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Principia Biopharma Reports Fourth Quarter and Full Year 2019 Financial Results

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.

Phio Pharmaceuticals and Helmholtz Zentrum München Announce Collaboration and Option Agreement with Medigene

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.

"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."

NuCana Reports Preliminary Data from Phase II Study of Single-Agent Acelarin (NUC-1031) in Patients with Platinum-Resistant Ovarian Cancer

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.

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."

Ligand’s Presentation at the Barclays Global Healthcare Conference Now a Webcast Only, New Slides Available on Ligand.com

On March 10, 2020 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported that scheduled in-person presentation at the Barclays Global Healthcare Conference in Miami Beach was changed by conference organizers to a webcast only, as the conference has been reconfigured (Press release, Ligand, MAR 10, 2020, View Source [SID1234555367]). The webcast will take place as previously scheduled at 2:35 p.m. ET and is available here. Conference one-on-one investor meetings have been moved to telephone calls, and the new slides Ligand will be using during those meetings have been posted to www.ligand.com.

The new slides contain recent pipeline and business updates, including:

Palvella Therapeutics completed enrollment in the Phase 2/3 pivotal study of PTX-022 for treatment of pachyonychia congenita, with topline data expected in Q4. PTX-022 is a Fast Track-designated program.
Gloria Pharmaceuticals, Ligand’s partner for the OmniAb-derived anti-PD-1 antibody zimberelimab in China, announced they are filing for marketing authorization in China based on positive Phase 2 data in classical Hodgkin’s lymphoma. This program joins C-Stone’s CS1001 as another OmniAb antibody that may be approved in 2020. OmniAb-derived zimberelimab is also in development in the U.S. by partner Arcus Biosciences.
In addition to zimberelimab, Ligand’s portfolio of partnered programs includes six new products that could launch by the end of 2022 including Takeda’s pevonedistat, Retrophin’s sparsentan, Eisai’s parempanel-IV, Palvella’s PTX-022, Xi’an Xintong’s Pradefovir and C-Stone’s CS-1001. Additional partnered programs could launch in this timeframe as well.
Retrophin announced enrollment of the first 190 patients in its pivotal Phase 3 DUPLEX study of Sparsentan in Focal Segmental Glomerulosclerosis. Topline efficacy data from the 36-week proteinuria endpoint analysis of the trial are expected in the first quarter of 2021.
Nucorion Pharmaceuticals completed filing of an IND for its novel liver-targeted hepatitis B program NC0-1010, obtained FDA acceptance of the IND and is on track for initiation of a Phase 1 clinical trial in the U.S in March. This is the first clinical-stage program to leverage Ligand’s novel, internally developed LTP Technology Platform.
Ligand continues to support Captisol orders from partners related to remdesivir, an investigational product candidate being actively assessed in Phase 2 and Phase 3 clinical studies to treat COVID-19, with the first studies expected to be completed in April.
"2020 is off to an excellent start, with a good flow of news and updates from our partnered portfolio. We are facing the most substantial calendar of partner events in our history and see the potential for several major new product launches over the next couple of years," said John Higgins, Chief Executive Officer of Ligand. "Our OmniAb business is doing very well, with two antibody-based drugs filed or expected to be filed for potential approval."

"We have also been very active working with partners to meet their Captisol needs to formulate clinical supplies of the antiviral drug remdesivir, which is being studied as a potential treatment for COVID-19," he added. "Our team has made substantial progress over the past few weeks working with partners to provide supply-chain support that was not anticipated at the start of the year."

About Captisol

Captisol is a patent-protected, chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. Captisol was invented and initially developed by scientists in the laboratories of Dr. Valentino Stella, University Distinguished Professor at the University of Kansas’ Higuchi Biosciences Center for specific use in drug development and formulation. This unique technology has enabled several FDA-approved products including Amgen’s KYPROLIS, Baxter International’s NEXTERONE, Acrotech Biopharma L.L.C.’s and CASI Pharmaceuticals’ EVOMELA, Melinta Therapeutics’ BAXDELA and Sage Therapeutics’ ZULRESSO. There are many Captisol-enabled products currently in various stages of development.

Kaleido Biosciences to Present During Chardan’s 2nd Annual Microbiome Medicines Summit

On march 10, 2020 Kaleido Biosciences, Inc. (Nasdaq: KLDO), a clinical-stage healthcare company with a chemistry-driven approach to leveraging the microbiome organ to treat disease and improve human health, reported that Alison Lawton, President and Chief Executive Officer, will present during Chardan’s 2nd Annual Microbiome Medicines Summit, a virtual event, on Monday, March 16, 2020 at 4:15 p.m. ET (Press release, Kaleido Biosciences, MAR 10, 2020, View Source [SID1234555366]).

A webcast of the presentation can be accessed on the Investors & Media section of Kaleido’s website at View Source An archived replay will be available for 90 days following the event.