On November 15, 2018 Akari Therapeutics, Plc (NASDAQ:AKTX), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where complement and or leukotriene systems are implicated, reported its financial results for the third quarter ended September 30, 2018 (Press release, Akari Therapeutics, NOV 15, 2018, View Source [SID1234531344]).

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"We are focused on moving our four priority clinical programs forward and expect initial data from our trials in patients with bullous pemphigoid (BP) and atopic keratoconjunctivitis (AKC) in the first quarter of 2019," 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 with initial data readouts expected in the first quarter 2019:
Phase II trial in patients with BP, a severe blistering skin disease
Phase I/II trial in patients with AKC, a sight-threatening surface of the eye condition
Coversin clinical trials in orphan diseases in which complement dysregulation is the primary disease driver:
Two trials open in PNH: a Phase III trial in naïve patients and a Phase II trial in patients who are resistant to eculizumab
An open Phase II trial in atypical hemolytic syndrome (aHUS), a severe thrombotic microangiopathy
Ongoing named patient program in pediatric patients with thrombotic microangiopathy (TMA) post bone marrow transplant
Long-term safety study for Coversin
Total cumulative number of patient-years on Coversin treatment approximately 15 years
All patients in the long term study have now been treated for more than 15 months and the first patient has now been treated for 34 months
No drug related serious adverse events and no neutralizing antibodies reported to date
Six PNH patients were transfusion dependent prior to treatment with Coversin, of which four in the long-term study are now transfusion independent; two remain on transfusion.
Third Quarter 2018 Financial Results

Research and development (R&D) expenses in the third quarter of 2018 were $3.3 million, as compared to $6.4 million in the same quarter the prior year. The decrease was due primarily to lower manufacturing costs for Coversin as the Company had previously manufactured clinical trial material for supply through 2019, partially offset by higher clinical trial activity.
General and administrative (G&A) expenses in the third quarter of 2018 were $2.4 million, as compared to $2.2 million in the same quarter last year. This increase was due primarily to higher professional fees.
Operating expenses were $3.0 million in the third quarter of 2018. Excluding a $2.7 million one-time litigation settlement gain, operating expenses were $5.7 million in the third quarter of 2018, as compared to $8.5 million in the same quarter the prior year. This decrease is primarily due to lower R&D expenses.
Total other expense for the third quarter of 2018 was $0.6 million, as compared to $1.8 million in the same quarter the prior year. This change was primarily attributed to a $0.7 million loss in fair value of the stock option liabilities in the third quarter of 2018, compared to a $1.7 million loss in the third quarter of 2017.
Net loss for the third quarter of 2018 was $3.6 million, compared to a net loss of $10.4 million for the same period in 2017. This year over year decrease in net loss was due primarily to the aforementioned $2.7 million litigation settlement gain, lower R&D expenses and change in fair value of the stock option and warrant liabilities, which were lower in the third quarter of 2018 compared to the prior year period.
As of September 30, 2018, the Company had cash of $10.1 million, as compared to cash of $28.1 million as of December 31, 2017.
In addition, on September 26, 2018, the Company entered into a securities purchase agreement (the "Purchase Agreement") with Aspire Capital Fund, LLC ("Aspire Capital"), which provides that, upon the terms, Aspire Capital is committed to purchase up to an aggregate of $20.0 million of the Company’s ADSs over the 30-month term of the Purchase Agreement. In consideration for entering into the Purchase Agreement, concurrently with the execution of the Purchase Agreement, the Company issued 30,000,000 ordinary shares to Aspire Capital and sold to Aspire Capital 25,000,000 ordinary shares for $0.02 per share (equivalent to $2.00 per ADS and $500,000).

