On November 4, 2019 Akari Therapeutics, Plc (Nasdaq: AKTX), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where complement (C5) and/or leukotriene (LTB4) systems are implicated, reported financial results for the third quarter ended September 30, 2019 and recent clinical progress (Press release, Akari Therapeutics, NOV 4, 2019, View Source [SID1234550590]).

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"We are excited about the positive clinical data we are accumulating in patients treated with nomacopan across our target rare disease indications. The rapid and sustained clinical clinical improvement combined with the positive long-term safety profile we have observed in patients, helps to offer further validation of nomacopan’s unique method of action as an inhibitor of both the complement and leukotriene pathways," said Clive Richardson, Chief Executive Officer of Akari Therapeutics. "We look forward to further progress across both our topical and subcutaneous clinical programs in 2020 as we look to drive nomacopan through the clinic and towards helping patients afflicted by these rare and debilitating inflammatory conditions. In addition, we seek to partner those programs in which a joint development approach can produce a faster outcome."

Third Quarter 2019 and Recent Business Highlights

Akari’s strategy is to focus on orphan inflammatory diseases with significant unmet medical need, where the role of the complement and leukotriene systems are implicated. Akari’s lead programs are in BP, AKC, and HSCT-TMA where clinical data with nomacopan has shown rapid and sustained clinical improvement in patients. These diseases have no approved treatments.

Further evidence for potential therapeutic benefits of inhibition of C5 and LTB4 by nomacopan

In the last three months, the Company has announced preclinical data in both BP and AKC demonstrating the likely combined role of C5 and LTB4 in these two severe inflammatory conditions:

In BP, an orphan condition with no approved treatment, the Company announced new data demonstrating synergistic benefits of nomacopan’s dual C5 and LTB4 inhibitory activity. This new study was undertaken by Dr. Christian Sadik’s team at University of Lubeck, Germany. These data were published in the August 2019 edition of JCI Insight [link].
In AKC, a surface of the eye disease with no approved treatment, the Company announced new data from the conjunctival tissue of patients showing for the first time the presence of both the C5a receptor and the leukotriene LTB4 receptor on the conjunctival surface of the eye.
Pediatric HSCT-TMA

A pivotal trial for HSCT-TMA with nomacopan is expected to start in the fourth quarter of 2019. This devastating condition has an estimated 80% mortality rate in children and has no approved treatments. In August 2019, the FDA granted Fast Track designation to nomacopan for the treatment of HSCT-TMA as well as orphan drug designation for treatment of pediatric patients.
Phase II clinical trial in patients with BP

Phase II trial results with nomacopan were presented at the 28th European Academy of Dermatology and Venereology (EADV) Congress on October 10, 2019. Four of the six patients were classified as at the upper limit of moderate BP. The four patients saw a rapid and significant improvement in symptoms, with a mean 63% decline in Bullous Pemphigoid Disease Area Index (BPDAI) score and mean 68% decline in blister score by day 42, with either no or minimal early steroid treatment. The data showed nomacopan’s potential as monotherapy with the additional potential benefit of reducing steroid use which has multiple adverse effects.
During the third quarter of 2019, the FDA granted orphan drug designation for nomacopan for the treatment of BP. The company is now exploring pivotal trial designs.
Phase I/II clinical trial in patients with AKC

Successfully completed Part A of the Phase I/II clinical trial in severe AKC patients who showed a rapid overall improvement of a mean 55% in the composite clinical score. The nomacopan eye drops were found to be comfortable and well tolerated with no reported drug related serious adverse events. Enrollment in the Part B placebo-controlled efficacy arm of the study in 16 patients continues, with a data read out expected in the first quarter of 2020.
Paroxysmal nocturnal hemoglobinuria (PNH) program

The Company continues to accumulate positive long-term treatment data, which includes over 20 cumulative patient-years of data with no reported drug related serious adverse events. Our ongoing Phase III PNH study in naïve patients is expected to have an interim data readout in mid 2020 with the next stage subject to our pen injector program which aims to hold one week’s supply of nomacopan stable at room temperature with a daily 0.3ml injection.
Third Quarter 2019 Financial Results

