On May 18, 2021 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, reported financial results for the first quarter 2021 and recent business progress (Press release, Abeona Therapeutics, MAY 18, 2021, View Source [SID1234580186]).

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"We are off to a fast start in 2021, reflecting our intense focus on execution," said Michael Amoroso, Chief Executive Officer of Abeona. "We are focused on completing enrollment in the EB-101 Phase 3 pivotal VIITAL study, gaining clarity on a regulatory path for ABO-102 in MPS IIIA, producing the first lot of Abeona-produced clinical grade product for ABO-102, and reporting additional neurocognitive and biomarker data from both the ABO-102 Transpher A and the ABO-101 Transpher B studies. We also have a robust preclinical pipeline, and we are conducting research assessing AAV capsids with the aim of IND-enabling studies in two to three eye indications. Importantly, our focus on building the right talent and experience on our leadership team positions us well to continue to advance our clinical programs toward delivering meaningful milestones later this year."

First Quarter and Recent Highlights

Corporate Updates

Appointed Michael Amoroso as President, Chief Executive Officer (CEO) and a member of the company’s Board of Directors.
Abeona strengthened its Board of Directors with the appointment of four new independent members who bring relevant operational leadership experience with life sciences companies, including in the areas of clinical development, manufacturing of cell therapy and gene therapy products, and corporate and financial compliance, to support the company’s focus on driving future growth and creating additional shareholder value.
EB-101 (Autologous, Gene-Corrected Cell Therapy)

Patient enrollment is ongoing for the EB-101 pivotal Phase 3 VIITAL study for RDEB. The company continues to expect to complete enrollment in the VIITAL study in 2021, depending upon the impact from the COVID-19 pandemic, including travel restrictions and safety concerns.
To support ongoing enrollment and commercial preparation, Abeona continues to work toward adding a second clinical site in the VIITAL study by the third quarter of 2021.
Presented data on long-term patient-reported outcomes following EB-101 treatment of RDEB wounds at the Society for Investigative Dermatology (SID) Virtual Meeting 2021, held from May 3-8, 2021. The results showed durable wound healing and reduction in pain through 6 years after treatment.
ABO-102 and ABO-101 (AAV-based Gene Therapies)

Presented new positive data from two ongoing Phase 1/2 clinical trials of ABO-102 in MPS IIIA and ABO-101 in MPS IIIB in late-breaking platform oral presentations at the 17th Annual WORLDSymposium in February 2021.
The FDA granted Abeona’s request and scheduled a Type B meeting in June 2021 to discuss the data-to-date from the ABO-102 Transpher A study and the potential path to a Biologics License Application (BLA) submission for ABO-102 in MPS IIIA.
Preclinical Pipeline

Presented new data supporting the potential of Cre-mediated dual AAV vector technology to enable delivery of large genes targeted for treatment of Stargardt disease during an oral presentation at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting, held virtually from May 1-7, 2021.
Abeona recently completed non-human primate (NHP) studies comparing several capsids with AAV8, the industry standard for intraocular administration, in order to further understand and characterize the company’s AAV capsids. The results showed that AAV204, part of Abeona’s in-licensed AIM capsid library, was superior to AAV8 using a recently developed route of ocular administration.
In a separate NHP experiment, the company’s AAV214 and AAVV214D5 capsids were tested versus AAV8 administered subretinally. Both capsids demonstrated nearly identical levels of transduction of photoreceptor and retinal pigmented epithelium (RPE) cells, which are the cell types most frequently affected in inherited retinal diseases, when compared with AAV8.
The results from the recently completed NHP studies support Abeona’s strategy to advance multiple preclinical eye programs into the clinic.
First Quarter Financial Results

Cash, cash equivalents and short-term investments totaled $86.8 million as of March 31, 2021, compared to $95.0 million as of December 31, 2020. Net cash used in operating activities was $13.6 million for the first quarter of 2021.

Research and development (R&D) expenses were $7.2 million for the first quarter of 2021, compared to $6.8 million in the comparable period in 2020. The increase in R&D expenses was primarily due to increased clinical and development work for the company’s gene and cell therapy product candidates, and increased salary and related costs. General and administrative (G&A) expenses were $6.6 million for the first quarter of 2021, compared to $6.4 million in the same period in 2020. The increase in G&A expenses was primarily due to increased professional fees, partially offset by decreased salary and related costs, and decreases in net other G&A expenses.

Net loss was $16.0 million for the first quarter of 2021, compared to net loss of $48.2 million for the comparable period in 2020. The decrease in net loss was primarily due to the non-cash impairment charge of $32.9 million related to the termination of the license agreement with REGENXBIO in the first quarter of 2020.

Conference Call Details

Abeona Therapeutics will host a conference call and webcast on Tuesday, May 25, 2021 at 8:30 a.m. ET, to discuss its first quarter 2021 financial results and business update. To access the call, dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) and Entry Code: 552097 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona’s website at www.abeonatherapeutics.com. The archived webcast replay will be available for 30 days following the call.

