.
·The Company plans to initiate a Phase 2 study in patients with ulcerative colitis in the second quarter of 2020, with topline data expected in the second half of 2021.
·Preclinical research findings on PN-943 were presented on February 14, 2020, at the 15th Congress of the European Crohn’s and Colitis Organization (ECCO) in Vienna.
·Preclinical research findings for PN-943 have been accepted for presentation at the 2020 Digestive Disease Week conference taking place May 2-5, 2020, in Chicago.

Financial Results

·Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities as of December 31, 2019 were $133.0 million. The Company expects current cash, cash equivalents and marketable securities and access to its debt facility to be sufficient to fund its planned operating and capital expenditures through year-end 2021.
·License and Collaboration Revenue: License and collaboration revenue of $2.7 million for the fourth quarter of 2019 was in line with $2.4 million for the same period of 2018. License and collaboration revenue for the full year 2019 was $0.2 million, compared to $30.9 million for 2018. The Company recognized $9.6 million of license and collaboration revenue for the full year 2019, which was offset by the one-time cumulative adjustment related to the application of revenue recognition principles following the May 2019 amendment of the Janssen Biotech collaboration agreement that reduced the revenue by $9.4 million for the year, resulting in net revenue recognition of $0.2 million for the full year 2019.
·Research and Development ("R&D") Expenses: R&D expenses for the fourth quarter and full year 2019 were $15.9 million and $65.0 million, respectively, as compared to $14.2 million and $59.5 million, respectively, for the same periods of 2018. The increases were primarily due to increased clinical development costs related to PTG-300 and PN-943, offset in part by lower clinical development costs related to PTG-200 and PTG-100.

·General and Administrative ("G&A") Expenses: G&A expenses for the fourth quarter and full year 2019 were $4.1 million and $15.7 million, respectively, as compared to $3.5 million and $13.7 million, respectively, for the same periods of 2018. The increases were primarily due to increases in salaries and employee-related expenses driven by increased headcount and professional services to support the growth in our operations.
·Net Loss: The fourth quarter net loss was $17.5 million, or a net loss of $0.63 per share, and the full year 2019 net loss was $77.2 million, or a net loss of $2.98 per share.

Conference Call and Webcast Information

Protagonist executives will host a conference call at 4:30 p.m. EDT/1:30 p.m. PDT today. To access the live call, dial 1-844-515-9178 (U.S./Canada) or 1-614-999-9313 (international) and refer to conference ID number 5591627. A live and archived webcast of the call will also be accessible in the Investors section of the Company’s website at www.protagonist-inc.com.

On March 10, 2020 Principia Biopharma Inc. (Nasdaq: PRNB), a late-stage biopharmaceutical company focused on developing treatments for immune-mediated diseases, reported financial results for the fourth quarter and full year ended December 31, 2019 (Press release, Principia Biopharma, MAR 10, 2020, View Source [SID1234555370]).

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"Our significant achievements in 2019 lay a strong foundation for us to advance our corporate strategy and the promise of our drug discovery platform. Including our program partnered with Sanofi, we now have proof of concept in three immune-mediated diseases: pemphigus, immune thrombocytopenia and multiple sclerosis. As a result of these clinical milestones, we are now well positioned to progress our clinical programs this year," said Martin Babler, president and chief executive officer of Principia.

Full Year 2019 and Recent Program Highlights

Rilzabrutinib for the treatment of pemphigus (pemphigus vulgaris (PV) and pemphigus foliaceus (PF))

Presented positive data from Phase 2 Part A trial at 2019 American Academy of Dermatology Late-Breaking session

Announced confirmatory preliminary data from Phase 2 Part B trial

Announced accelerated enrollment of Phase 3 pivotal trial– anticipating results in second half of 2021

Anticipated Upcoming Milestones:

1H20 – Presentation of data from Phase 2 Part B trial

Rilzabrutinib for the treatment of Immune Thrombocytopenia (ITP)

Presented positive data from ongoing Phase 1/2 trial in highly treatment-resistant and refractory patients at 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting

Anticipated Upcoming Milestones:

2H20 – Presentation of ITP data from Phase 1/2 trial

Rilzabrutinib for the treatment of IgG4-Related Disease (RD)

Announced expansion into IgG4-RD, an immune-mediated disease of chronic inflammation and fibrosis

Anticipated Upcoming Milestones:

