On September 27, 2022 Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company focused on advanced therapies for autoimmune and inflammatory diseases, reported financial results for the quarter ended June 30, 2022, as well as pre-clinical and clinical progress on nomacopan and long-acting PAS-nomacopan (Press release, Akari Therapeutics, SEP 27, 2022, View Source [SID1234621438]). Investigational nomacopan is a bispecific recombinant inhibitor of complement C5 and leukotriene B4 (LTB4) currently being investigated in a Phase 3 clinical trial for use in severe pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA) and a pre-clinical program is developing long-acting PASylated nomacopan in geographic atrophy (GA) in dry age-related macular degeneration (dAMD).

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"We sharpened our pipeline focus on two programs that have the potential to address areas of significant unmet need for patients, including our Phase 3 clinical trial in severe pediatric HSCT-TMA that we believe is the shortest path to value creation for our investors," said Rachelle Jacques, President and CEO of Akari Therapeutics. "The recently announced financing provides us with the cash needed to bring our HSCT-TMA program to our next important milestone, which is the Part A data readout we expect in the first half of 2023, and to advance our promising pre-clinical program investigating PAS-nomacopan in geographic atrophy."

Akari Recent Progress and Highlights

Amidst a very challenging capital markets environment, Akari successfully closed a registered direct offering with healthcare-focused institutional and accredited investors, including participation of certain existing investors for the issuance of an aggregate 15,100,000 American Depositary Shares (ADSs) at $0.85 per ADS and a concurrent private placement of series A warrants to purchase 15,100,000 ADSs and series B warrants to purchase 15,100,000 ADSs for aggregate gross proceeds of approximately $12.8 million
Proceeds from the offering will support Akari’s narrowed pipeline focus, including the Phase 3 Part A clinical trial of nomacopan in severe pediatric HSCT-TMA and the promising pre-clinical program developing long-acting PAS-nomacopan in GA
The Phase 3 Part A clinical trial of nomacopan in severe pediatric HSCT-TMA remains on track for a data readout in the first half of 2023
Akari continues to participate in the U.S. Food and Drug Administration (FDA) Model Informed Drug Development (MIDD) program that, along with a robust PK/PD model, supported dose selection and rapid advancement into the Phase 3 Part A clinical trial of nomacopan in severe pediatric HSCT-TMA
Akari clinical data from 38 subjects in previous nomacopan clinical studies and healthy volunteers supported a PK/PD model and model simulations of 10,000 virtual patients that were used to inform FDA MIDD interactions that confirmed PK/PD model suitability and dosing selected for the Phase 3 Part A clinical trial of nomacopan in severe pediatric HSCT-TMA
Positive results from recent pre-clinical studies support the advancement of long-acting PAS-nomacopan toward IND/IMPD for clinical trials in GA as well as the potential for the investigational treatment to address areas of unmet patient needs
PK measurements have indicated the half-life of early generation PAS-nomacopan in a standard ophthalmic pre-clinical model can be accurately predicted, which supports work to develop new generation PAS-nomacopan that may enable dosing intervals of more than three months between intravitreal injections with the potential of an acceptable low dose volume
Pivotal data recently released on a late-stage complement-only inhibitor (not nomacopan) further reinforce the efficacy of C5 inhibition in GA. Pre-clinical PAS-nomacopan is a bispecific inhibitor of both complement C5 and LTB4, which is important because previously presented data show LTB4 inhibition by PAS-nomacopan may also reduce the risk of sight-threatening choroidal neovascularization (CNV), a safety risk associated with current late-stage complement-only inhibitors.
Second Quarter 2022 Financial Results

As of June 30, 2022, the Company had cash of approximately $8.2 million, compared to cash of approximately $9.4 million as of December 31, 2021. Following the end of the second quarter, Akari closed a registered direct offering of 15,100,000 ADSs along with a concurrent private placement of series A warrants to purchase 15,100,000 ADSs and series B warrants to purchase 15,100,000 ADSs, resulting in approximately $12.8 million in gross proceeds.