On November 15, 2018 Ipsen (Euronext: IPN; ADR: IPSEY) reported that the European Commission (EC) has approved Cabometyx (cabozantinib) 20, 40, 60 mg as a monotherapy for hepatocellular carcinoma (HCC) in adults who have previously been treated with sorafenib (Press release, Ipsen, NOV 15, 2018, View Source [SID1234531360]). This approval allows for the marketing of Cabometyx (cabozantinib) in this indication in all 28 member states of the European Union, Norway and Iceland.

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"Today’s EC approval for the use of Cabometyx provides a much needed new option for HCC patients. Until now, physicians in Europe had only one approved therapy for the 2nd line treatment of this aggressive and difficult-to-treat cancer.1,2 We are proud to offer Cabometyx as an innovative treatment that has been shown to extend survival in previously treated patients with HCC," said Harout Semerjian, Chief Commercial Officer, Ipsen. "This new indication reinforces Ipsen’s commitment to improve patients’ lives through the expansion of the clinical benefit of Cabometyx in the treatment of solid tumors."

Philippe Merle, M.D., Ph.D., Hepatology and Gastroenterology specialist at La Croix-Rousse Hospital, Lyon, stated: "Patients with HCC in Europe can now benefit from a treatment that has, through the CELESTIAL trial, proven effective in prolonging life and delaying disease progression. This is a very encouraging development for liver cancer patients, and provides physicians with a new therapeutic option for this complex disease."

The EC approval is based on the results of the global placebo-controlled CELESTIAL phase 3 pivotal trial which met its primary endpoint of overall survival (OS), with cabozantinib providing a statistically significant and clinically meaningful improvement in OS compared with placebo in patients with advanced HCC who have been previously treated with sorafenib.3 In July 2018, CELESTIAL phase 3 pivotal trial results were published in the New England Journal of Medicine.3

The EC has also approved Cabometyx for the treatment of advanced renal cell carcinoma (aRCC) both in treatment-naïve adults with intermediate or poor risk (May 2018) and in adults following prior vascular endothelial growth factor (VEGF)-targeted therapy (September 2016).

The detailed recommendations for the use of this product are described in the Summary of Product Characteristics (SmPC), available here.

About CELESTIAL

CELESTIAL is a randomized, double-blind, placebo-controlled global phase 3 study of cabozantinib versus placebo in patients with advanced HCC who have been previously treated with sorafenib. The study was conducted at more than 100 sites globally in 19 countries. The trial was designed to enroll 760 patients with advanced HCC who previously received sorafenib and may have received up to two prior systemic cancer therapies for HCC and had adequate liver function. Enrollment of the trial was completed in September 2017, and 773 patients were ultimately randomized. Patients were randomized 2:1 to receive 60 mg of cabozantinib once daily or placebo and were stratified based on etiology of the disease (hepatitis C, hepatitis B or other), geographic region (Asia versus other regions) and presence of extrahepatic spread and/or macrovascular invasion (yes or no). No cross-over was allowed between the study arms.

The primary endpoint for the trial is OS, and secondary endpoints include objective response rate (ORR) and progression-free survival (PFS). Exploratory endpoints include patient-reported outcomes, biomarkers and safety.

Based on available clinical trial data from various published trials conducted in the second-line setting of advanced HCC, the CELESTIAL trial statistics for the primary endpoint of OS assumed a median OS of 8.2 months for the placebo arm. A total of 621 events provide the study with 90 percent power to detect a 32 percent increase in median OS (HR = 0.76) at the final analysis. Two interim analyses were planned and conducted at 50 percent and 75 percent of the planned 621 events. The independent data monitoring committee for the study recommended that the trial should be stopped for efficacy following review of the second planned interim analysis. CELESTIAL trial met its primary endpoint, with cabozantinib providing a statistically significant and clinically meaningful improvement in OS compared to placebo in patients with advanced HCC. The safety data in the study were consistent with the established profile of cabozantinib.

