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 Sysmex Inostics has reported the test to provide additional key information for investigation of the validity of ctDNA for molecular monitoring and recurrence surveillance for breast cancer patients who are receiving novel therapies (Press release, Sysmex Inostics, MAR 10, 2020, View Source [SID1234555386]).

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In addition to a high detection rate even for patients with very low frequency mutations, the SafeSEQ Breast Cancer Panel has now also demonstrated the ability to delineate true changes in the level of ctDNA from random sampling or technical variation. This is in contrast to other liquid biopsy methods which report mutations detected along with the ratio of mutant molecules to wildtype molecules (mutant allele frequency, MAF), but are unable to relate changes in MAF to changes in the disease status of the patient with confidence. For instance, clinical data presented at San Antonio Breast Cancer Symposium (SABCS) demonstrated the importance of accurate detection of ctDNA with SafeSEQ testing to monitor tumor response in breast cancer patients receiving neoadjuvant treatment.

As a highly refined clinical development diagnostic, the SafeSEQ breast cancer panel has demonstrated exquisite sensitivity for detecting mutations in ESR1, which are known to arise in patients with estrogen receptor-positive breast cancer who have progressed on adjuvant hormone therapy. Extensive ESR1 ctDNA testing by Sysmex Inostics’ OncoBEAM enhanced digital PCR has shown that these mutations occur at very low frequencies in circulation. Patients who are receiving an investigational ESR1-directed therapy as part of a clinical trial may exhibit an ESR1 mutation well below 1% MAF at baseline which could then decrease further, sometimes below 0.1% MAF, several months after initiation of therapy. Using a traditional qualitative ctDNA next generation sequencing-based test would not only make detection of these low-frequency mutations difficult, but it would also not be possible to determine whether this reduction in MAF represents a true decrease in ctDNA that could reflect a clinical response. This is because the reduction in MAF may result from an increase in wildtype DNA rather than a decrease in tumor-derived DNA, and the magnitude of change in MAF may be within the range of random variation that is inherent to the assay. Quantitative ctDNA detection is also important for other clinical applications such as monitoring tumor response for patients receiving neoadjuvant treatment as illustrated by SafeSEQ data presented at the recent San Antonio Breast Cancer Symposium, Poster P6-10-05.

The updated SafeSEQ Breast Cancer Panel can overcome these challenges by determining whether the change in the absolute number of ESR1 mutant molecules detected at each timepoint, irrespective of the wildtype background, is sufficient to suggest a true biological cause such as a clinical response. This ability for true quantitative mutation detection is based on a rigorous linearity validation study and is especially important given the increasing interest in ctDNA-based monitoring and surveillance to support rapid advancements in therapies for breast cancer patients.

"Without quantitatively measuring ctDNA in a way that is specific for the patient’s disease and instead employing qualitative methods based on mutant allele frequency that have traditionally been used for tissue analysis, you’re neglecting some of the unique aspects of ctDNA testing that can make it an extremely powerful tool for clinical development," noted Matt Ryder, Sysmex’s Associate Director of Biomarkers & Companion Diagnostics. "We are confident that the SafeSEQ Breast Cancer Panel is now one of the most informative tests available to support development of novel therapies for breast cancer, and we look forward to helping our pharma partners explore the clinical validity of ctDNA-based therapeutic monitoring and recurrence surveillance."

The updated SafeSEQ Breast Cancer Panel is available now for use as a clinical trial assay in Sysmex Inostics’ Baltimore-based CLIA laboratory.

On March 10, 2020 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company focused on the development and commercialization of therapeutics for people living with kidney disease, reported financial results for the fourth quarter and full-year ended December 31, 2019 (Press release, Akebia, MAR 10, 2020, View Source [SID1234555355]). The Company will host a conference call today, Tuesday, March 10, 2020, at 9:00 a.m. Eastern Time to discuss its fourth quarter and full-year 2019 financial results and recent business highlights.

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"2019 was a year of considerable progress for Akebia and with many milestones on the horizon, 2020 is shaping up to be equally, if not more, exciting," stated John P. Butler, Chief Executive Officer of Akebia. "Our highest priority remains the successful execution of our global Phase 3 program for vadadustat, our investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI). We have a tremendous amount of confidence in our vadadustat clinical program and believe we’ve developed an exciting path forward to drive significant value for all our stakeholders. We believe our ability to potentially access a Priority Review Voucher (PRV) for the vadadustat New Drug Application (NDA) to expedite FDA review would meaningfully enhance the potential of bringing vadadustat to patients as quickly as possible, subject to regulatory approval."

Butler continued, "We look forward to sharing data from our global Phase 3 studies of vadadustat, starting with top-line data from INNO2VATE on track for the second quarter of 2020 followed by PRO2TECT in mid-2020. We expect vadadustat to be the first drug of the HIF-PHI class to deliver clear data that directly compare its outcomes to the current standard of care in dialysis-dependent and non-dialysis dependent patients for the treatment of anemia due to chronic kidney disease (CKD)."

