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 Syndivia, a research-driven biotechnology company focused on the development of new therapeutic modalities for cancers, reported that it will benefit from €2 m deep tech financing from Bpifrance (Press release, Syndivia, MAR 10, 2020, View Source [SID1234555385]). Proceeds will be used to further fund the advancement of one company’s drug candidate towards the clinic.

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The Deeptech plan was initiated by the French government and has been deployed by Bpifrance since 2019 to support deep tech startups resulting from research based on breakthrough innovations. The main objective is to allow these companies to grow, and to stimulate innovation by strengthening the links between academia and the entrepreneurial world.

Sasha Koniev, CEO of Syndivia, said, "We are very grateful to Bpifrance for this generous support. This financing will largely accelerate the development of our lead immunoconjugate for various cancer indications, which to date have a very limited number of effective treatment options."

Commenting on this news, Alban Stamm, in charge of innovation at Bpifrance, said, "Syndivia, which initially emerged from the research of academic laboratories supported by SATT Conectus and reinforced with iLab 2014 (emergence) and 2015 (development) grants, succeeded in bringing together all the ingredients to meet technological challenges. At Bpifrance, we appreciate Syndivia’s progress to date and especially value the team: initially composed of young researchers with strong entrepreneurial capacity, it surrounded itself with an experienced board bringing together all the key success factors of a biotech startup."

About Bpifrance

Bpifrance is the French national investment bank. It finances businesses – at every stage of their development – through loans, guarantees, equity investments and export insurances. Bpifrance also provides extra financial services such as training and consultancy to help entrepreneurs meet their challenges.

On March 10, 2020 Trillium Therapeutics Inc. (NASDAQ/TSX: TRIL), a clinical stage immuno-oncology company developing innovative therapies for the treatment of cancer, reported financial and operating results, including an update on its transformation program, for the year ended December 31, 2019 (Press release, Trillium Therapeutics, MAR 10, 2020, View Source [SID1234555348]).

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2019 Transformation Program

2019 was a critical year in Trillium’s evolution toward a more clinical development-focused CD47 immuno-oncology company. Key developments, as part of a wide-ranging transformation program, were:

Transitioned leadership to a new CEO, Jan Skvarka, who joined in September. Mr. Skvarka is an experienced healthcare executive, who was previously CEO of a PureTech Health portfolio company (Tal Medical), and leading Partner in the Healthcare practice at Bain & Company. He holds an MBA from Harvard Business School.
Established office in Cambridge, MA, which will allow the company to tap the local talent pool. The CEO office and clinical development are now based in Cambridge.
Restructured the company’s footprint by reducing staff by 40% to create a more efficient organization with stronger clinical development focus, as well as extend the cash runway.
Substantially revised the company strategy, namely:
Refocused on intravenous TTI-621 & TTI-622 programs and large hematologic malignancy indications, specifically acute myeloid lymphoma & myelodysplastic syndromes (AML/MDS), peripheral T-cell lymphoma (PTCL), diffuse large B-cell lymphoma (DLBCL) and multiple myeloma;
Deprioritized a lead intratumoral TTI-621 program with intended focus on early-stage cutaneous T-cell lymphoma (CTCL).
Zeroed in on execution of TTI-621 & TTI-622 dose escalation studies, which were declared mission critical path in the near term.
Subsequently, on January 7, 2020, the company announced a data update that confirmed TTI-621 monotherapy activity at initial low dose escalation levels (up to 0.5 mg/kg) across several hematologic malignancies. As such, TTI-621 is the only CD47 blocker that has shown meaningful single agent activity. Further dose optimization is in progress, and TTI-621 is now enrolling patients at a 1.4 mg/kg dose level, or 7 times the initially selected dose at which monotherapy activity was observed.

Following the (i) transformation program, (ii) changes in strategic direction, and (iii) encouraging data update, the company raised $117 million in January 2020, in a substantially oversubscribed public offering. Among key investors were experienced healthcare funds, such as Boxer Capital, Logos Capital, New Enterprise Associates, Venrock Healthcare Capital Partners and Vivo Capital. Paul Walker joined the Board of Directors, and Ali Behbahani became Board Observer; both are General Partners at NEA.

Cash Position and Guidance

As of February 28, the company had approximately $130 million in cash and cash equivalents.

Over the next three years (2020-22), the company plans to accomplish the following goals:

Complete TTI-621 and TTI-622 dose escalation studies, expected in 2020 (potentially in 2021, depending on the dose selected);
Conduct 3-4 new hematologic malignancy studies in AML/MDS, PTCL, DLBCL and/or multiple myeloma; and
Initiate and complete a solid tumor exploratory effort with TTI-621 in several key indications.
In 2020, the company will provide the following updates:

TTI-622 study update at the 2020 ASCO (Free ASCO Whitepaper) annual meeting;
TTI-621 study update mid-year and at the 2020 ASH (Free ASH Whitepaper) annual meeting;
TTI-621 and TTI-622 study updates once maximum tolerated doses or recommended phase 2 doses are identified.
Annual 2019 Financial Results:

As of December 31, 2019, Trillium had cash and cash equivalents and marketable securities, and working capital of $22.7 million and $9.8 million, respectively, compared to $33.4 million and $25.1 million, respectively at December 31, 2018. The decrease in cash and cash equivalents and marketable securities, and the decrease in working capital were due mainly to cash used in operations, partially offset by the cash received from the February 2019 public offering. The decrease in working capital was due mainly to cash used in operations and an increase to accounts payable and accrued liabilities due to timing of clinical trial related payments.

-2-

Net loss for the year ended December 31, 2019 of $41.6 million was higher than the loss of $32.9 million for the year ended December 31, 2018. The net loss was higher due mainly to a warrant liability revaluation loss of $5.7 million, the write down of Fluorinov intangible assets of $3.0 million, higher manufacturing costs, and a net foreign currency loss of $0.8 million in the current year compared to a net foreign currency gain of $2.7 million in the prior year. The higher loss was partially offset by lower clinical trial expenses.

In January 2020, the Company completed an underwritten public offering for gross proceeds of $117 million comprising 41,279,090 common shares and 1,250,000 Series II Non-Voting Convertible First Preferred Shares, each issued at $2.75 per share.

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.