On March 27, 2019 Phio Pharmaceuticals Corp. (NASDAQ: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (sd-rxRNA) therapeutic platform, reported its financial results for the fourth quarter and year ended December 31, 2018 and provided a business update (Press release, Phio Pharmaceuticals, MAR 27, 2019, View Source [SID1234534681]).

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"One of the Company’s major accomplishments during the year was the successful transition to focus on developing novel immuno-oncology treatments, a strategic decision made in early 2018," said Dr. Gerrit Dispersyn, President and CEO of Phio Pharmaceuticals. "Based on our internal R&D, and together with our corporate and academic partners, we have been demonstrating the potential of using our self-delivering RNAi platform in immuno-oncology applications. Our results to date suggest that we can improve existing and developing forms of adoptive cell therapies (ACT) based on T-cells and on NK cells. In addition, through direct intratumoral use of self-delivering RNAi compounds, we showed that we have the potential to reprogram the tumor micro-environment. With this latter approach, we hope to lower the barriers that hamper ACT success in solid tumors, a significant clinical unmet need. Based on these successes, we now have three well defined immuno-oncology R&D pipeline programs that may provide compelling alternatives to other therapeutic approaches such as antibody therapy and/or genetic engineering. Our progress laid the groundwork for us to successfully complete an equity offering in October, providing us the capital to confidently execute our R&D pipeline programs towards clinical development and further partnerships."

Year in Review and Recent Corporate Updates

On March 1, 2019, Gerrit Dispersyn, Dr. Med. Sc. became the Company’s President and Chief Executive Officer. Dr. Dispersyn succeeded Geert Cauwenbergh, Dr. Med. Sc., who retired as CEO of the Company and remains as a member of the Company’s Board of Directors.
Effective November 19, 2018, the Company changed its corporate name from RXi Pharmaceuticals Corporation to Phio Pharmaceuticals Corp. This change reflects the Company’s transition from a platform company in dermatology and ophthalmology to one that is focused on developing groundbreaking immuno-oncology therapeutics.
Research conducted using the Company’s self-delivering RNAi platform in the field of immunotherapy to treat cancer was published in a leading peer-reviewed journal, Molecular Therapy. The published paper is titled, "Self-Delivering RNAi (sd-rxRNA) Targeting PD-1 using Adoptive Cell Therapy Approach for the Treatment of Malignant Melanoma."
Presented new data on NK cells at the 16th Annual Discovery on Target Conference and the 33rd Annual Meeting & Pre-Conference Programs of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper).
Data demonstrated potent silencing activity as well as phenotypic effect of NK cells treated with sd-rxRNA compounds targeting checkpoints such as Cbl-b and TIGIT. By treating NK cells ex-vivo with sd-rxRNA compounds the anti-tumor response of these cells can be improved.
Entered into collaborations with industry and academic leaders in immuno-oncology:
The Karolinska Institutet in Sweden is exploring the use of our sd-rxRNA compounds against targets involved in T-cell and NK cell differentiation and/or in the immune cell tumor-induced stress response with the aim of producing anti-tumor adoptive cell therapy grafts with improved functionality and persistence.
Iovance Biotherapeutics, Inc. is evaluating the potential synergies of our novel sd-rxRNA therapeutic compounds with their autologous cell therapy based on tumor-infiltrating lymphocytes for the use in the treatment of cancer.
Completed and reported positive results from the Company’s clinical trials in dermatology and ophthalmology:
Study RXI-109-1501, a Phase 1/2 clinical trial for retinal scarring in subjects with wet age-related macular degeneration with evidence of subretinal fibrosis, successfully met its primary objective as shown by the absence of dose-limiting and serious toxicities. The secondary objective of the study was also met with improved or stable disease in the study eyes of several subjects.
Study RXI-SCP-1502, a Phase 2 clinical trial with Samcyprone for the treatment of cutaneous warts, successfully met its primary effectiveness objectives as shown by high levels of immunotherapeutic and therapeutic response. The immunotherapeutic response rate was 97.7% across all enrolled subjects and from a therapeutic response viewpoint, more than 70% of all warts showed a positive wart response rate. The study results showed furthermore that Samcyprone was safe and well tolerated.
Successfully completed equity offerings in April and October 2018 for total net proceeds of $17.4 million, which the Company expects to provide ample funding for operations into the second half of 2020.
Select Financial Results

Cash Position

At December 31, 2018, the Company had cash of $14.9 million as compared with $3.6 million at December 31, 2017.