Research and development expenses for the second quarter 2022 were approximately $2.9 million, as compared to approximately $2.2 million in the same quarter the prior year. This increase of 31% or $0.7 million was primarily due to increased expenses due to the timing of manufacturing of nomacopan to support ongoing clinical trials.

General and administrative expenses for the second quarter 2022 were approximately $3.0 million, as compared to approximately $2.1 million in the same quarter the prior year. This increase of 43% or $0.9 million was primarily due to increased consulting fees for services such as strategic advisory, investor relations, and other activities.

For the second quarter 2022, total other income was approximately $136,000 as compared to total other expense of approximately $16,000 in the second quarter of 2021. This $152,000 increase was primarily attributed to foreign currency exchange gains in the current period as compared to foreign currency exchange losses in the prior period.

Net loss for the second quarter 2022 was approximately $5.7 million, as compared to approximately $4.3 million for the period of 2021. This increase was primarily due to the aforementioned higher research and development expenses as well as higher general and administrative expenses.

Net cash used in operating activities for the six months ended June 30, 2022 was approximately $13.6 million, as compared to approximately $12.3 million for the six months ended June 30, 2021. This increase was primarily due to the aforementioned higher net loss as well as an increase in prepaid expenses and other current assets.

On 26 September 2022 Arovella Therapeutics Ltd, a biotechnology company focused on developing its invariant Natural Killer T (iNKT) cell platform for cancer treatment, reported it collaborate with clinical stage immune-oncology company Imugene Limited (ASX: IMU), using its onCARlytics platform (Press release, Imugene, SEP 26, 2022, View Source [SID1234629026]). This will test Arovella’s CAR19-iNKT (ALA-101) cell therapy with Imugene’s onCARlytics platform to seek and destroy solid tumours. The read out from the preclinical studies performed through the collaboration is expected in H1 2023.

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Arovella’s lead iNKT product, ALA-101, contains a Chimeric Antigen Receptor (CAR) that targets tumour cells producing CD19 on their surface. Typically, CD19 expression is on the cell surface of blood cancers. Imugene’s onCARlytics platform enables solid tumour cancers to express CD19 on their surface, which creates the opportunity to use ALA-101 to seek and destroy the solid tumour cells. Currently, ALA-101 is being developed for CD19-producing blood cancers. Working with Imugene raises the possibility of using ALA-101 to treat solid tumour cancers.

Imugene is evaluating a range of CD19 targeting therapies in combination with onCARlytics of which Arovella’s ALA-101 will be included, allowing Arovella to benchmark its iNKT therapy for the treatment of solid tumours. Initial pre-clinical data from Arovella demonstrates that ALA-101 cells outperform conventional T cells in haematological malignancies that produce CD19 and CD1d. Achieving compelling data in this study would open up a new therapeutic area of potential indications in solid tumours for Arovella’s iNKT cell therapy products.

Solid tumours represent 90% of diagnosed cancer cases1, and as of 2021, the solid tumour market was valued at US$209.61 billion2.

Imugene’s CEO and MD, Leslie Chong, commented, "Our onCARlytics platform opens up the possibility to treat solid tumours with existing CD19 targeting drugs. Solid tumours account for more than 90% of cancers diagnosed, and our technology has the potential to change the outcomes for these patients. We are excited to see how Arovella’s iNKT cell platform and other drugs in our trial perform on the back of our platform. We are delighted to work with another Australian-based biotechnology company focussing on cancer treatment."

Arovella’s CEO and MD, Dr Michael Baker, commented, "We are excited to collaborate with Imugene to evaluate potential of our iNKT product in combination with their onCARlytics product for treatment of solid tumours. Imugene’s onCARlytics is one of the most promising products to be developed for solid tumours. We believe strongly in the unique capability of our iNKT program and look forward to seeing how ALA-101 performs in these models. Arovella is excited by the prospect of expanding its use of its iNKT cell therapy platform to treat solid tumours."