Agreement with Vifor Pharma regarding a Priority Review Voucher

In February 2020, Akebia entered into a letter agreement with Vifor (International) Ltd. (Vifor Pharma) relating to Vifor Pharma’s agreement with a third party to purchase a PRV issued by the U.S. Food and Drug Administration (FDA), subject to satisfaction of customary closing conditions. Akebia will pay Vifor Pharma $10.0 million following the closing of the PRV purchase. In exchange, Vifor Pharma is obligated to reserve the PRV for the vadadustat NDA for the treatment of anemia due to CKD in dialysis-dependent and non-dialysis dependent patients until Akebia and Vifor Pharma agree on the financial and other terms under which it will assign the PRV to Akebia or make a mutual decision to sell the PRV. A PRV entitles the holder to priority review of an NDA or a Biologics License Application for a new drug, which reduces the target FDA review time to six months after official acceptance of the submission and could lead to expedited approval.

Jason A. Amello, Chief Financial Officer of Akebia stated, "We are pleased to have extended our cash runway well into 2021 through planned, disciplined spending and the identification of operating efficiencies, coupled with the sales of common stock via our At-the-Market facility over the last few months." The Company’s cash runway, consistent with previous commentary, includes the receipt of a $15.0 million regulatory milestone from Mitsubishi Tanabe Pharma Corporation, Akebia’s development and commercialization collaboration partner in Japan for vadadustat, assuming approval of vadadustat in Japan.

Fourth Quarter and Full-Year 2019 Financial Results

Cash Position: Cash, cash equivalents and available-for-sale securities as of December 31, 2019 were $147.7 million.

Revenues: Total revenue was $69.6 million for the fourth quarter of 2019 compared to $59.9 million for the fourth quarter of 2018(1), and $335.0 million for the full-year 2019 compared to $207.7 million for the full-year 2018(1).

Collaboration revenue was $40.6 million for the fourth quarter of 2019 compared to $53.0 million in the fourth quarter of 2018, and $223.9 million for the full-year 2019 compared with $200.9 million for the full-year 2018. The change in both periods is due to the timing in which vadadustat development expenses are incurred and the associated revenue is recognized on a percentage-of-completion basis.

Net product revenue was $28.9 million for the fourth quarter of 2019 compared with $6.8 million in the fourth quarter of 2018(1), and $111.1 million for the full-year 2019 compared to $6.8 million for the full-year 2018(1). Pro forma net product revenue for the full-year 2018, inclusive of pre-merger net product revenue recorded by Keryx Biopharmaceuticals, Inc. (Keryx), was approximately $96 million.

COGS: Cost of goods sold was $38.1 million for the fourth quarter of 2019, which includes non-cash charges, related to the application of purchase accounting as a result of the merger with Keryx, of $18.8 million for inventory step-up and $9.1 million for amortization of intangibles. Cost of goods sold was $145.3 million for the full-year 2019, which includes non-cash charges of $70.4 million for inventory step-up and $36.4 million for amortization of intangibles.

R&D Expenses: Research and development expenses were $80.4 million for the fourth quarter of 2019 compared to $87.1 million for the fourth quarter of 2018(1), and $323.0 million for the full-year 2019 compared to $291.0 million for the full-year 2018(1). The change in both periods was largely attributable to a change in costs associated with our research and development programs, including vadadustat.

SG&A Expenses: Selling, general and administrative expenses were $44.9 million for the fourth quarter of 2019 compared to $55.1 million for the fourth quarter of 2018(1) (which included $41.7 million of merger-related expenses), and $149.5 million for the full-year 2019 compared to $87.1 million for the full-year 2018(1) (which included $49.5 million of merger-related expenses).

Net Loss: Net loss was $94.5 million for the fourth quarter of 2019 compared to $60.1 million for the fourth quarter of 2018(1), and $279.7 million for the full-year 2019 compared to $143.6 million for the full-year 2018(1).

Includes only 18 days of operating results of Keryx following completion of Akebia’s merger with Keryx on December 12, 2018, whereby Keryx became Akebia’s wholly owned subsidiary.

Conference Call

Akebia will host a conference call at 9:00 a.m. Eastern Time today, Tuesday, March 10th, to discuss its fourth quarter and full-year 2019 financial results and recent business highlights. To listen to the conference call, please dial (877) 458-0977 (domestic) or (484) 653-6724 (international) using conference ID number 6572299. The call will also be webcast LIVE and can be accessed via the Investors section of the Company’s website at View Source

A replay of the conference call will be available two hours after the completion of the call through March 16, 2020. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference conference ID number 6572299. An online archive of the conference call can be accessed via the Investors section of the Company’s website at View Source

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 Zimmer Biomet Holdings, Inc. (NYSE and SIX: ZBH), a global leader in musculoskeletal healthcare, reported that its Board of Directors has approved the payment of a quarterly cash dividend to stockholders for the first quarter of 2020 (Press release, Zimmer Holdings, MAR 10, 2020, View Source [SID1234555387]).

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The cash dividend of $0.24 per share is payable on April 30, 2020 to stockholders of record as of the close of business on March 27, 2020.