On August 29, 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 second quarter ended June 30, 2019 and recent clinical progress (Press release, Akari Therapeutics, AUG 29, 2019, View Source [SID1234539160]).

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"We are pleased with the progress we have made advancing our BP, HSCT-TMA and AKC programs and are encouraged by the initial data we have received to date in these programs," said Clive Richardson, Chief Executive Officer of Akari Therapeutics. "Both AKC and BP have further planned clinical readouts this year, providing a potential opportunity for advancing both programs into pivotal trials in 2020 and further supporting the novel therapeutic role of combined C5 and LTB4 treatment. In addition, we are planning to start a pivotal clinical trial for HSCT-TMA in the fourth quarter of this year."

Second Quarter 2019 and Recent Business Highlights

Pediatric HSCT-TMA
The Company continues to progress towards a pivotal trial for HSCT-TMA with nomacopan, which is expected to start in the fourth quarter of 2019. This condition has an estimated 80% mortality rate in children with this severe disease, with currently no approved treatments. In a March 2019 meeting, a framework for the trial design was agreed with the U.S. Food and Drug Administration (FDA). In August 2019, the FDA granted Fast Track designation for nomacopan for the treatment of HSCT-TMA in pediatric patients.
Phase II clinical trial in patients with BP
Initial results from the first three patients with mild-to-moderate BP in the ongoing Phase II trial with nomacopan demonstrated a rapid reduction in BP Disease Area Index (BPDAI) score and blistering of 52% and 87%, respectively, by day 42. There were no drug related serious adverse events. The Company anticipates new safety and efficacy data in mild-to-moderate patients from this study to be given as an oral presentation at the the 28th European Academy of Dermatology and Venereology (EADV) Congress, October 10, 2019.
In early August, the Company announced new data demonstrating synergistic benefits of nomacopan’s dual C5 and LTB4 inhibitory activity in pemphigoid disease, generated by Dr. Christian Sadik’s group at University of Lubeck, Germany, and published in the August 2019 edition of JCI Insight [link].
Phase I/II clinical trial in patients with AKC
Successfully completed Part A of TRACKER, a Phase I/II clinical trial evaluating the safety and efficacy of topical nomacopan in patients with moderate-to-severe AKC. Results showed a rapid response and an overall improvement of 55% in the composite clinical score, which was composed of an improvement in symptoms of 62% and signs of 52% by Day 56. Three patients were treated with twice daily nomacopan eye drops in addition to standard of care for up to 56 days, with one patient completing 14 days and then withdrawing for reasons unrelated to the study. All patients had been on maximal topical cyclosporine, the standard of care, for at least three months prior to entry. The nomacopan eye drops were found to be comfortable and well tolerated with no serious adverse events. Enrollment in the Part B placebo-controlled efficacy arm in 16 patients continues to progress, with data read out planned for the fourth quarter of 2019.
Clive Richardson has been appointed permanent Chief Executive Officer of Akari after having served as interim Chief Executive Officer since May, 2018
Upcoming Events and Milestones

HSCT-TMA pivotal clinical trial expected to start in the fourth quarter of 2019.
Mild-to-moderate BP trial data to be presented at EADV Congress, October 10, 2019.
Completion of Part B of AKC Phase I/II trial by the fourth quarter of 2019.
Second Quarter 2019 Financial Results

