On September 1, 2020 Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage 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, 2020, as well as recent clinical progress (Press release, Akari Therapeutics, SEP 1, 2020, View Source [SID1234564221]).

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"Akari continues to make good clinical progress, and since the beginning of the year has successfully completed its Phase II BP program and now has a clear pathway towards U.S. regulatory approval from the FDA. The HSCT-TMA program is set to commence recruitment in the fourth quarter of 2020 and we have initiated a COVID-19 pneumonia clinical program across multiple countries that leverages the unique dual C5 and LTB4 inhibition of nomacopan," said Clive Richardson, Chief Executive Officer of Akari Therapeutics.

Second Quarter 2020 and Recent Clinical Highlights

Akari’s lead programs are in BP, HSCT-TMA, COVID-19 pneumonia and AKC. These diseases have no approved treatments.

Patients with COVID-19 pneumonia

Clinical studies both underway and planned for patients with COVID-19 pneumonia in the U.S., U.K., and Brazil. In the U.S., a proposed multi-center randomized clinical program is in regulatory submission following the current treatment of patients via expanded access programs. In Brazil, patient recruitment has been completed for a proof of principle clinical study; potential progression into a follow-on randomized study is expected in early fourth quarter 2020. In the UK, nomacopan has been selected by the AGILE COVID-19 clinical trial initiative, and in addition data from approximately 50 COVID-19 pneumonia patients are expected in early fourth quarter 2020 from an observational study focused on identifying potential therapeutic biomarkers for optimizing use of nomacopan.
Nomacopan has been shown in clinical trials to inhibit both complement C5 activation and leukotriene B4 (LTB4), and has significant potential to simultaneously inhibit both microthrombi as well as block multiple cytokines (the cytokine storm) which together drive COVID-19 pneumonia and associated organ damage.
Phase II clinical trial in patients with BP

In August 2020, Akari announced a successful end-of-Phase II meeting with the U.S Food and Drug Administration (FDA) regarding its proposed pivotal Phase III program for the treatment of BP, which is now expected to start in the first half of 2021. The FDA has agreed to a two-part pivotal trial with Part A and Part B having the same structure, duration, endpoints and target population of moderate and severe BP patients. This follows positive topline results from Akari’s fully recruited Phase II study of nomacopan in BP patients previously announced in May 2020. The study achieved no reported drug-related serious adverse events. Seven of the nine treated patients showing a rapid and clinically significant reduction in Bullous Pemphigoid Disease Area Index (BPDAI) score, which measures the body surface area affected by blisters. Of the seven responders, three showed an 80%+ reduction in BPDAI score and three an approximately 40% reduction in BPDAI score within six weeks of starting nomacopan.
The European Medicines Agency (EMA) and the FDA have granted orphan drug designation for nomacopan for the treatment of BP.
Phase III clinical trial in pediatric patients with HSCT-TMA

During the first quarter of 2020, Akari initiated a pivotal Phase III trial for HSCT-TMA with nomacopan following the opening of an Investigational New Drug (IND) application by the FDA. As a result of the COVID-19 pandemic, site initiations were delayed and Akari expects enrollment to now commence in the fourth quarter of 2020. Akari has both FDA fast track for pediatric HSCT-TMA patients and orphan drug designation status for this program.
Phase I/II clinical trial in patients with AKC

As previously disclosed, enrollment into Part B of this study was paused due to the COVID-19 crisis. The study has now been unblinded to help evaluate next steps. Of the 12 patients recruited, a complete data set was available on 10 patients – two from Part A and eight from Part B. In Part B, of the eight patients recruited, six were placebo (four AKC patients and two other surface of the eye diagnosis) and two were treated with nomacopan (two AKC patients).
As a first-in-eye Phase I/II study, the primary endpoint measure was safety. Aggregating data from the eight AKC patients from Part A and Part B shows no ocular treatment emergent serious adverse events during the eight-week treatment period. Nomacopan is delivered topically without preservatives and is pH neutral. A comfort score measured after each eye drop installation showed the drug was comfortable and well tolerated. In all four efficacy categories (signs, symptoms, visual acuity and tear film break up) the four nomacopan treated AKC patients achieved a higher improved mean score than the four placebo AKC patients, however, the patient numbers are too small to show statistical significance on efficacy measures between the two treatment groups.
The interim data are encouraging and show that nomacopan eyedrops are safe and comfortable. Subject to operational constraints created by COVID-19, Akari aims to collect additional patient data with topical nomacopan. Akari recently received FDA approval for an IND for treatment with topical nomacopan which opens up the potential to broaden our topical ophthalmological program.
Second Quarter 2020 Financial Results

