On May 16, 2022 Avacta Group plc (AIM: AVCT), a clinical stage oncology drug company developing innovative cancer therapies and powerful diagnostics based on its proprietary pre|CISION and Affimer platforms, reported that AffyXell, its joint venture with South Korean drug maker Daewoong Pharmaceutical, has expanded its strategic partnership with GenScript ProBio, a leading biopharmaceutical manufacturer (Press release, AffyXell Therapeutics, MAY 16, 2022, View Source [SID1234614677]).

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AffyXell is a joint venture established in January 2020 by Daewoong Pharmaceutical and Avacta, developing the next generation of cell and gene therapies based on mesenchymal stem cells which incorporate Affimer immunotherapies. This new class of stem cell therapy is designed to produce Affimer proteins, in situ in the body, that reduce inflammatory or autoimmune responses to the stem cell therapy to potentially enhance their therapeutic effects.

GenScript ProBio, part of Genscript Biotech Corporation (HKG: 1548), is a leading global contract development and manufacturing organisation providing a best-in-class, one-stop platform for research and development of biological drugs, DNA plasmids and lentiviruses, and for clinical production of cell and gene therapy products.

AffyXell and GenScript ProBio are extending their strategic manufacturing partnership, which they entered into in December 2021, that covers AffyXell’s first drug development programme to include additional future programmes. The partnership covers process development and production of viral vectors required for the production of AffyXell’s future cell therapy products. In addition, as part of this strategic alliance, GenScript has now committed to take an equity position in AffyXell at a future funding round, and the two companies will collaborate in the area of business development, including potential out licensing.

Dr Alastair Smith, Chief Executive Officer of Avacta, commented: "This is an important strategic partnership for AffyXell with a world leading contract development and manufacturing partner. The fact that GenScript ProBio are prepared to take a strategic equity stake in AffyXell is a very strong validation of the potential for AffyXell’s next generation cell and gene therapies, and the future valuation of the business."

"We are delighted with the excellent progress being made by AffyXell with the Affimer immunomodulators provided by Avacta under our joint venture agreement."

Jongsang Ryu, Chief Executive Officer of AffyXell, commented: "We were able to attract strategic investment from the CDMO partner as the excellence and potential of our next-generation cell therapy platform have been recognized."

"This agreement will be an opportunity to accelerate the development of therapies targeting intractable diseases, providing meaningful options for the suffering patients."

Dr Brian Min, Chief Executive Officer of GenScript ProBio commented: "We are pleased to partner with AffyXell and are honored to support this next-generation cell therapy project with GenScript ProBio’s stable and high-yield viral vector platform. We expect many patients to benefit from this innovative genetically modified mesenchymal stem cells therapy soon."

On May 16, 2022 Exicure, Inc. (NASDAQ: XCUR), an early-stage biotechnology company focused on the development of next generation nucleic acid therapies targeting RNA to address both genetic and non-genetic neurological disorders and hair loss disorders, reported financial results for the quarter ended March 31, 2022 and provided an update on its business strategy and corporate progress (Press release, Exicure, MAY 16, 2022, View Source [SID1234614704]).

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"Exicure is off to a promising start in 2022 as we have made progress with our preclinical SCN9A program for the treatment of pain with several potential therapeutic candidates identified and we are conducting initial in vivo animal studies to support candidate selection in 2023," commented Matthias Schroff, Ph.D., Chief Executive Officer of Exicure. "We continue to advance our partnered programs with Ipsen and AbbVie, and the recently announced private placement investment led by CBI USA, Inc. ("CBI USA") is expected to provide us with additional resources as we continue our mission to pursue treatments for patients with unmet medical needs," concluded Dr. Schroff.