About Hepatocellular Carcinoma (HCC)

Hepatocellular carcinoma is the most common form of liver cancer in adults.4 The disease originates in cells called hepatocytes found in the liver. With approximately 800’000 new cases diagnosed each year, HCC is the sixth most common cancer and the second-leading cause of cancer deaths worldwide.5,6 According to the GLOBOCAN data, it is estimated that across the European Union (EU-28) nearly 60’000 new patients will be diagnosed with liver cancer in 2020.7 Without treatment, patients with the disease in advanced stage usually survive between 4 and 8 months.8

About Cabometyx (cabozantinib)

Cabometyx is an oral small molecule inhibitor of tyrosine kinase receptors, including VEGFR, MET, AXL and RET. In preclinical models, cabozantinib has been shown to inhibit the activity of these receptors, which are involved in normal cellular function and pathologic processes such as tumor angiogenesis, invasiveness, metastasis and drug resistance.

In February of 2016, Exelixis and Ipsen jointly announced an exclusive licensing agreement for the commercialization and further development of cabozantinib indications outside of the United States, Canada and Japan. This agreement was amended in December of 2016 to include commercialization rights for Ipsen in Canada.

Cabometyx tablets are approved in the United States for the treatment of patients with advanced RCC. On September 9, 2016, the European Commission approved Cabometyx tablets for the treatment of advanced RCC in adults who have received prior vascular endothelial growth factor (VEGF)-targeted therapy in the European Union, Norway and Iceland. Cabometyx is also approved in Australia, Brazil, Canada, Hong Kong, South Korea, Switzerland, Taiwan and Ukraine. Cabometyx is available in 20 mg, 40 mg or 60 mg doses. The recommended dose is 60 mg orally, once daily.

On May 17, 2018, Ipsen announced that the European Commission approved Cabometyx for the first-line treatment of adults with intermediate- or poor- risk advanced renal cell carcinoma in the European Union, Norway and Iceland.

On November 15, 2018, Ipsen announced that the European Commission approved Cabometyx for the second-line treatment of hepatocellular carcinoma in adults who have previously been treated with sorafenib in the European Union, Norway and Iceland.

On November 15, 2018 Myovant Sciences (NYSE: MYOV), a leading clinical-stage biopharmaceutical company focused on women’s health and endocrine diseases, reported its participation in the following upcoming investor conferences (Press release, Myovant Sciences, NOV 15, 2018, http://investors.myovant.com/news-releases/2018/11-15-2018-132944791 [SID1234531386]):

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Evercore ISI HealthConX Conference in Boston, MA. President and Chief Executive Officer, Lynn Seely, M.D., will participate in a fireside chat on Tuesday, November 27, 2018, at 9:30 a.m. ET.
Citi’s 2018 Global Healthcare Conference in New York, NY. Myovant will host meetings with investors on Wednesday, December 5 and Thursday, December 6, 2018.

A live webcast of the Evercore presentation will be accessible on the Events page under the Investors and Media section of the Myovant website at www.myovant.com. Please connect to the company’s website at least 15 minutes prior to the presentation to ensure adequate time for any software download that may be required to listen to the webcast. A replay of the webcast will be available for 30 days following the conference.

On November 15, 2018 DNAtrix, a leader in oncolytic virus immunotherapies for cancer, reported that it will present interim results from the ongoing Phase 2 trial of its oncolytic virus DNX-2401 (tasadenoturev) with pembrolizumab for patients with recurrent glioblastoma at the upcoming 2018 Annual Meeting of the Society for Neuro-Oncology (SNO) which is being held in New Orleans, Louisiana from November 15th – 18th (Press release, DNAtrix, NOV 15, 2018, View Source [SID1234531404]).

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CAPTIVE / KEYNOTE-192 is a Phase 2 multicenter, dose escalation study evaluating a single intratumoral injection of DNX-2401 followed by standard dosing with pembrolizumab every three weeks to determine the optimal dose, safety, and efficacy in patients with recurrent glioblastoma. Preliminary results demonstrate that DNX-2401 with pembrolizumab is well tolerated, and associated with promising survival.

