On September 27, 2018 CytoSen Therapeutics, a private biopharmaceutical company accelerating innovation in Natural Killer (NK) cell therapy, reported it has received a favorable response from the U.S. Food and Drug Administration ("FDA") to its pre-Investigational New Drug ("pre-IND") meeting package for the proposed Phase 2 trial of CytoSen’s adoptive NK cell therapy, CSTD002-NK, for the reduction of relapse in high risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) in patients undergoing haploidentical hematopoietic stem cell transplantation (HSCT) (Press release, CytoSen Therapeutics, SEP 27, 2018, View Source [SID1234529634]). The FDA agreed that the Company’s pre-clinical studies, in combination with results from the clinical trial conducted at The University of Texas MD Anderson Cancer Center adequately support the Company’s proposed Phase 2 clinical trial. The FDA also deemed the proposed dose regimen, safety assessment and study endpoints as appropriate, and confirmed that interim results from the clinical study could be considered for a Regenerative Medicine Advanced Therapy (RMAT) designation.

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"We are pleased with the feedback and clarification we received from the FDA, which reinforces our clinical development strategy and serves as a critical next step to initiate our planned AML-transplant study," said Trent Carrier, Ph.D., CEO of CytoSen. "We look forward to commencing the trial in the first half of 2019."

CSTD002-NK is a high dose, adoptive haploidentical NK cell therapy expanded ex vivo using CytoSen’s patented nanoparticle platform. The proposed Phase 2 trial is CytoSen’s first clinical study utilizing the nanoparticle technology, which produces highly activated NK cells within a scalable manufacturing platform.

About CytoSen’s NK Cell Platform

Natural Killer cells play a significant role in the body’s innate immune response, circulating through the body to rapidly recognize and kill cancerous cells. CytoSen’s nanoparticle processing technology enables improved and sustained growth of high-dose NK cells that have potent anti-cancer cytotoxicity as well as increased expansion and activation capabilities. CytoSen’s lead clinical product, CSTD002-NK, is poised to initiate Phase 2 trial enrollment in the U.S. in the first half of 2019 as a best-in-class NK cell therapy for the treatment of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) in HSCT patients.

CytoSen’s technology platform is based on the work of Dean Lee, M.D., Ph.D., Co-Founder of CytoSen and Chair of CytoSen’s Scientific Advisory Board and Director of the Cellular Therapy and Cancer Immunotherapy Program for Nationwide Children’s Hospital’s Division of Hematology/Oncology/BMT and Center for Childhood Cancer and Blood Diseases, and Stefan Ciurea, M.D., Associate Professor, Department of Stem Cell Transplantation, MD Anderson Cancer Center. Data from Phase I/II clinical trials at MD Anderson demonstrated compelling results in treating AML, providing validation for CytoSen’s strategic clinical development program.

On September 27, 2018 Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a Fortress Biotech (NASDAQ: FBIO) company, reported that James F. Oliviero, President and Chief Executive Officer of Checkpoint, will present a company overview at the Ladenburg Thalmann 2018 Healthcare Conference on Tuesday, October 2, 2018, at 2 p.m. EDT (Press release, Checkpoint Therapeutics, SEP 27, 2018, View Source [SID1234529734]). The conference will be held at the Sofitel New York in New York City.

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A live webcast of the presentation will be available on the Events page of the Investors & Media section of Checkpoint’s website: www.checkpointtx.com.

On September 27, 2018 Akari Therapeutics, Plc (NASDAQ:AKTX), a biopharmaceutical company focused on the development and commercialization of innovative therapeutics to treat orphan autoimmune and inflammatory diseases where complement and or leukotriene systems are implicated, reported its financial results for the second quarter ended June 30, 2018 (Press release, Akari Therapeutics, SEP 27, 2018, View Source [SID1234529898]).

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"We made several important advancements since the beginning of 2018 and now have a diversified clinical portfolio with four ongoing disease programs and up to a $20 million funding facility provided by Aspire Capital Fund, LLC," commented Clive Richardson, Interim Chief Executive Officer of Akari Therapeutics.

Clinical development highlights and upcoming milestones

Coversin clinical trials focused on orphan diseases mediated by both the complement and leukotriene pathways:
Phase II trial in patients with bullous pemphigoid, a severe blistering skin disease, with initial data expected in the first quarter of 2019
Phase I/II trial in patients with atopic keratoconjunctivitis, a sight threatening surface of the eye condition, with initial data anticipated in the first quarter of 2019
Coversin clinical trials in orphan diseases in which complement dysregulation is the primary disease driver:
Phase III trial in naïve patients with paroxysmal nocturnal haemoglobinuria (PNH) and a Phase II trial in patients with PNH who are resistant to eculizumab
Phase II trial in atypical haemolytic syndrome, a severe thrombotic microangiopathy
All patients who completed phase 2 have opted to enroll into the Coversin long term Phase III safety study. Currently there is over 15 years of safety data on Coversin with no drug related severe adverse events to date.
Second Quarter 2018 Financial Results and Business Highlights

Operating expenses, which include research and development (R&D) expenses and general and administrative (G&A) expenses, were $8.0 million in the second quarter of 2018, as compared to $7.3 million in the same quarter the prior year. Operating expenses for the six months ended June 30, 2018 were $12.3 million, as compared to $15.6 million for the same period the prior year.
R&D expenses in the second quarter of 2018 were $5.1 million, as compared to $3.8 million in the same quarter the prior year. The increase was due primarily to higher manufacturing costs for Coversin as the Company manufactured clinical trial material for supply through 2019.
G&A expenses in the second quarter of 2018 were $2.9 million, as compared to $3.6 million in the same quarter last year. This decrease was due primarily to lower legal fees, partially offset by higher rent expense.
Total other expense for the second quarter of 2018 was $43,000, as compared to total other income of $7.0 million in the second quarter of 2018. This change was primarily attributed to a $7.0 million gain in fair value of the stock option and warrant liabilities in the second quarter of 2017, compared to a $153,000 loss in the second quarter of 2018.