Corporate Progress

Recent highlights include:

On May 10, 2022, Exicure announced a $5.0 million raise in a private placement transaction priced at market premium
Agreed to sell an aggregate of 26,021,011 shares of the Company’s common stock to certain accredited investors in a private placement in public equity ("PIPE") financing at a purchase price of $0.1937 per share, representing an approximately 45% premium to the 10-day volume weighted-average share price from May 9, 2022.
New investor CBI USA led the transaction; existing investor, Abingworth LLP, also participated.
Transaction is expected to close on or about May 19, 2022, subject to the satisfaction of customary closing conditions.
In connection with the PIPE, CBI USA received the right to nominate a member to the Company’s board of directors (the "Board"), effective as of the closing date. CBI USA will also have the right to designate one individual to attend all meetings of the Board in a nonvoting observer capacity.
Net proceeds from the transaction expected to support the Company’s advancement of its preclinical program, including the development of its SCN9A product candidate, as well as other working capital and general corporate purposes.
Corporate highlights for the first quarter of 2022 include:

Repaid in full all outstanding indebtedness and other obligations under the Company’s credit facility with MidCap, effective March 15, 2022.
Continued to advance the Company’s SCN9A preclinical discovery program. Exicure anticipates results from initial in vivo animal studies by year-end 2022, with the goal of therapeutic candidate selection in the second half of 2023.
Progressed work with partnered programs towards potential pre-clinical milestones in 2023.
Actively pursuing out-license opportunities for the Company’s clinical asset, cavrotolimod.
Continuing to pursue near-term partnering opportunities for pain and other neuroscience programs.
First Quarter 2022 Financial Results

Cash Position: Cash, cash equivalents and short-term investments were $27.6 million as of March 31, 2022, as compared to $48.3 million as of December 31, 2021. The Company expects that its cash and cash equivalents, together with the expected $5.0 million gross proceeds from the PIPE transaction in May 2022, will enable it to fund its current operations into the first quarter of 2023.

Revenue: Revenue was $2.6 million for the quarter ended March 31, 2022, reflecting an increase of $1.6 million from revenue of $1.0 million for the quarter ended March 31, 2021. The increase in revenue of $1.6 million is mostly due to the recognition of non-cash revenue of $2.1 million associated with the Company’s collaboration with Ipsen Biopharm Limited, partially offset by a decrease in revenue of $0.5 million associated with the Company’s collaboration with AbbVie Inc.

Research and Development (R&D) Expense: Research and development expenses were $7.1 million for the quarter ended March 31, 2022, as compared to $10.3 million for the quarter ended March 31, 2021. The decrease in R&D expense for the three months ended March 31, 2022 of $3.1 million reflects a reduction in employee headcount and fewer discovery, preclinical, and clinical program activities resulting from the restructuring activities that the Company announced in December 2021.

General and Administrative (G&A) Expense: General and administrative expenses were $3.2 million for the quarter ended March 31, 2022, as compared to $2.9 million for the quarter ended March 31, 2021. The increase in G&A expense of $0.3 million for the three months ended March 31, 2022 was mostly due to higher legal costs and retention award expense for current employees, partially offset by a decrease in recruiting costs, investor relations costs, and stock-based compensation.

Net Loss: The Company had a net loss of $8.3 million for the quarter ended March 31, 2022, as compared to a net loss of $12.5 million for the quarter ended March 31, 2021. The decrease in net loss was primarily driven by lower R&D expense and higher non-cash revenue during the period.

Going Concern: Given the Company’s current cash position, operating plans and forecasted negative cash flows from operating activities over the next twelve months, management believes there is substantial doubt regarding the Company’s ability to continue as a going concern within one year after the date that its unaudited condensed consolidated financial statements for the quarter ended March 31, 2022 are issued. The Company will require substantial additional financing to address the Company’s working capital and other financing needs to pursue its business strategy.

On May 16, 2022 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, reported that Dr. Neal Walker, President and CEO of Aclaris, will virtually present a company overview at the H.C. Wainwright Global Investment Conference, which will be available beginning on Tuesday, May 24, 2022 at 7:00 a.m. ET (Press release, Aclaris Therapeutics, MAY 16, 2022, View Source [SID1234614581]). Management will be available May 23rd throughout the day for virtual 1×1 meetings.