Revenues

Revenues for the year ended December 31, 2018 were $138,000, as compared with $15,000 for the year ended December 31, 2017. Revenues for the years ended 2018 and 2017 related to the work performed by the Company as a sub-awardee under the government grant awarded to BioAxone Biosciences, Inc. for the development of a novel sd-rxRNA compound, BA-434, that targets PTEN for the treatment of spinal cord injury.

Research and Development Expenses

Research and development expenses for the year ended December 31, 2018 were $4.3 million as compared with $5.4 million for the year ended December 31, 2017. The decrease was primarily due to the completion of the work in the Company’s dermatology and ophthalmology programs, including clinical trial-related and manufacturing-related expenses, and a decrease in payroll expenses due to a reduction in headcount as compared with the prior year period.

Acquired In-process Research and Development Expenses

The Company recorded acquired in-process research and development expense of $4.7 million during the year ended December 31, 2017. The expense related to the fair value of consideration given, which includes transaction costs, liabilities assumed and cancellation of notes receivable, and the deferred tax impact of the Company’s acquisition of MirImmune. The Company had no such expense during the year ended December 31, 2018.

General and Administrative Expenses

General and administrative expenses for the year ended December 31, 2018 were $3.2 million as compared with $4.0 million for the year ended December 31, 2017. The decrease was primarily due to a decrease in payroll expenses due to a reduction in headcount as compared with the prior year period, as well as a decrease in professional fees for legal-related services.

Income Tax

The Company had no income tax benefit or expense for the year ended December 31, 2018. The Company recognized an income tax benefit of $1.6 million for the year ended December 31, 2017 due to the tax-related impact of the Company’s acquisition of MirImmune.

Net Loss

Net loss for the year ended December 31, 2018 was $7.4 million, or $1.04 per share, compared with $12.5 million, or $5.52 per share, for the year ended December 31, 2017. The decrease was primarily due to a decrease in acquired in-process research and development expense and the changes in operating expenses, as discussed above.

On March 27, 2019 THERADIAG (ISIN: FR0004197747, Ticker: ALTER), a company specialized in in vitro diagnostics and theranostics, reported its consolidated full-year results for the financial year ended on December 31, 2018 approved by the Board of Directors on March 26, 2019 (Press release, Theradiag, MAR 27, 2019, View Source;utm_medium=rss&utm_campaign=theradiag-reports-its-2018-full-year-results [SID1234534665]).

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"The healthy increase in Lisa Tracker kit sales (up 17%) and the measures we took to reorganize the Company in late 2017 paid off in 2018, generating a major improvement in Theradiag’s performance. Before non-recurring items, we nearly broke even in 2018, with a net loss of €0.4 million. The brisk expansion in the Tracker business augurs very well for the future, and so the outlook for 2019 is bright. Innovation remains a top priority to support our growth, and we continue to hold discussions with pharmaceutical companies concerning further deals, including in the United States", commented Bertrand de Castelnau, Theradiag’s Chief Executive Officer.

2018 revenue
Theradiag’s consolidated revenue came to €8.9 million in FY 2018, down from €9.0 million in FY 2017 as a result of the contraction in theranostics revenue reflecting the fact that no non-recurring theranostics orders were logged in the first half of 2018. In contrast, a high level of activity was recorded in the same period of 2017 after Theradiag entered into several agreements with pharmaceutical companies.

Overall, 96% of theranostics revenue came from sales of kits for routine use. A steady increase over the past three quarters paved the way for an overall rise of 17% in FY 2018 sales compared to FY 2017.

IVD revenue remained stable, edging 1% higher.

Export sales of theranostics kits for routine use advanced by 19%.

Reduction in operating expenses, driving a 78% increase in operating income and almost eliminating the net loss before non-recurring items
The restructuring decisions made in late 2017 (shutdown of Prestizia’s operations and reorganization of the teams) and the favourable evolution of the product mix delivered benefits in FY 2018, including a 17% reduction in operating expenses.

As a result, the operating loss decreased by 78% from €2,515,000 in FY 2017 to €563,000 in FY 2018 and the net loss was cut from €5,959,000 to €787,000.

Before non-recurring items, Theradiag’s bottom line came close to breakeven in FY 2018, with a net loss of €372,000.

The FY 2018 net loss was adversely affected by a €415,000 restructuring charge.