Research and development (R&D) expenses in the second quarter of 2019 were $3.6 million, as compared to R&D expenses of $5.1 million in the same quarter the prior year. This decrease was primarily due to lower manufacturing expenses as the Company had previously manufactured clinical trial material for supply through 2019, which was slightly offset by higher clinical trial costs and personnel expenses. R&D expenses for the six months ended June 30, 2019 were $1.3 million reflecting the receipt of a Q1 R&D tax credit of $4.9 million.
General and administrative (G&A) expenses in the second quarter of 2019 were $2.4 million, as compared to $2.9 million in the same quarter last year. This decrease was primarily due to lower expenses associated with professional services, personnel and rent, partially offset by higher stock-based non-cash compensation expenses.
Total other income for the second quarter of 2019 was $1.9 million, as compared to total other expense of $43,000 in the same period the prior year. This change was primarily due to $2.0 million of higher income related to the change in the fair value of the stock option liabilities in 2019 compared to 2018, and to higher foreign exchange gains of approximately $39,000 in 2019 as compared to 2018.
Net loss for the second quarter of 2019 was $4.1 million, compared to a net loss of $8.0 million for the same period in 2018. The decrease in net loss in the second quarter of 2019 was due primarily to the change in the fair value of the stock option liabilities and foreign exchange gains previously cited, accompanied by lower operating expenses in the second quarter of 2019.
As of June 30, 2019, the Company had cash of $2.7 million, as compared to cash of $5.4 million as of December 31, 2018. On July 3, 2019, the Company sold to certain institutional investors, accredited investors and an existing shareholder, RPC Pharma Ltd., an affiliated entity of Dr. Ray Prudo, Akari’s Chairman, an aggregate 2,368,392 registered American Depository Shares (ADSs) of Akari at a purchase price of $1.90 per ADS, resulting in gross proceeds of approximately $4.5 million. Additionally, for each ADS purchased by investors, the investors received an unregistered warrant to purchase one-half ADS. The warrants have an exercise price of $3.00 per ADS, were exercisable upon their issuance and will expire five years from the issuance date.
As of June 30, 2019, the Company has sold to Aspire Capital Fund, LLC (Aspire Capital) a total of $2.0 million of ordinary shares. Subsequent to June 30, 2019, the Company sold to Aspire Capital a further $3.5 million of ordinary shares and approximately $14.5 million remains available for draw down under the purchase agreement entered into with Aspire Capital.

On August 28, 2019 Clovis Oncology, Inc. (Nasdaq: CLVS) reported that Patrick J. Mahaffy, Chief Executive Officer and President, will present at the 2019 Wells Fargo Healthcare Conference on Thursday, September 5, 2019 at 8:35 AM Eastern Time (Press release, Clovis Oncology, AUG 28, 2019, View Source [SID1234539055]). The conference will be held at the Westin Copley Place in Boston, MA.

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A live webcast of the presentation can be accessed through the investor relations section of the Company’s website at www.clovisoncology.com. Following the live presentation, a replay of the webcast will be available on the Company’s website for 30 days.

On August 28, 2019 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a biotechnology company focused on developing targeted cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported that nationally and internationally renowned clinician and oncologist, Dr. Manuel Hidalgo, has confirmed that he will be Principal Investigator (PI) for PharmaCyte’s planned clinical trial in locally advanced, inoperable pancreatic cancer (LAPC) now that he is at Weill Cornell Medical Center (Press release, PharmaCyte Biotech, AUG 28, 2019, View Source [SID1234539071]).

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Dr. Hidalgo, a leading physician-scientist who specializes in pancreatic cancer and drug development, was recently appointed Chief of the Division of Hematology and Medical Oncology at Weill Cornell Medicine and NewYork-Presbyterian/Weill Cornell Medical Center. Previously, Dr. Hidalgo was a Professor of Medicine at the Harvard Medical School and the Chief of the Division of Hematology Oncology and Director of the Rosenberg Clinical Cancer Center at the Beth Israel Deaconess Medical Center.

Dr. Hidalgo commented, "With PharmaCyte proceeding with the GMP production of the clinical trial product for its LAPC trial, I am more excited than ever to be a part of PharmaCyte’s clinical trial team. I remain enthusiastic about being the PI for the trial. I am also optimistic about the potential that PharmaCyte’s treatment may hold for patients suffering from LAPC and possibly other types of solid cancerous tumors. For example, PharmaCyte’s therapy for cancers involving the pancreas, liver and breast may offer an effective way to specifically target those cancers with little to no side effects from the chemotherapy."

On August 28, 2019 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported it has received a positive recommendation from the independent Data Monitoring Committee (DMC) to continue enrolling into the company’s Phase 3 "DOM–INNATE" study (Dusquetide treatment in Oral Mucositis – by modulating INNATE immunity) for SGX942 (dusquetide) in the treatment of oral mucositis in patients with head and neck cancer (HNC) (Press release, Soligenix, AUG 28, 2019, View Source [SID1234539087]). Following its prospectively defined interim analysis, including unblinded assessment of the study’s primary efficacy endpoint, the DMC recommended that approximately 70 additional subjects be randomized into the trial, increasing the study sample size from 190 to 260 evaluable subjects. The DMC’s recommendation indicates that a beneficial SGX942 effect has been observed; however, to maintain the rigorous assumption of 90% statistical power for the primary efficacy endpoint, an increase was required to take into account any potential variability and/or distribution changes observed in the Phase 3 study patient population that may have differed from the initial protocol design assumptions. No safety concerns were reported by the DMC based on the interim analysis. The study remains on target to complete enrollment and provide topline results in the first half of 2020.