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A webcast of the presentation may be accessed through the "Events" page of the "Investors" section of Aclaris’ website, www.aclaristx.com. The webcast will be archived for at least 30 days on the Aclaris website.

On May 16, 2022 Proteros biostructures GmbH ("Proteros"), an expert in integrated structure-based drug discovery, reported an expansion of its collaboration with AstraZeneca (LSE/STO/Nasdaq: AZN) focused on the discovery and development of novel epigenetic drugs (Press release, AstraZeneca, MAY 16, 2022, View Source [SID1234614614]). The new multi-year agreement builds on an ongoing collaboration with AstraZeneca announced in June 2021, to include the development of small molecule inhibitors targeting a second cancer-associated epigenetic protein.

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Under the terms of this new agreement, Proteros will receive research funding and will be eligible for success-based research, development, and commercial milestone payments of up to USD 75 million plus tiered royalties on annual net sales. The collaboration combines Proteros’ expertise in the identification and characterization of inhibitors with novel binding mechanisms for technically challenging drug targets with AstraZeneca’s leadership in the discovery and development of oncology medicines.

"The expansion of our agreement with AstraZeneca with an additional drug discovery program reflects our successful ongoing collaboration to identify selective inhibitors for notoriously challenging disease targets," said Dr. Torsten Neuefeind, CEO of Proteros. "This agreement strengthens our collaboration with a global biopharmaceutical leader and we look forward to joining forces again to discover novel inhibitors with the potential to effectively treat cancer patients in the future."

The Proteros platform will ensure high selectivity to a specific target variant within and across multiple protein families through biochemical, biophysical and cellular assays supported by rapid turnaround in the structural determination of drug-target interactions by X-ray crystallography and cryo-Electron Microscopy technologies.

On May 16, 2022 Inhibikase Therapeutics, Inc. (Nasdaq: IKT) (Inhibikase or Company), a clinical-stage pharmaceutical company developing therapeutics to modify the course of Parkinson’s disease and related disorders, reported financial results for the first quarter ended March 31, 2022 and highlighted recent developments (Press release, Inhibikase Therapeutics, MAY 16, 2022, View Source [SID1234614646]).

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"As we kicked off 2022, we have worked diligently to advance our clinical and preclinical programs as well as extend our thought leadership in Parkinson’s disease. This past month we brought together leading key opinion leaders in the field of movement disorders to host an educational event highlighting the current unmet need, competitive and regulatory landscape in Parkinson’s disease as well as delineated our development strategy for IkT-148009 as a potential therapeutic option for patients," commented Milton Werner, Ph.D., President and Chief Executive Officer of Inhibikase. "As we look ahead, we anticipate dosing the first patients in our Phase 2a study for IkT-148009 in Parkinson’s disease this quarter. This study will allow us to evaluate our selective c-Abl kinase inhibitor in Parkinson’s patients dosed for a 12-week period. In addition, we anticipate submitting our IND application for IkT-001Pro for stable phase Chronic Myelogenous Leukemia as well as report preclinical data from at least one study of IkT-148009 in Multiple Systems Atrophy in the second quarter. As we advance these programs forward, we will continue to be good stewards of capital to maximize shareholder value. We expect our current cash on hand to be sufficient to fund our operations into the third quarter of 2023."

Recent Developments and Upcoming Milestones:
Phase 2a clinical study for IkT-148009: Inhibikase anticipates dosing the first patients in its Phase 2a study of IkT-148009, known as the "201 Trial," in the second quarter. The trial will be a 3:1 randomized, double-blind, twelve-week dosing trial and will evaluate the safety and tolerability of three doses of IkT-148009 in up to 120 patients diagnosed with Parkinson’s disease who have not yet progressed to the need for symptomatic therapy. The trial will also measure a hierarchy of motor and non-motor function inside and outside of the brain as secondary endpoints and will evaluate whether treatment with IkT-148009 leads to a reduction or clearance of pathogenic alpha-synuclein aggregates as exploratory endpoints.