As of September 30, 2019, the Company had cash of $6.3 million. In addition, the Company received in October 2019 $2.9 million in research and development tax credits from the UK tax authorities. This compares to cash of $5.4 million as of December 31, 2018.
During the third quarter of 2019, the Company sold to Aspire Capital Fund, LLC (Aspire Capital) a total of $4.6 million of ordinary shares. As of September 30, 2019, approximately $13.4 million of the original $20 million remains available for draw down under the equity purchase agreement entered into with Aspire Capital.
Research and development (R&D) expenses in the third quarter of 2019 were $1.8 million, as compared to R&D expenses of $3.3 million in the same quarter the prior year. This decrease was primarily due to the recognition of the aforementioned R&D tax credit of $2.9 million in the third quarter of 2019, proceeds of which were received in October 2019.
General and administrative (G&A) expenses in the third quarter of 2019 were $1.4 million, as compared to $2.4 million in the same quarter last year. This decrease was primarily due to lower expenses associated with professional fees and rent.
Total other income for the third quarter of 2019 was $0.4 million, as compared to total other expense of $0.6 million in the same period the prior year. This change was primarily due to $1.0 million of higher income related to the change in the fair value of the stock option liabilities in 2019 compared to 2018.
Net loss for the third quarter of 2019 was $2.8 million, compared to a net loss of $3.6 million for the same period in 2018. The decrease in net loss in the third quarter of 2019 was due primarily to lower net R&D and G&A expenses, as well as change in the fair value of the stock option liabilities previously cited, offset by the one-time litigation settlement gain that was recorded in the third quarter of 2018.

On November 4, 2019 Genoscience Pharma is reported a new data from in vivo study of GNS561 alone or in combination with a PD-1 inhibitor in a transgenic mouse model of hepatocarcinoma (HCC) (Press release, GenoScience, NOV 4, 2019, View Source [SID1234550467]). In addition to good tolerance, GNS561 in combination with a PD-1 inhibitor showed significant antitumoral activity, with a 77% decrease in the macronodule counts compared to control group. This new data shows GNS561 allows PD-1 inhibitor to recover its antitumoral efficacy.

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Genoscience Pharma, a clinical-stage biotechnology company dedicated to discovering and developing anticancer treatment drugs, today announces that its poster demonstrating promising results from a combination study with a PD-1 inhibitor in a transgenic mouse model of hepatocarcinoma (HCC) was selected for presentation at the American Association for the Study of Liver Diseases (AALSD) Liver Meeting 2019 being held November 8-12, 2019 in Boston, MA.

The in vivo study was performed in a transgenic immunocompetent mouse model of HCC (ASV-B). Animals were treated by vehicle, GNS561 or PD-1 inhibitor as monotherapy or GNS561 in combination with PD-1 inhibitor. Results showed an outstanding anticancer response, with a 77% decrease of the macronodule count in the combination group compared to controls.

"We are delighted to be presenting this positive in vivo study at the AASLD Liver Meeting 2019. These results may open a new horizon in the area of immuno-oncology by enlarging indication of the use of immune checkpoint inhibitors in tumor types that are marginally sensitive to immunotherapy or for patients developing resistance to checkpoint inhibitors. We believe our results provide a strong rationale for combining our drug to a PD-1 inhibitor antibody in clinical trials with HCC patients" said Pr Eric Raymond, Chief Medical Officer at Genoscience Pharma.

"We are looking forward to assessing this combination in HCC patients, for which immunotherapy hasn’t answered the current medical need" commented Pr Philippe Halfon, president and founder of Genoscience Pharma.

Genoscience Pharma will be also presenting three additional posters at the conference. Data on the primary results of the ongoing Phase 1b assessing GNS561 in patients with primary or secondary liver cancers will be presented as well as efficacy preclinical data against intra-hepatic cholangiocarcinoma and liver fibrosis.