"I am excited by the early results of the trial. We have had some very remarkable responses. If I had not done the case myself, I would not have believed the complete response we have seen in one of our patients," said Gelareh Zadeh, MD, Associate Professor at the Department of Surgery University of Toronto, and presenting author for the CAPTIVE / KEYNOTE-192 study.

"There are limited treatment options for patients with this devastating disease, and we are encouraged by the initial data showing safety and disease control with the combination of DNX-2401 and pembrolizumab," added Frank Tufaro, PhD, CEO of DNAtrix. "We are pleased that enrollment is nearly complete and we are looking forward to maturation of the survival data."

DNAtrix collaborators will present additional results from studies of DNX-2401 (a.k.a. Delta-24-RGD) and murine DNX-2440 (a.k.a. Delta-24-RGDOX), an oncolytic adenovirus expressing the immune modulator OX40 ligand. Details of the presentations are as follows:

Interim results of a phase II multicenter study of the conditionally replicative oncolytic adenovirus DNX-2401 with pembrolizumab (Keytruda) for recurrent glioblastoma; CAPTIVE Study (KEYNOTE-192)
Date: Saturday, November 17
Abstract Number: ATIM-24
Presenter: Gelareh Zadeh, MD, University Health Network, University of Toronto, Toronto, ON, Canada
To access the abstract, click here >

Inflammatory reprogramming of gliomas using Delta-24-RGDOX and immunometabolic adjuvants
Date: Friday, November 16
Abstract Number: EXTH-27
Presenter: Teresa Nguyen, The University of Texas MD Anderson Cancer Center, Houston, TX, USA
To access the abstract, click here >

Local oncolytic adenovirus treatment affects both the innate and adaptive arms of the immune system and provides an avenue for enhancing immunotherapies for GBM
Date: Friday, November 16
Abstract Number: EXTH-03
Presenter: Martine Lamfers, PhD, Erasmus Medical Center, Rotterdam, Netherlands
To access the abstract, click here >

In Situ Autovaccination Mediated by Oncolytic Adenovirus Delta-24-RGDOX Induces Efficacious Immunity Against Metastatic Melanoma
Date: Saturday, November 17
Abstract Number: EXTH-30
Presenter: Hong Jiang, PhD, The University of Texas MD Anderson Cancer Center, Houston, TX, USA
To access the abstract, click here >

Delta-24-RGD in combination with positive regulators of the immune synapsis for gliomas in adults and children
Date: Sunday, November 18
Oral Presentation: Juan Fueyo, MD, The University of Texas MD Anderson Cancer Center, Houston, TX, USA

For more information about ongoing DNAtrix clinical studies, visit the ClinicalTrials.gov website: NCT02798406 (DNX-2401 + pembrolizumab for recurrent glioblastoma), NCT03178032 (DNX-2401 for newly diagnosed pediatric diffuse intrinsic pontine glioma, DIPG), and NCT03714334 (DNX-2440 for recurrent glioblastoma).

About DNX-2401 (Tasadenoturev)
DNX-2401 is an investigational oncolytic immunotherapy designed to treat cancer. DNX-2401 sets off a chain reaction of tumor cell killing by selectively replicating within cancer cells (but not normal cells), causing tumor destruction and further spread of the oncolytic virus to adjacent tumor cells. This process then triggers an immune response directed against the tumor. Previous studies demonstrated that DNX-2401 was well tolerated, provided clinical benefit, and extended survival for patients with recurrent glioblastoma.

On November 15, 2018 Diffusion Pharmaceuticals Inc. (Nasdaq: DFFN), a cutting-edge biotechnology company developing new treatments for life-threatening medical conditions by improving the body’s ability to bring oxygen to the areas where it’s needed most, reported that Chief Scientific Officer Dr. John L. Gainer will join leading clinicians and researchers from around the world in presenting a poster at the 2018 Annual Meeting of the Society for NeuroOncology (SNO) on Saturday, November 17, 2018 from 5:00-7:00pm EST (Press release, Diffusion Pharmaceuticals, NOV 15, 2018, View Source [SID1234531433]).