Hosted virtual investor event featuring Key Opinion Leaders in Parkinson’s disease: In April 2022, Inhibikase held a virtual investor event highlighting the Company’s clinical progress of IkT-148009, provided an overview of the current Phase 2 program and described the current unmet need and competitive landscape in Parkinson’s disease. A replay of the event can be accessed here.

Phase 1b study in clinical trial of IkT-148009: In March 2022, Inhibikase presented data from the first cohort of its Phase 1b study for IkT-148009 at the Alzheimer’s & Parkinson’s Diseases Conference (AD/PD). Data suggested that the safety and tolerability profile in patients closely matched that of older healthy volunteers. Pharmacokinetics of IkT-148009 in volunteers and subjects was also similar, further suggesting that the pharmacology of IkT-148009 is consistent across patient groups and penetrates the Central Nervous System. The Company expects to complete dosing of the second cohort of the Phase 1b study in the second quarter of 2022 and present additional data at a medical meeting later this year.

Investigational New Drug application (IND) for IkT-001Pro for stable-phase Chronic Myelogenous Leukemia (CML): In the first quarter of 2022, Inhibikase completed clinical batch manufacturing of its pill formulated IkT-001Pro, a prodrug formulation of imatinib mesylate. The Company is conducting the necessary stability studies for the IND submission package and expects to submit the IND application in the second quarter of 2022. Following clearance by the FDA, the Company expects to initiate bioequivalence studies in accordance with the 505(b)(2) regulatory pathway.

Preclinical studies evaluating IkT-148009 in animal models of Multiple System Atrophy (MSA): Inhibikase expects to report preclinical data evaluating IkT-148009 in at least one of two animal models of MSA in the second quarter of 2022. Depending on the preclinical results in animal models of MSA and subject to agreement with the FDA and equivalent regulatory bodies in the European Union, Inhibikase may advance IkT-148009 into a Phase 2a clinical study in the fourth quarter of 2022.
First Quarter 2022 Financial Results
Net Loss: Net loss for the quarter ended March 31, 2022, was $4.7 million, or $0.18 per share, compared to a net loss of $2.6 million, or $0.26 per share for the first quarter in 2021.

R&D Expenses: Research and development expenses were $3.0 million for the quarter ended March 31, 2022, compared to $2.4 million in the quarter ended March 31, 2021. The increase was driven by a $2.1 million increase in non-grant related research offset by a decrease of $1.2 million in grant related research expenditures and a decrease of $0.4 million in non-cash stock compensation expense. The non-grant related research was expended primarily in connection with the Company’s Phase I PD clinical trial.

SG&A Expenses Selling, general and administrative expenses for the quarter ended March 31, 2022 were $1.7 million compared to $1.6 million for the first quarter in 2021. The increase was primarily due to increased headcount resulting in increased compensation expense of $.2 million, increased legal fees, board fees, investor relation and consulting fees of $0.1 million and a net increase of $0.2 million for other normal operating expenses offset by decreased non-cash stock-based compensation expense of $0.4 million.

Cash Position: Cash and cash equivalents were $36.6 million as of March 31, 2022. The Company expects that existing cash and cash equivalents will be sufficient to fund its operating expenses and capital expenditure requirements into the third quarter of 2023.

Conference Call and Webcast
The conference call is scheduled to begin at 8:00am ET Tuesday, May 17, 2022. Participants should dial 877-407-4018 (United States) or 201-689-8471 (International) with the conference code 13729218. A live webcast may be accessed using the link here, or by visiting the investors section of the Company’s website at www.inhibikase.com. After the live webcast, the event will be archived on Inhibikase’s website for approximately 90 days after the call.