The details for the Company’s poster presentation are as follows:

Presenting Author: Philippe Halfon, MD, PhD.
Abstract title: GNS561, a New Oral Clinical-Stage Small Molecule Combined with a PD-1 Inhibitor Showed Remarkable Anti-Tumor Effects in a Transgenic Immunocompetent Hepatocellular Carcinoma Mouse Model (ASV-B). Poster 1993.
Presentation date and time: November 11, 2019 (presentation from 12.30 pm to 1.30 pm)

On November 4, 2019 Sentinel oncology Limited reported an expansion of it’s collaboration with PhoreMost Limited to accelerate the development of SOL686, a novel allosteric Polo-like kinase 1 (Plk1) inhibitor (Press release, Sentinel Oncology, NOV 4, 2019, View Source [SID1234550397]). The programme adds to Sentinel Oncology’s NeuroOncology pipeline, has started formal preclinical studies and is positioned to enter clinical development as a glioma treatment by 2021.

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Mitotic PLKs are widely recognised as playing crucial roles in disease causing pathways, including K-Ras mutant cancers. Traditional approaches to drugging PLK enzymes have focused on targeting their active site; however this tactic has been hindered by toxicity-associated adverse events. Sentinel Oncology’s allosteric PLK1 inhibitor takes the novel approach of targeting the Polo-box domain (PBD) of the PLKs, thereby aiming to mitigate adverse events seen by active site inhibitors.

The programme has demonstrated a promising combination of specific drug-like properties, mode of action and target validation data obtained so far. The programme, which originated from the laboratory of Prof Ashok Venkitaraman at the University of Cambridge, has been optimised by both PhoreMost and Sentinel Oncology to the point where SOL686 was recently identified as a candidate drug for the treatment of glioma. Both PhoreMost and Sentinel Oncology received funding from Innovate UK for the drug discovery programme.

On November 4, 2019 Mateon Therapeutics, Inc. (OTCQB:MATN) (‘Mateon") reported that they have completed their acquisition of PointR Data Inc. (PointR), a privately-held, developer of high performance cluster computer and artificial intelligence technologies (Press release, Mateon Therapeutics, NOV 4, 2019, View Source [SID1234550276]). This will create a publicly traded artificial intelligence ("AI") and blockchain driven immuno-oncology company to apply machine learning and AI approaches to its pipeline of first in class TGF-β immunotherapies for late stage cancers such as gliomas, pancreatic cancer and melanoma.

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Leveraging its deep partnership with IBM, the PointR team will combine its own AI Vision technology with industry standard Blockchain to transform drug manufacturing and real-world evidence monitoring for clinical trials. The combined system has the potential to automatically record individual key steps in cGMP manufacturing operations including the flow of people, raw materials and operations in trusted perpetual blockchain ledgers that are indisputable. This has the potential to create much more efficient GMP manufacturing operations while simultaneously improving reliability and data security.

As the result of the acquisition, Saran Saund, former CEO of PointR, will assume the role of Chief Business Officer (CBO)/ General Manager of AI Division at Mateon and Burcak Beser, former CTO of PointR, will assume the role of Sr. VP/CTO of AI Division at Mateon.

Silicon Valley entrepreneur, Saran has been founder, CEO and GM at startups and public companies. Passionate about applying technology innovations to real world markets, he successfully founded an AI consortium to accelerate enterprise adoption of AI which engaged leading universities and technology vendors. A startup veteran, his track record includes senior leadership roles at companies that were acquired by leaders such as Marvell (MRVL) and Qualcomm (QCOM). His startup Cybercash (CYCH) had a successful IPO on NASDAQ. Saran started his career at Xerox PARC pushing 1’s and 0’s as a software engineer.

In the last 20 years, as a Silicon Valley technologist, Burcak has founded startups and established multi-site cross-organizational teams that led to designs and deployments of world-class systems. With 144 patent applications, of which 88 have been issued, Burcak’s innovative solutions are connecting billions of people in the world together. Passionate about AI, he sees working on AI as an opportunity to solve problems that were considered insurmountable. Burcak spends most of his time building systems to make these solutions a reality. He finished his Masters in Artificial Intelligence in 1991, winning the best thesis-of-the-year award and highest honors.

"The PointR acquisition is clearly transformative for Mateon Therapeutics," said Vuong Trieu, Ph.D., Chairman and Chief Executive Officer of Mateon. "We welcome the addition of Saran Saund and Burcak Beser to our management team, their engagement greatly enhances our ability to move forward as the biotech company of the future."