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"We are pleased to have received the DMC’s recommendation to continue enrolling to the adjusted target of 260 subjects in order to maintain our conservative power calculation," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "Since reaching the 90 subject enrollment threshold required for the conduct of the interim analysis in April, we currently have over 160 subjects enrolled in the study. With this new level of clarity from the DMC’s analysis of the interim Phase 3 study data and given our current rate of patient enrollment, we are confident that we will remain on target to announce topline results in the first half of 2020. We have invested a significant amount of the Company’s resources over the last several years into the oral mucositis development program and it is gratifying to have received this feedback from the DMC. Given our current cash resources, we anticipate that the available funds are sufficient to cover the additional study subjects needed. We believe SGX942 has the potential to be a valuable therapy in the treatment of oral mucositis, which is an area of high unmet medical need."

"The DMC’s recommendation from the interim analysis is very encouraging and provides for a more precise understanding of the patient population and treatment effect, as it is based on the actual data from the ongoing Phase 3 clinical trial," stated Richard Straube, MD, Senior Vice President and Chief Medical Officer of Soligenix. "Our understanding of the historic variability in oral mucositis patients, especially placebo patients, and the variability that can occur when going from small Phase 2 to larger Phase 3 clinical trials, is precisely the reason we included the interim analysis. It is to ensure that we do not get misdirected by our initial set of assumptions and stop the trial with a substantial but non-statistically significant benefit in the SGX942 group."

Dr. Straube continued, "Although we remain blinded to the potential reasons that informed the DMC’s recommendation to increase the sample size, we do know that the DMC’s recommendation reflects that they saw a prospectively defined promising signal in the primary endpoint, which will allow us to aggressively pursue completing the trial in order to demonstrate SGX942’s potential to successfully ameliorate the devastating impact of oral mucositis in patients with HNC receiving chemoradiation therapy (CRT). Further, the added subjects will also allow us to more rigorously assess any ancillary benefits of SGX942 (reduced infection, increased survival and increased tumor clearance rate) as well as build a more robust safety database that is important to support potential marketing authorizations with the US and EU health authorities. We would like to thank the DMC members for their assistance, as well as our esteemed medical advisory board and our dedicated clinical investigators for their ongoing efforts in the design and conduct of this important clinical trial."

About the Phase 3 DOM–INNATE Study

Based on the positive and previously published Phase 2 results (Study IDR-OM-01), the pivotal Phase 3 clinical trial (Study IDR-OM-02) is a highly powered, double-blind, randomized, placebo-controlled, multinational trial originally targeted to enroll approximately 190 subjects with squamous cell carcinoma of the oral cavity and oropharynx, scheduled to receive a minimum total cumulative radiation dose of 55 Gy fractionated as 2.0-2.2 Gy per day with concomitant cisplatin chemotherapy given as a dose of 80-100 mg/m2 every third week. Subjects are randomized to receive either 1.5 mg/kg SGX942 or placebo given twice a week during and for two weeks following completion of CRT. The primary endpoint for the study is the median duration of severe oral mucositis, assessed by oral examination at each treatment visit and then through six weeks following completion of CRT. Oral mucositis is evaluated using the WHO (World Health Organization) Grading system. Severe oral mucositis is defined as a WHO Grade of ≥3. Subjects are to be followed for an additional 12 months after the completion of treatment. Soligenix has been working with leading oncology centers internationally, a number of which participated in the Phase 2 study.

About Oral Mucositis

Mucositis is the clinical term for damage done to the mucosa by anticancer therapies. It can occur in any mucosal region, but is most commonly associated with the mouth, followed by the small intestine. It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of mucositis, that mucositis affects approximately 500,000 people in the US per year and occurs in 40% of patients receiving chemotherapy. Mucositis can be severely debilitating and can lead to infection, sepsis, the need for parenteral nutrition and narcotic analgesia. The gastrointestinal damage causes severe diarrhea. These symptoms can limit the doses and duration of cancer treatment, leading to sub-optimal treatment outcomes.

The mechanisms of mucositis have been extensively studied and have been recently linked to the interaction of chemotherapy and/or radiation therapy with the innate defense system. Bacterial infection of the ulcerative lesions is now regarded as a secondary consequence of dysregulated local inflammation triggered by therapy-induced cell death, rather than as the primary cause of the lesions.