Net loss for the second quarter of 2018 was $8.0 million, compared to a net loss of $0.3 million for the same period in 2017. This year over year increase in net loss was due primarily to the aforementioned $7.0 million gain in fair value of the stock option and warrant liabilities in the second quarter of 2017.
The Company has entered into a Securities Purchase Agreement of up to $20 million with Aspire Capital Fund, LLC ("Aspire Capital"). Under the terms of this agreement, Aspire Capital has committed to purchase up to $20 million of Akari’s American Depositary Shares (ADSs) at Akari’s request from time to time during a 30-month period beginning on the effective date of a registration statement related to the transaction and at prices based on the market price at the time of each sale. As consideration for Aspire Capital’s obligation under the Agreement, Akari issued 30,000,000 ordinary shares at $0.02 per ordinary share (equivalent to $2.00 per ADS) to Aspire Capital as a commitment fee and sold to Aspire Capital 25,000,000 ordinary shares at $0.02 per share (equivalent to $2.00 per ADS).
As of June 30, 2018, the Company had cash of $15.1 million, as compared to cash of $28.1 million as of December 31, 2017

On September 26, 2018 ImmuPharma PLC (LSE:IMM), ("ImmuPharma" or the "Company"), the specialist drug discovery and development company, reported its interim results for the six months ended 30 June 2018 (the "Period") (Press release, ImmuPharma, SEP 26, 2018, View Source [SID1234529587]).

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Key Highlights

Lupuzor

Lupuzor demonstrated a superior response rate over placebo (52.5% vs 44.6% "responders") in the primary analysis on the Full Analysis Set of all 202 patients. However, due to the high response rate in the placebo group, this superior response did not allow statistical significance to be reached (p = 0.2631) and the trial’s primary end point was not met.

Across the whole study population, in those patients who had anti-dsDNA autoantibodies, LupuzorTM demonstrated a superior response rate over placebo (61.5% vs 47.3%, p = 0.0967). Although these results were not statistically significant, further data analysis demonstrated that in the Europe cohort (130 patients) LupuzorTM plus standard of care showed statistically significant reductions in disease activity compared to placebo plus standard of care in 79 patients who were anti-dsDNA autoantibody positive (71.1% vs 48.8%, p = 0.0218).
The study confirmed the outstanding safety profile of LupuzorTM, with no serious adverse events reported.
As announced on 7 September 2018, agreement signed with a specialist provider to enter LupuzorTM into a ‘Managed Access Programme’
Open label extension study completes recruitment
Other programs

Nucant (cancer) program – Clinical Development Collaboration with Incanthera Limited Peptide platform program / Ureka subsidiary – ImmuPharma to begin divestment process Financial Position £10 million fundraising (before expenses) successfully completed in January 2018 Stable financial performance over the Period, in line with market expectations

– Net assets of £9.9 million (31 December 2017: £3.6 million).

– Loss for the period of £4.1 million (H1 2017: £3.0 million)

Research and Development expenses of £2.5 million (H1 2017: £2.3 million)
– Basic and diluted loss per share of 2.94p (H1 2017: 2.34p)

Appointment of new joint brokers

Stanford Capital Partners and SI Capital appointed as joint brokers, working in conjunction with current NOMAD and broker, Northland Capital Partners
Commenting on the Interims and outlook Tim McCarthy, Chairman, said:

"The Board is pleased to announce the interim results for the six months ended 30 June 2018. It has been a busy period for the Board following the announcement of the Phase III trial results for Lupuzor in April 2018. We remain focused on delivering a business strategy which provides the optimum route forward for ImmuPharma and its shareholders, based on its current assets, resources and knowhow. We were obviously disappointed with the outcome of the Phase III trial results but are excited to be progressing the Managed Access Programme with a new strategic partner, which allows lupus patients early access to LupuzorTM. In the medium term, we remain focussed on achieving the full regulatory approval of LupuzorTM which we believe has the potential to be a ground breaking drug for lupus patients with blockbuster potential in commercial terms.

Our Nucant programme and Ureka subsidiary have been part of our portfolio for a number of years. We are equally excited by the potential of both. We believe the strategy we announced earlier this month, together with a robust financial position, will create enhanced value for shareholders going forward."

This announcement contains inside information for the purposes of Article 7 of Regulation (EU) 596/2014. ("MAR")

On September 26, 2018 Syros Pharmaceuticals (NASDAQ: SYRS), a leader in the development of medicines that control the expression of genes, reported that its Chief Executive Officer, Nancy Simonian, M.D., will present a corporate overview at the 2018 Cantor Global Healthcare Conference. Details are as follows (Press release, Syros Pharmaceuticals, SEP 26, 2018, View Source [SID1234529603]):

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2018 Cantor Global Healthcare Conference
Date: Wednesday, October 3
Presentation Time: 8:35 a.m. ET
Location: InterContinental New York Barclay 111 E 48th St, New York, NY

A live webcast of the presentation will be available on the Investors & Media section of the Syros website at www.syros.com. An archived replay will be available for approximately 30 days following the fireside chat.