As of June 30, 2020, the Company had cash of approximately $12.7 million, compared to cash of $5.7 million as of December 31, 2019. Subsequent to the end of the second quarter of 2020, the Company received an additional $3.4 million in research & development tax credits.
On June 30, 2020, the Company entered into a securities purchase agreement with Aspire Capital Fund, LLC (Aspire Capital) which provides that Aspire Capital is committed to purchase up to an aggregate of $30.0 million of the Company’s ADSs, with each ADS representing one hundred ordinary shares, during a 30-month term of the purchase agreement. As of June 30, 2020, $30.4 million remains available under the facility together with a facility entered into with Aspire in 2018.
Research and development (R&D) expenses in the second quarter of 2020 were approximately $3.0 million, as compared to approximately $3.6 million in the same quarter the prior year. This decrease was primarily due to lower drug manufacturing costs and reductions of discretionary clinical expenses due to the COVID-19 outbreak delaying certain ongoing trials.
General and administrative (G&A) expenses in the second quarter of 2020 were approximately $2.9 million, as compared to approximately $2.4 million in the same quarter last year. This increase was primarily due to a one-time non-cash financing expense of $900,000 related to the 2020 purchase agreement with Aspire Capital. Higher expenses for insurance were partially offset by lower expenses for stock-based compensation, office rent and legal fees.
Total other expense for the second quarter of 2020 was approximately $2.4 million, as compared to total other income of $1.9 million in the same period the prior year. This change of $4.3 million was primarily due to approximately $4.3 million of loss related to the change in the fair value of the options and warrants liabilities in the second quarter of 2020 compared to the same period in 2019.
Net loss for the second quarter of 2020 was approximately $8.3 million, compared to a net loss of approximately $4.1 million for the same period in 2019. The increase in net loss was primarily due to higher Total Other Expense in 2020 and the aforementioned one-time non-cash financing expense related to the 2020 purchase agreement with Aspire Capital.
Important Message Regarding COVID-19

Akari’s clinical trial sites are based in areas currently affected by the global outbreak of the coronavirus, and public health epidemics such as this can adversely impact the Company’s business as a result of disruptions, such as travel bans, quarantines, and interruptions to access the trial sites and supply chains, which could result in material delays and complications with respect to research and development programs and clinical trials. Moreover, as a result of coronavirus, there is a general unease of conducting unnecessary activities in medical centers. As a consequence, the Company’s ongoing trials have been halted or disrupted. For example, the Phase I/II clinical trial in patients with AKC study has been halted and the Company expects that recruitment in the Phase III clinical trial in pediatric patients with HSCT-TMA will be delayed until the fourth quarter of 2020. It is too early to assess the full impact of the coronavirus outbreak on trials for nomacopan, but coronavirus is expected to affect Akari’s ability to complete recruitment in the original timeframe. The extent to which the coronavirus impacts operations will depend on future developments, which are highly uncertain and cannot be predicted with confidence, including the duration and continued severity of the outbreak, and the actions that may be required to contain the coronavirus or treat its impact. In particular, the continued spread of the coronavirus globally, could adversely impact the Company’s operations and workforce, including research and clinical trials and the ability to raise capital, could affect the operations of key governmental agencies, such as the FDA, which may delay the development of the Company’s product candidates and could result in the inability of suppliers to deliver components or raw materials on a timely basis or at all, each of which in turn could have an adverse impact on the Company’s business, financial condition and results of operation.

On September 1, 2020 Immunomic Therapeutics, Inc., ("ITI"), a privately-held clinical-stage biotechnology company pioneering the study of nucleic acid immunotherapy platforms, reported that results from multiple ATTAC clinical studies of dendritic cell vaccines have been published online by American Association for Cancer Research (AACR) (Free AACR Whitepaper) in an article titled, "Once, Twice, Three Times a Finding: Reproducibility of Dendritic Cell Vaccine Trials Targeting Cytomegalovirus in Glioblastoma (Press release, Immunomic Therapeutics, SEP 1, 2020, View Source [SID1234564220])."