1H20 – Initiation of Phase 2 trial for IgG4-RD

PRN473 Topical for the treatment of immune-mediated diseases

Initiated a third BTK inhibitor clinical program, a Phase 1, randomized, double blind, placebo-controlled, single and multiple dose clinical trial

Anticipated Upcoming Milestones:

2020 – Phase 1 trial results

PRN2246/SAR442168 for the treatment of Multiple Sclerosis (MS)

Presented positive Phase 1 data at Americas Committee for Treatment and Research in Multiple Sclerosis Forum 2019

In February 2020, Sanofi announced PRN2246/SAR442168 met its primary endpoint and was well tolerated in the Phase 2b trial with no new safety findings. Sanofi also announced it expects to initiate four Phase 3 clinical trials in relapsing and progressive forms of MS in the middle of 2020

PRN1371 for the treatment of bladder cancer

Suspended clinical program to focus our portfolio on immune-mediated diseases

General Corporate Updates

Raised $242 million through a public offering of 8,625,000 shares of common stock

Announced generic name for PRN1008 – rilzabrutinib

Fourth Quarter and Full Year 2019 Financial Results

Cash Position: Cash, cash equivalents, and marketable securities were $367.8 million as of December 31, 2019, compared to $180.6 million as of December 31, 2018. The increase in Principia’s cash position is mainly due to net proceeds of $226.5 million from our follow-on offering completed in the fourth quarter of 2019.

Revenues: We did not recognize any collaboration revenue for the three months ended December 31, 2019, compared to $26.1 million for the same period in 2018. Collaboration revenue for the full year of 2019 was $35.2 million, compared to $69.1 million for the full year of 2018. The decrease was due to the revenue recognition for portions of an upfront payment of $40.0 million received in 2017 and milestone payments totaling $25.0 million received in 2018 from Sanofi and an upfront payment of $15.0 million received in June 2017 from AbbVie Biotechnology Limited, which were fully recognized as of year-end 2018. The revenue recognized for the year ended 2019 was primarily for the achievement of a milestone in our Sanofi collaboration.

R&D Expenses: Total research and development expenses were $21.5 million for the three months ended December 31, 2019, including stock-based compensation expense of $1.9 million, compared to $13.7 million for the same period in 2018, including stock-based compensation expense of $0.8 million. For the full year of 2019, total research and development expenses were $74.1 million, including stock-based compensation expense of $6.6 million, compared to $40.5 million for full year of 2019, including stock-based compensation expense of $1.4 million. The increase in total research and development expenses was mainly driven by an increase in rilzabrutinib program costs, attributed to various manufacturing campaigns to supply drug products for our rilzabrutinib clinical trials and the initiation of a global Phase 3 trial in pemphigus in November 2018, as well as an increase in employee-related expenses.

G&A Expenses: General and administrative expenses were $5.1 million for the three months ended December 31, 2019, including stock-based compensation expense of $1.3 million, compared to $4.2 million for the same period in 2018, including stock-based compensation expense of $0.6 million. For the full year of 2019, general and administrative expenses were $19.8 million, including stock-based compensation expense of $5.5 million, compared to $11.5 million for the full year of 2018, including stock-based compensation

expense of $1.4 million. The increase in total general and administrative expenses was primarily driven by increased employee-related expenses and facility costs.

Net Income (Loss): For the three months ended December 31, 2019, net loss was $24.9 million compared to net income of $9.4 million for the same period in 2018. For the full year of 2019, net loss was $53.8 million, compared to net income of $18.2 million for the full year of 2018.

Financial Guidance: The company’s current cash position is anticipated to fund operations beyond the Phase 3 data readout in pemphigus, irrespective of any opt-in decision related to the Sanofi agreement, based on the current operating plan.

On March 10, 2020 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (INTASYL) therapeutic platform, reported that it has reached a new agreement with Medigene AG ("Medigene") in relation to Phio Pharmaceuticals’ previously announced research collaboration in August 2019 with the Helmholtz Zentrum München to design and develop novel candidates for the use of INTASYL compounds in adoptive cell therapy to enhance immune cell function (Press release, Phio Pharmaceuticals, MAR 10, 2020, View Source [SID1234555369]). Under the terms of the new agreement, Medigene will contribute expertise regarding clinical development as well as proprietary research material and has an option to an exclusive license for the clinical and/or commercial exploitation of the potential immune cell enhancers against certain fee payments.