The research partnership is material to Arovella as it includes development of Arovella’s iNKT cell therapy platform licensed from Imperial College London (see ASX announcement dated 18 June 2021) with Arovella’s onCARlytics CD19 oncolytic virus licensed from the City of Hope. The combination has the potential to be a novel approach to treating certain solid tumour cancers.

The research collaboration agreement is effective as of 26 September 2022 and has an initial term of twelve months. The strategic collaboration may be terminated upon completion of the research, or by mutual agreement. Arovella will fund the preclinical studies from its planned research budget, and no further funding is required for the initial research completed through the partnership. Currently each party has full intellectual property (IP) rights (patents) to their individual background technology. In the event new IP is generated from the research collaboration (each a "Combination Invention"), the parties shall discuss in good faith the filing, prosecution, maintenance, enforcement, defense of any patent applications thereto, as well as each party’s right to use, such Combination Invention. After the results from this research agreement are known and can be quantified, the parties will negotiate in good faith (and without obligation) whether to jointly develop or commercialise on the outcomes of the strategic collaboration on commercially reasonable terms.

On September 26, 2022 ReCode Therapeutics, a genetic medicines company using superior delivery to power the next wave of mRNA and gene correction therapeutics, reported that Company leadership will participate in two upcoming investor conferences in October (Press release, ReCode Therapeutics, SEP 26, 2022, View Source;utm_medium=rss&utm_campaign=recode-therapeutics-to-participate-in-upcoming-october-investor-conferences [SID1234621545]):

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Chardan’s 6th Annual Genetic Medicines Conference

BMO Biopharma Spotlight Series: Gene Editing & Therapeutics

On September 26, 2022 AnPac Bio-Medical Science Co., Ltd. ("AnPac Bio," the "Company" or "we") (ANPC), a biotechnology company with operations in the United States and China focused on early cancer screening and detection, reported that on September 26, 2022, the Company and a group of nine institutional and individual investors (the "Investors") have signed legally binding agreements for the investment in the Company by the Investors of approximately $3.67 Million (Press release, Anpac Bio, SEP 26, 2022, View Source [SID1234621440]).

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Under the terms of the various Shares Purchase Agreements, the Investors will invest in a private offering, in the aggregate, $3.67 Million to purchase 36.7 million of the Company’s Class A shares. It is expected that the investment will be completed in late September 2022.

Dr. Chris Yu, Co-Chairman of the board and Co-CEO of the Company, commented: "This equity investment is important to the Company’s operations as well as research and development including its Class III medical device clinical trial. We believe the investments signal continued confidence in the long term and fundamental prospects of our Company and its technology. We are entering into the critical Class III medical device clinical trial at three well-known hospitals. The successful completion of this clinical trial and obtaining a Class III medical device license is very important to our efforts to commercialize our cancer detection technology and Company valuation. We believe we have a very strong innovation track record in addition to the world’s largest multi-cancer detection sample size (in the new generation cancer detection technology space) according to a 2021 report by US marketing and research firm Frost & Sullivan."

On September 26, 2022 Servier, an international pharmaceutical company, and Oncodesign Precision Medicine (OPM), a subsidiary of Oncodesign (ALONC – FR0011766229) specialized in precision medicine, reported that Servier has exercised its exclusive license option for their drug candidate, an inhibitor of the LRRK2 (Leucine-Rich-Repeat Kinase 2) kinase target (Press release, Servier, SEP 26, 2022, View Source [SID1234621437]).

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In March 2019, Servier and Oncodesign started a research and development collaboration on LRRK2 kinase inhibitors, from the OPM’s Nanocyclix platform, as potential therapeutic agents for Parkinson’s disease. This collaboration is based on the complementary expertise of Servier and OPM in neurodegenerative diseases and kinase inhibitors. In June 2021, the two companies announced the selection of a preclinical drug candidate. Servier expects to start a "Phase 1 study in healthy volunteers" before the end of 2022.