Healthy cash position
At December 31, 2018, Theradiag’s available net cash stood at €3.43 million, compared to €5.16 million at December 31, 2017. Tight cost management helped to reduce Theradiag’s annual cash burn by one third.

"The company’s recovery is underway and its profitability is on track, with a sufficient cash position, allowing the Company to invest in its development." added Pierre Morgon, Chairman of the Board of Directors of Theradiag.

Highlights of 2018

Termination of the commercial agreements with HOB Biotech
As a result of HOB Biotech’s failure to comply with the contractual terms of the 2015 agreements between the two companies, Theradiag was unable to distribute the agreed products in Europe or to market its reagents in China. Legal proceedings were launched against HOB Biotech to remedy the loss suffered by Theradiag.

Partnership established with Biogaran
Theradiag entered into a partnership agreement with pharma group Biogaran to supply its Lisa Tracker kits with Biogaran’s biosimilar drugs. Theradiag’s monitoring kits are thus listed in France by Biogaran to support the biosimilar drugs it supplies. Theradiag will take responsibility for providing training to laboratories in how to use kits and follow up on clinician requests concerning monitoring.

Keen interest from the scientific community in biotherapy monitoring evident at the ECCO Congress
At the 13th edition of the ECCO Congress, over 90 publications presented biotherapy monitoring data. Of these, around 20 included results obtained using Lisa Tracker kits, confirming the scientific community’s keen interest in biotherapy monitoring. The increasing volume of publications also reflects the more widespread use of monitoring kits in what is also a growing number of centers.

Changes in Theradiag’s governance
Pierre Morgon succeeded Gérard Tobelem at Chairman of the Board of Directors.

Bertrand de Castelnau replaced Michel Finance as Chief Executive Officer and was also co-opted as a director replacing Dominique Costantini.

Bertrand de Castelnau, 58, has over 25 years’ experience in diagnostics. He began his career as a commercial attaché in Islamabad, Pakistan. Subsequently, he joined Roche in Basle, Switzerland as a general auditor and was then offered a position in the Diagnostics division before being handed responsibility for Roche Diagnostics’ Asia-Pacific region (based in Singapore). Next Bertrand took charge of Guerbet’s operations for four years, then ran the Horiba ABX group and the Horiba Medical segment for ten years, before joining DiaSys as head of sales and marketing and Chief Financial Officer.

The Board of Directors now has the following members:

Pierre Morgon, Chairman of the Board of Directors
Sylvie Bratel, Independent director
Bertrand de Castelnau, Director
Vincent Fert, Director
John Li, Director
Dominique Takizawa, Independent director
Subsequent events

Award of the 12th CE mark to the Lisa Tracker range
Theradiag has been awarded a CE mark for the Cosentyx (secukinumab) monitoring kit used in the treatment of psoriasis, psoriatic arthritis and ankylosing spondylitis, reaffirming its leadership position in biotherapy monitoring, with the most extensive line available in the market.

FDA inspection
During February, Theradiag was inspected by the FDA. It was not informed of any non-compliance issues and did not receive any comments (no Form 483 observations).

Next financial press release

Interim 2019 revenue on Thursday, July 25, 2019, after market close

On March 27, 2019 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported participation in the Jefferies 6th Annual IO Cell Therapy Summit taking place in Boston (Press release, Bellicum Pharmaceuticals, MAR 27, 2019, View Source [SID1234534667]). Bellicum’s President & CEO Rick Fair and Head of Research, Aaron Foster, Ph.D., will provide an overview of the company and its development programs during a fireside chat on April 5, 2019 at 9:10 a.m. EDT.

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On March 27, 2019 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported plans to expedite development of avapritinib, BLU-667 and BLU-782 based on recent clinical progress and interactions with regulatory authorities (Press release, Blueprint Medicines, MAR 27, 2019, View Source [SID1234534710]).

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"The updates we are announcing today for avapritinib, BLU-667 and BLU-782 represent the acceleration of multiple clinical-stage programs, including planned marketing applications for avapritinib and BLU-667, and highlight our commitment to executing a portfolio-based strategy," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "In addition, as we prepare to submit marketing applications for avapritinib for the treatment of PDGFRA-driven and fourth-line GIST in the United States in the second quarter and Europe in the third quarter, we are rapidly building global commercial capabilities to enable us to deliver this important new medicine, if approved, to patients in need."