It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of oral mucositis, that oral mucositis in HNC is a subpopulation of approximately 90,000 patients in the US, with a comparable number in Europe. Oral mucositis almost always occurs in patients with HNC treated with CRT and is severe, causing inability to eat and/or drink, in >80% of patients. It is common (40-100% incidence) in patients undergoing high dose chemotherapy and hematopoietic cell transplantation, where the incidence and severity of oral mucositis depends greatly on the nature of the conditioning regimen used for myeloablation.

In the pediatric population, head and neck cancer is a rarer occurrence and is caused by different underlying pathologies. The major types of HNC in children are lymphoma, sarcomas (including rhabdomyosarcomas), and neuroblastoma rather than squamous cell carcinoma, the major type of adult HNC cancers. Hematopoietic stem cell transplantation (HSCT), especially allogeneic transplantation with higher risk of oral mucositis, is more frequently used in the pediatric population than in adults when treating a number of primary tumor types, as seen in leukemia and lymphoma,. Both treatment of HNC and HSCT are associated with high risk of oral mucositis in the pediatric population.

Oral mucositis remains an area of unmet medical need where there are currently no approved drug therapies in the context of any solid tissue tumors.

About Dusquetide

Dusquetide (the active ingredient in SGX942) is an innate defense regulator (IDR), a new class of short, synthetic peptides. It has a novel mechanism of action whereby it modulates the body’s reaction to both injury and infection towards an anti-inflammatory, anti-infective and tissue healing response. IDRs have no direct antibiotic activity but, by modulating the host’s innate immune system responses, increase survival after infections caused by a broad range of bacterial Gram-negative and Gram-positive pathogens. It also accelerates resolution of tissue damage following exposure to a variety of agents including bacterial pathogens, trauma and chemo- and/or radiation therapy. Preclinical efficacy and safety has been demonstrated in numerous animal disease models including mucositis, colitis, macrophage activation syndrome (MAS) as well as bacterial infections, including melioidosis.

SGX942 has demonstrated safety in a Phase 1 clinical study in 84 healthy human volunteers. Positive efficacy results were demonstrated in an exploratory Phase 2 clinical study in 111 patients with oral mucositis due to CRT for HNC. Soligenix is working with leading oncology centers in the US and Europe to advance SGX942 in oral mucositis with the conduct of a pivotal Phase 3 clinical trial referred to as the "DOM–INNATE" study (Dusquetide treatment in Oral Mucositis – by modulating INNATE immunity).

SGX942 has received Fast Track Designation from the FDA for the treatment of oral mucositis as a result of radiation and/or chemotherapy treatment in HNC patients, as well as Promising Innovative Medicine designation in the United Kingdom by the Medicines and Healthcare Products Regulatory Agency for the treatment of severe oral mucositis in HNC patients receiving CRT. In addition, products containing the same active ingredient, dusquetide, have been granted Fast Track Designation as an adjunctive therapy with other antibacterial drugs, for the treatment of melioidosis and Orphan Drug Designations in the treatment of MAS and the treatment of acute radiation syndrome.

Soligenix has a strong intellectual property position in the IDR technology platform, including composition of matter for dusquetide and related analogs. Dusquetide was developed pursuant to discoveries made by Professors B. Brett Finlay, PhD and Robert Hancock, PhD of the University of British Columbia, Canada. Soligenix has received partial funding from NIH for its oral mucositis clinical studies. The Phase 2 study was supported with a Phase I SBIR grant (#R43DE024032) award, with the Phase 3 study being supported by a Phase II SBIR grant (#R44DE024032) award.

In addition, a high level review of the IDR technology platform is available here.

On august 28, 2019 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported that management will participate in the following investor conferences in September (Press release, Autolus, AUG 28, 2019, View Source [SID1234550687]):

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Dr. Christian Itin, chairman and chief executive officer, will present at the Wells Fargo 2019 Healthcare Conference in Boston on Wednesday, September 4 at 3:00 p.m. ET and will host one-on-one meetings with attendees. A live webcast of the presentation will be available on the investor relations section of Autolus’ website at View Source An archived replay will be available on the company’s website for a period of 90 days after the conference.
Dr. Itin will participate in a fireside chat at the Morgan Stanley 17th Annual Global Healthcare Conference in New York on Monday, September 9 at 9:20 a.m. ET and will host one-on-one meetings with attendees. A live audio webcast of the fireside chat will be available on the investor relations section of Autolus’ website at View Source An archived replay will be available on the company’s website for a period of 90 days after the conference.
Andrew Oakley, chief financial officer, will present at the H.C. Wainwright 21st Annual Global Investment Conference in New York on Tuesday, September 10 at 2:10 p.m. ET and will host one-on-one meetings with attendees.