The exercise of the option triggers a €7 million milestone payment by Servier to OPM. In total, Servier could pay OPM up to €320 million in R&D, regulatory and commercial milestones, plus potential royalties on future sales.

Jan Hoflack, PhD, Chief Scientific Officer and Managing Director of Oncodesign and OPM, said: « The approval of the CTA dossier by the ANSM and the EC, and the exercise of the license option by Servier, precede the first evaluation in humans of our key LRRK2 inhibitor drug candidate. This drug candidate is the first molecule from our Nanocyclix technology to enter the clinic for therapeutic evaluation. LRRK2 is a well-known kinase in the pharmaceutical field since 2005. This kinase has proven to be very difficult to target with inhibitors but has the potential to slow the progression of Parkinson’s disease. This would be a major progress for patients suffering from this disease, for which only symptomatic treatments currently exist. Our collaboration with Servier, which started in 2019, led to the discovery of a promising drug candidate in less than 4 years, demonstrating the maturity of the Nanocyclix technology, our expertise in the kinase field, as well as Servier’s expertise in the neurosciences field, coupled with strong synergy and efficiency within the joint Servier/Oncodesign/OPM team. »

Philippe Genne, PhD, Chairman and CEO and founder of Oncodesign and OPM explains: « I am very pleased with the rapid and successful development of this collaboration with the Servier Group, a long-standing partner of our company. More than ever, this term has a meaning here, as this is the first compound from Oncodesign’s research to reach the clinical development stage. This project is also an important program in OPM’s growth strategy. We will be able to capitalise on this solid base for the promising development of this young and experienced biotech. There is still a long way to go before the candidate becomes a drug, but this step was crucial and announces further successes for the benefit of patients. »

Ross Jeggo, PhD, Global Head of Neuroscience and Immuno-inflammation Therapeutic Area states: « The progress of the research and development program for this drug candidate in Parkinson’s disease is the result of the combined expertise of Servier and its partner Oncodesign. This collaboration reflects the Group’s commitment to focus its research on diseases with high medical needs, working closely with partners who share their know-how and technologies to create innovation synergies and accelerate the discovery of therapeutic solutions for the benefit of patients. »

About Parkinson’s disease

Parkinson’s disease is the most common neurodegenerative disorder responsible for motordysfunction, affecting 1% of the world’s population aged over 60[4]. In total, about 170,000 people are treated in France, with approximately 25,000 new cases reported[5] each year. It is a chronic disease with a progressive evolution of symptoms: slowed movements, tremors, rigidity. Parkinson’s disease is characterized by a progressive loss of dopaminergic neurons and by an accumulation of the protein α-synuclein in the brain. Current treatments, which are only symptomatic, are based on dopamine supplementation to compensate for the loss of dopaminergic neurons and reduce motor disorders, but their effectiveness diminishes with time. To date, there is no neuroprotective therapy able to slow down the progression of the disease. Modifying the course of the disease remains the main objective in the research and development of new treatments for Parkinson’s disease.

About the LRRK2 target

Parkinson’s disease is considered as an idiopathic disease, i.e. without a clearly identified origin in the vast majority of cases. Pathogenic mutations in LRRK2 protein are the most common monogenic form of Parkinson’s disease; a high level of LRRK2 protein activity is observed in these patients and also in idiopathic patients. The pathological features and clinical symptoms of an idiopathic patient and a patient with LRRK2 mutations are similar. LRRK2 is a multi-domain protein, which contains both kinase and GTPase enzymatic activities, where the pathogenic mutations are located. Inhibiting LRRK2 would therefore have neuroprotective potential, able to modify the progression of Parkinson’s disease. Last May, the first Phase I and Phase Ib clinical trials with other LRRK2 inhibitors showed very encouraging results. The collaboration program between Servier and OPM aims to develop a differentiating compound that addresses the major medical needs of Parkinson’s disease patients.