Summary of Accelerated Regulatory Submission Plans and Recent Clinical Progress

Avapritinib: Gastrointestinal Stromal Tumors (GIST)

Blueprint Medicines now plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for avapritinib for the treatment of both PDGFRα D842V mutant GIST and fourth-line GIST in the third quarter of 2019.
Avapritinib: Advanced Systemic Mastocytosis (SM)

Blueprint Medicines now plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for avapritinib for the treatment of advanced SM in the first quarter of 2020, subject to continuing discussions with the FDA under its breakthrough therapy program to determine the required clinical data for an NDA submission.
BLU-667: RET-Altered Cancers

Top-line interim data from the Phase 1 ARROW clinical trial of BLU-667 for patients with RET-fusion non-small cell lung cancer (NSCLC) and RET-mutant medullary thyroid cancer (MTC) who were response evaluable and treated at the recommended Phase 2 dose of 400 mg once daily, as of November 14, 2018 with follow-up through a data cutoff date of March 1, 2019, showed the following:
In 34 patients with RET-fusion NSCLC previously treated with platinum-based chemotherapy, the overall response rate (ORR) was 62 percent.(1)
In 16 patients with RET-mutant MTC previously treated with the approved multi-kinase inhibitors cabozantinib and/or vandetanib, the ORR was 63 percent.(2)
BLU-667 was generally well-tolerated, and most adverse events (AEs) reported by investigators were mild or moderate (Grade 1 or 2). Across all doses and treatment cohorts (n=217), only eight patients discontinued treatment with BLU-667 due to treatment-related AEs, with one Grade 5 AE (pneumonia/lung infection) determined by the investigator to be possibly related to BLU-667.
Detailed clinical safety and efficacy data from the ARROW trial in RET-fusion NSCLC patients and RET-mutant MTC patients have been submitted for presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2019.
The enrollment target has been reached and patient screening is now closed for the registration-enabling ARROW trial cohort for patients with previously treated RET-fusion NSCLC. Blueprint Medicines anticipates reaching the enrollment target for the registration-enabling ARROW trial cohort for patients with previously treated MTC in the second quarter of 2019.
Based on the early achievement of the enrollment target for the RET-fusion NSCLC cohort, Blueprint Medicines now plans to submit an NDA to the FDA for BLU-667 for the treatment of patients with NSCLC previously treated with platinum-based chemotherapy in the first quarter of 2020. Blueprint Medicines continues to expect to submit an NDA to the FDA for BLU-667 for the treatment of patients with RET-mutant MTC previously treated with an approved multi-kinase inhibitor in the first half of 2020.
BLU-782: Fibrodysplasia Ossificans Progressiva (FOP)

Based on the progress of the ongoing Phase 1 clinical trial in healthy volunteers and input from clinical experts, Blueprint Medicines now plans to initiate a Phase 2a clinical trial of BLU-782 in patients with FOP in the fourth quarter of 2019.
Footnotes:
(1) 95 percent confidence interval (CI): 44-78 percent; five responses pending confirmation.
(2) 95 percent CI: 35-85 percent; three responses pending confirmation.

On March 27, 2019 Syndax Pharmaceuticals, Inc. ("Syndax" or the "Company") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that it has entered into an agreement with Biotechnology Value Fund, L.P. ("BVF") and other leading life sciences investors for the purchase of 4,366,666 shares of common stock at a purchase price of $6.00 per share, representing a premium of 30% to the share price as of market close on Tuesday, March 26, as well as warrants to purchase up to 2,183,331 shares of common stock at an exercise price of $12.00 per share, and warrants to purchase up to 2,183,335 shares of common stock at an exercise price of $18.00 per share (Press release, Syndax, MAR 27, 2019, View Source [SID1234534668]).

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Syndax anticipates aggregate gross proceeds from the offering will be approximately $26.2 million (excluding any proceeds to be received upon exercise of the warrants). Closing of the transaction is expected to occur on or about March 29, 2019.

The securities described above are being offered by Syndax pursuant to a shelf registration statement previously filed with the Securities and Exchange Commission (the "SEC"), which the SEC declared effective on April 20, 2017. A final prospectus supplement related to the offering will be filed with the SEC, and will be available on the SEC’s website located at View Source

This press release does not constitute an offer to sell or the solicitation of an offer to buy the securities, nor shall there be any sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state. Any offering of the securities under the resale registration statement will only be by means of a prospectus.