"With the completion of the acquisition of PointR, we will be working hard at the commercialization and monetization of PointR Data technologies," said Saran Saund, CBO of Mateon, "Since the signing of the definitive agreement in August, we have been exploring implementation of AI and Blockchain technologies into Mateon’s workflow – in particular the application of PointR Data Vision AI in pharmaceutical manufacturing and patient monitoring. We look forward to announcing multiple collaborations in the near future."

Leading Silicon Valley VC and Chairman of PointR, Balaji Baktha said, "The traditional drug discovery process can take up to a decade of development timeline costing over a billion dollars and yet has a track record of a 90 percent failure rate for new drugs entering clinical development. AI and blockchain technologies can dramatically improve the process and help millions of under-served patients. This merger promises to transform the industry by reducing the overall timelines and costs."

On November 4, 2019 Santhera Pharmaceuticals (SIX: SANN) reported a strategic change to its management team with the appointment of Dario Eklund as CEO, effective December 1, 2019 (Press release, Santhera Pharmaceuticals, NOV 4, 2019, View Source [SID1234550275]). Thomas Meier will continue as a Board Member and will chair the newly formed Board of Director’s Scientific Committee. He will also serve as a special advisor to Dario Eklund, ensuring a smooth transition in Santhera’s leadership.

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Thomas Meier, PhD, Chief Executive Officer of Santhera, said: "This is a very exciting time for Santhera. Puldysa (idebenone) is under review by the European Medicines Agency as treatment for patients with Duchenne muscular dystrophy (DMD) and vamorolone, a first-in-class steroidal drug, is in a pivotal DMD trial. We anticipate market entries for these products from 2020 onwards and we are gearing up commercial preparations both in Europe and the US. As we transition to become a leader in innovative treatments for rare neuromuscular and pulmonary diseases it is important for Santhera to be led by a CEO with international commercial experience. Dario shares my belief in Santhera’s potential and excitement about the future opportunities for the Company and he will contribute the perfect blend of global commercial expertise and management skills to lead Santhera into the growth phase ahead. As member of the Board I look forward to supporting Dario in his new role."

Dario Eklund joins Santhera from Vifor Pharma, where he most recently served as Chief Commercial Officer with full P&L responsibility for a global business with turnover in excess of CHF 1 billion and more than 1’000 employees. He was a member of Vifor’s Corporate Executive Committee and a member of the Board of Directors of the joint venture with Fresenius Medical Care (Vifor Fresenius Medical Care Renal Pharma). Prior to Vifor, Dario spent a decade in Boston as Vice President and a member of the Executive Committee at Organogenesis Inc., a Nasdaq-listed world leading company in regenerative medicine and cell therapy with three approved products. During his time the company experienced strong growth and rapid expansion with sales coming from Europe and USA (annual growth rates of 20-25% over a decade). He started his career with various roles in sales, global marketing, business development and general management at Novartis and Sanofi in a number of territories.

Elmar Schnee, Chairman of Santhera, said: "On behalf of the Board, I would like to express my sincere appreciation to Thomas for his well-recognized achievements as founder and member of Santhera’s executive team since 2004, first as Chief Scientific Officer and then as CEO. Under his leadership, Santhera developed from a hopeful start-up into a fully integrated pharmaceutical company with a first product on the market and a very promising and innovative rare disease product pipeline addressing neuromuscular and pulmonary diseases with high unmet medical need. We are extremely pleased that he will continue to contribute his drive and scientific excellence as Chair of the Scientific Committee and member of the Board of Directors, supporting our ambition to turn innovative ideas into novel products. As we seek to broaden the scope of our business, Dario is a perfect fit as CEO. He has an exceptional track record as a commercial leader with experience in large pharma and biotech environments, and we are confident that he will successfully lead Santhera as we move along a strong path for the future."

Dario Eklund, CEO elect of Santhera, commented: "It’s an exciting time to be joining the Santhera team and to help drive its next phase of development. With two late-stage DMD drug candidates covering essentially the entire disease spectrum in all patients, Santhera is poised to become the world leader in an area where the unmet medical need is significant. I am looking forward to leading a strong and dedicated team and working alongside the Board and management team of Santhera to deliver on the Company’s vision."