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"We are pleased that Medigene will be contributing to our research alliance with the Helmholtz Zentrum München," said Dr. Gerrit Dispersyn, President and CEO of Phio. "The breadth of the INTASYL platform and the compound’s capability to augment the anti-tumor effectiveness of various cell-based approaches are key differentiators of the platform, and Medigene’s clinical development experience with personalized T cell therapies will be helpful in our efforts to identify potential new targets and therapies that INTASYL can enhance. The capabilities that Medigene will bring to the collaboration will be additive to the ongoing research efforts we have undertaken with Prof. Dr. Elfriede Nößner and her team at the Helmholtz Zentrum München as we remain committed to exploring and identifying novel targets to strengthen our immuno-oncology product candidate portfolio."

"We are delighted to continue to work with Phio Pharmaceuticals and Helmholtz Zentrum München within this new agreement," said Prof. Dolores J. Schendel, CEO and CSO of Medigene. "We look forward to the outcomes of this collaborative research effort and exploring the potential future options."

Said Prof. Dr. Elfriede Nößner, Head of Immunoanalytics at Helmholtz Zentrum München, "I am very excited about this 3-party agreement. Connecting pharma and clinical stage biotechnology with academic research is an enormous asset to the growing field of immunotherapy. It promises seamless translation of basic science knowledge to patient benefit."

On March 10, 2020 NuCana plc (NASDAQ: NCNA) reported preliminary results from part one of the Phase II study of single-agent Acelarin in patients with platinum-resistant ovarian cancer (PRO-105) (Press release, Nucana BioPharmaceuticals, MAR 10, 2020, View Source [SID1234555368]). This part of the study compared a 500mg/m2 dose of Acelarin versus a 750mg/m2 dose of Acelarin in patients who were heavily pre-treated (at least 3 prior lines of chemotherapy). This study is now closed to recruitment and data analysis from part one of the study is ongoing.

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Forty-five patients with platinum-resistant ovarian cancer were evaluable for response and all responses had confirmatory scans. Based on an assessment by blinded independent central review, one patient achieved a complete response and two patients achieved partial responses. In addition, 16 patients achieved stable disease.

Patients who entered PRO-105 were heavily pre-treated, having received a median of five prior lines of treatment, and 72% had at least one comorbidity at study entry. Highlighting the fragility of this difficult-to-treat patient population, 45% of patients did not complete the first cycle of treatment with Acelarin despite not having any disease progression or any serious Grade 3 or 4 adverse events. However, for 23 patients in the study who received two or more cycles of Acelarin, the confirmed response rate was 13% and the disease control rate was 83%. These data are still being analyzed and the findings remain preliminary and subject to change.

NuCana’s CEO, Hugh S. Griffith, remarked: "We are pleased with this favorable disease control rate and Acelarin’s ability to achieve confirmed complete and partial responses in this very heavily pre-treated patient population. We are further encouraged by these results in light of the recent CLIO study in less heavily pre-treated patients with platinum-resistant ovarian cancer, where no patients in the chemotherapy group achieved a complete response and only one patient achieved a confirmed partial response which resulted in a confirmed overall response rate of 3%."

The CLIO study reported on the efficacy of the current chemotherapy standards of care, namely paclitaxel, pegylated liposomal doxorubicin, topotecan and gemcitabine. Thirty-three patients received chemotherapy in the CLIO trial and were significantly less heavily pre-treated than those in the PRO-105 trial, with a median of 3 prior lines of treatment. However, given differences in trial design and statistical analyses, comparisons across studies should be interpreted with caution.

Part two of the PRO-105 study was designed to then investigate the optimal dose identified in part one in an expansion cohort. In December 2019, consistent with NuCana’s previous announcement that it is prioritizing resources on its key programs of Acelarin in biliary tract cancer and NUC-3373 in colorectal cancer, the company determined not to proceed with part two of the PRO-105 study.

Mr. Griffith concluded: "The advent of PARP inhibitors has changed the ovarian cancer treatment landscape markedly in recent years resulting in a more complex regulatory pathway for single-agent therapy. In addition, we have been very encouraged by the synergy we have observed with Acelarin in combination with platinum agents, both in patients with biliary tract cancer and ovarian cancer. As such, any further development of Acelarin in patients with ovarian cancer would likely involve combining it with a platinum agent."