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ITI is developing several dendritic cell vaccines for the treatment of cancer, including ITI-1000 for glioblastoma (GBM), with leaders in cancer immunotherapy for brain tumors, John Sampson, M.D., Ph.D. from Duke University and Duane Mitchell, M.D., Ph.D. from the University of Florida. ITI’s dendritic cell vaccine is designed to target the pp65 viral antigen of Cytomegalovirus (CMV) that is expressed in GBM, but not in normal brain cells.

In the ATTAC studies, the GBM patients’ white blood cells are removed, matured into dendritic cells (DCs), and modified to generate a vaccine to the pp65 viral protein when fused to the LAMP1 protein for antigen presentation. This DC vaccine is then returned to the patient. As observed in the ATTAC studies, ITI believes this approach may harness the body’s immune system to recognize, attack and destroy tumor cells that express CMV in GBM and potentially other cancers. The published results from the three original ATTAC clinical studies are summarized below:

Three separate clinical trials conducted by Drs. Mitchell and Sampson utilized Cytomegalovirus specific dendritic cell vaccines in patients with newly diagnosed glioblastoma.

The three small studies (total n = 26; NCT#s 00639639, 2366728) revealed that overall 5-year survival increased from a historical low of 5% to 25%. However, in two of the studies, vaccination site pre-conditioning with either Td or GM-CSF:

Enhanced the immune response by increasing migration of DCs to regional lymph nodes to "present" the pp65 CMV antigen to immune cells.
Increased the percentage of long-term, 5-year survivors to nearly 35% in that subset of treated patients who received vaccination site pre-conditioning with either Td or GM-CSF.
This data is preliminary, and additional studies are needed.

"These study results not only advance our understanding of a virus’ role in cancer, but they also signal tremendous hope to patients and their families suffering from this devastating disease," Sampson said. "I look forward to continued evaluation of ITI’s dendritic cell vaccines, including ITI-1000, in the ongoing, randomized, placebo-controlled ATTAC-II study."

"GBM is the most aggressive form of brain cancer, often resulting in a patient’s death within one to two years from diagnosis. Historically, it is a very difficult disease to treat and current treatment options offer limited benefit to extend survival," added Mitchell. "The results demonstrated with CMV-specific dendritic vaccines in the ATTAC studies are very encouraging, particularly in the observation of a significant fraction of long-term survivors and favorable safety profile of the vaccine platform. We remain steadfast in our pursuit to identify effective treatments for patients with GBM and look forward to the continued evaluation of ITI’s vaccines in addressing this clear and pressing unmet medical need."

The AACR (Free AACR Whitepaper) article can be found here.

About Glioblastoma (GBM)

According to the American Association of Neurological Surgeons, GBM is an aggressive brain cancer that often results in death within 15 months of diagnosis. GBM develops from glial cells (astrocytes and oligodendrocytes), grows rapidly, and commonly spreads into nearby brain tissue. GBM is classified as Grade IV, the highest grade, in the World Health Organization (WHO) brain tumor grading system. The American Brain Tumor Association reports that GBM represents about 15% of all primary brain tumors and approximately 10,000 cases of GBM are diagnosed each year in the U.S.

About ITI-1000 and the Phase 2 (ATTAC-II) Study

ITI-1000 is an investigational dendritic cell vaccine therapy currently in a Phase 2 clinical trial (ATTAC-II) for the treatment of GBM. ITI-1000 was developed using Immunomic’s proprietary investigational lysosomal targeting technology, UNITE, in the context of cell therapy. In May 2017, Immunomic exclusively licensed a patent portfolio from Annias Immunotherapeutics for use in combination with UNITE and ITI-1000, allowing Immunomic to combine UNITE with a patented and proprietary CMV immunotherapy platform. The ATTAC-II study (NCT02465268) is a Phase II randomized, placebo-controlled clinical trial enrolling patients with newly diagnosed GBM that will explore whether dendritic cell (DC) vaccines, including ITI-1000, targeting the CMV antigen pp65 improves survival. This study is enrolling up to 120 subjects at 3 clinical sites in the United States. For more information on the ATTAC-II study, please visit www.clinicaltrials.gov.