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SNO’s 23rd Annual Scientific Meeting and Education Day will be held from Thursday, November 15, 8:00am through Sunday, November 18, 1:00pm at the Marriott Hotel, New Orleans, Louisiana. SNO is a "multidisciplinary organization dedicated to promoting advances in neuro-oncology through research and education" whose annual conference is an opportunity for top health care professionals who specialize in central nervous system tumors to gather and present the latest advancements to fellow researchers, hospital administrators, and potential investors.

Dr. Gainer, who in addition to serving as the Chief Scientific Officer of Diffusion is also the inventor of Trans Sodium Crocinate (TSC), will present a poster on the Company’s Phase 3 INTACT (INvestigation of TSC Against Cancerous Tumors) clinical trial for the treatment of glioblastoma multiforme (GBM) brain cancer in biopsy-only patients. Phase 2 of Diffusion’s INTACT trial showed potentially breakthrough results in the use of TSC in treatment: a fourfold increase in the two-year survival rate of biopsy-only patients, which jumped from 10 percent to 40 percent. This result is especially promising for these patients and their families: biopsy-only patients have historically faced lower survival rates than patients for whom resection (surgery for removal of the tumor) is an option.

"Dr. Gainer’s focus on innovation is what drew me to his research in the first place, and resulted in our co-founding of Diffusion, where we continue our efforts to find new and effective treatments for life-threatening diseases like GBM brain cancer," said David Kalergis, Chairman and CEO of Diffusion. "At the 2018 Annual Meeting of the Society of NeuroOncology, Dr. Gainer will have an opportunity to present the exciting results from our Phase 2 GBM cancer trial and share our continued Phase 3 efforts for biopsy-only participants – work which represents real potential and promise for patients and their families"

"Finding effective and life-saving treatment for patients living with biopsy-only GBM brain cancer has long been a significant challenge for researchers, scientists, and physicians, so I’m looking forward to my poster presentation at the 2018 Annual Meeting of the Society of NeuroOncology, as it’s a chance to share the progress we’ve made on this front," said Dr. John L. Gainer, Chief Scientific Officer of Diffusion. "Hypoxic tumors are resistant to radiation therapy, and our Phase 2 results showed that using TSC – which was developed to fight hypoxia by bringing oxygen to the areas where it’s needed most – in conjunction with radiation and temozolomide (TMZ) treatment led to a significant increase in survival for biopsy only patients. Our Phase 3 study will focus on these biopsy-only patients, and I am eager to share that work with my colleagues from around the world at SNO’s 2018 Annual Meeting."

Diffusion’s Phase 3 INTACT program is using TSC to target inoperable GBM brain cancer. Historically, these biopsy-only patients have faced lower survival rates and increased obstacles to inclusion in clinical trials. Current standard of care for these GBM patients following diagnosis involves 6 weeks of radiation therapy along with daily temozolomide (TMZ), followed by six 4-week chemotheraphy sessions which include a high dosage of TMZ. Phase 2 of INTACT saw treatment with TSC added to the initial six weeks of radiation therapy and TMZ treatment. Phase 3 of INTACT will include treatment with TSC during the initial six week of radiation therapy and TMZ treatment, but will also include administration of TSC during the six months of chemotheraphy. The Company is currently engaged in an FDA-requested, 8-patient lead-in, scheduled to complete in January 2019. Randomization can begin following read out and reporting of that data."

Dr. Gainer’s presentation at SNO is just the latest development for Diffusion, which recently received FDA approval to enroll patients in an ambulance-based Phase 2 clinical trial testing TSC for the treatment of both ischemic and hemorrhagic stroke. The Company also recently appointed William "Bill" Hornung to the position of Chief Financial Officer.