About UNITE

ITI’s investigational UNITE platform, or UNiversal Intracellular Targeted Expression, works by fusing pathogenic antigens with the Lysosomal Associated Membrane Protein 1, an endogenous protein in humans, for immune processing. In this way, ITI’s vaccines (DNA or RNA) have the potential to utilize the body’s natural biochemistry to develop a broad immune response including antibody production, cytokine release and critical immunological memory. This approach puts UNITE technology at the crossroads of immunotherapies in a number of illnesses, including cancer, allergy and infectious diseases. UNITE is currently being employed in a Phase II clinical trial as a cancer immunotherapy. ITI is also collaborating with academic centers and biotechnology companies to study the use of UNITE in cancer types of high mortality, including cases where there are limited treatment options like glioblastoma and acute myeloid leukemia. ITI believes that these early clinical studies may provide a proof of concept for UNITE therapy in cancer, and if successful, set the stage for future studies, including combinations in these tumor types and others. Preclinical data is currently being developed to explore whether LAMP1 nucleic acid constructs may amplify and activate the immune response in highly immunogenic tumor types and be used to create immune responses to tumor types that otherwise do not provoke an immune response.

On September 1, 2020 Selecta Biosciences, Inc. (NASDAQ: SELB), a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance platform, ImmTORTM, reported that it has entered into a debt financing facility for up to $35 million with Oxford Finance and Silicon Valley Bank (SVB) (Press release, Selecta Biosciences, SEP 1, 2020, View Source [SID1234564219]). Proceeds from the financing will be used to retire $12.6 million of existing debt, advance Selecta’s ImmTOR pipeline in gene therapy and autoimmune diseases, and for selected business development activities.

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"This partnership with Oxford Finance and SVB bolsters our financial position and extends our cash runway as we look to accelerate the development of our ImmTOR pipeline in gene therapy and autoimmune diseases," said Brad Dahms, Chief Financial Officer of Selecta. "We are pleased to continue our relationship with SVB and are excited to partner with Oxford Finance on this facility at such an important time for Selecta."

"We are thrilled to support Selecta’s efforts to continue development of its ImmTOR platform," said Christopher A. Herr, Senior Managing Director at Oxford Finance. "The potential for ImmTOR in gene therapy and autoimmune diseases align with our strategy of funding innovative life sciences companies".

"Selecta is driving important advancements in biologic therapies through its ImmTOR platform," said Ryan Roller, Director of Life Science and Healthcare at Silicon Valley Bank. "We are pleased to expand our relationship with the Selecta team and have the opportunity to support the Company’s next phase of growth."

Under the terms of the financing agreement, Oxford Finance and Silicon Valley Bank will provide Selecta with up to $35 million of borrowing capacity available in two tranches: $25 million at closing, and an additional $10 million available through September 30, 2021 upon the achievement of two development milestones: enrollment of the first patient in the Phase 1 clinical trial of Selecta’s gene therapy program in methylmalonic acidemia, and the enrollment of the first patient in each of the two Phase 3 clinical trials of SEL-212 in chronic refractory gout. The facility will require Selecta to make only interest payments through at least April 1, 2022. If the development milestones are met, the interest-only period will be extended to October 1, 2022. The entire facility matures on August 1, 2025. There are no financial covenants in the agreement.

On September 1, 2020 Incyte (Nasdaq:INCY) reported that it will present at the Morgan Stanley 18th Annual Global Healthcare (Virtual) Conference on Wednesday, September 16, 2020 at 9:30 a.m. EDT (Press release, Incyte, SEP 1, 2020, View Source [SID1234564218]).

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The presentation will be webcast live and can be accessed at Investor.Incyte.com and will be available for replay for 30 days.

On September 1, 2020 Invitae Corporation (NYSE: NVTA), a leading medical genetics company, reported that members of its management team will virtually participate in a fireside chat at the 18th Annual Morgan Stanley Global Healthcare Conference on Monday, September 14, 2020 at 4:30 p.m. Eastern / 1:30 p.m. Pacific (Press release, Invitae, SEP 1, 2020, View Source [SID1234564217]).

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Invitae’s (NVTA) mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. www.invitae.com (PRNewsFoto/Invitae Corporation)

The live webcast of the fireside chat may be accessed by visiting the investors section of the company’s website at ir.invitae.com. A replay of the webcast will be available shortly after the conclusion of the fireside chat.