On May 18, 2021 Grey Wolf Therapeutics ("Grey Wolf"), a drug discovery company focused on developing first-in-class immuno-oncology agents, reported that it has been awarded £1.1M ($1.5M) in grant funding following a successful application to the prestigious Innovate UK Biomedical Catalyst (BMC) competition (Press release, Grey Wolf Therapeutics, MAY 18, 2021, View Source [SID1234580182]). Peter Joyce, CEO of Grey Wolf, noted that the company was "Delighted to receive this grant from Innovate UK, which will allow Grey Wolf to work with The University of Oxford ("Oxford") and the University of Southampton ("Southampton") to expand the ERAP1 program and accelerate the company’s path through clinical development".

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Over the past decade, immunotherapies have begun to transform cancer treatments, with significant impacts on clinical outcomes and survival rates. However, it has since become apparent that only a small subset of cancers and patient groups respond to these therapies. Clinical data suggests that one of the reasons behind this is due to low neoantigen expression at the cell surface.

In response to the urgent need for new therapeutics, Grey Wolf are developing small molecule inhibitors that target ERAP1 and ERAP2; two key enzymes in the antigen presentation pathway. Rather than targeting the immune system, this approach aims to directly alter the tumour cells to improve neoantigen expression and therefore, immune visibility.

The ERAP enzymes are responsible for trimming peptides prior to loading onto Major Histocompatibility Complex (MHC) Class I and presentation at the cell surface. These enzymes often over- or under-trim peptides in the endoplasmic reticulum, leading to a potential loss of immunogenic peptides and neoantigen presentation. Grey Wolf ERAP1 inhibitors significantly alter the neoantigen presentation on the surface of tumour cells, leading to greater recognition and destruction by the immune system.

The investment awarded through Innovate UK will be focused on identifying patient subgroups where ERAP1 inhibition could be particularly efficacious based on either cancer type or genetic background, thus providing the foundation for future breakthrough designations and accelerated approval. Collaborating with the world leading capabilities of Nicola Ternette’s group in Oxford and Edd James’ group at the Centre for Cancer Immunology in Southampton will be vital to the success of the project. The Ternette Lab are recognised leaders in the field of immunopeptidomics and the study of antigen presentation by MHC. Nicola Ternette and her team will use the technique to characterise neoantigens that are expressed following ERAP1 inhibition. As experts in ERAP biology, the James lab will examine the mechanistic effects of ERAP1 inhibition on T cell and tumour biology using a number of bespoke pharmacological models.

Peter Joyce, CEO, Grey Wolf Therapeutics: "We are delighted to have been awarded this highly competitive funding from Innovate UK which will enable us to accelerate the development of our ERAP1 inhibitor program and broaden the potential utility of this first-in-class therapy for patients. The award allows Grey Wolf to also build on our strong associations with two world class research groups at the University of Oxford and Southampton and represents a great opportunity to demonstrate the UKs leading role in global biotechnology."

Assoc. Prof. Nicola Ternette, The University of Oxford: "The funding from Innovate UKwill enable us to use Grey Wolf’s highly selective and potent ERAP1 inhibitors to mine their effects across the immunopeptidomes of different cancer types and genetic backgrounds. The truly exciting aspect of our research here will be gaining a fundamental understanding of the biology that can directly translate into clinical development of the therapy."

Prof. Edd James, University of Southampton: "We’re excited to build on our collaboration with Grey Wolf through this grant and apply our unique understanding of ERAP1 biology to exploit the translational potential of this approach."

On May 18, 2021 Epigenomics AG (Frankfurt Prime Standard: ECX, OTCQX: EPGNY, the "Company") reported that it has fully placed the new shares from the capital increase resolved on April 27, 2021 (Press release, Epigenomics, MAY 18, 2021, View Source [SID1234580181]). The offer was oversubscribed multiple times. Accordingly, the Company’s share capital will be increased from currently EUR 9,852,690.00 by EUR 1,970,537.00 to EUR 11,823,227.00 by 1,970,537 new registered shares of the Company against cash contributions.

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The gross proceeds from the capital increase amount to approximately EUR 2.2 million.

The capital increase needs to be registered in the commercial register, which the Executive Board will apply for shortly. The inclusion of the new shares under the Company’s existing listing (ISIN DE000A3H2184) of the remaining shares is currently expected to take place at or around May 26, 2021.

On May 18, 2021 Calliditas Therapeutics reported that Summary of Q1 2021 January 1 – March 31, 2021 (Press release, Calliditas Therapeutics, MAY 18, 2021, View Source [SID1234580180])

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No net sales for the three months ended March 31, 2021 were recognized. For the three months ended March 31, 2020 net sales amounted to SEK 0.5 million.
Operating loss amounted to SEK 150.8 million and SEK 72.3 million for the three months ended March 31, 2021 and 2020, respectively.
Loss before income tax amounted to SEK 136.2 million and SEK 63.7 million for the three months ended March 31, 2021 and 2020, respectively.
Loss per share before and after dilution amounted to SEK 2.51 and SEK 1.65, for the three months ended March 31, 2021 and 2020, respectively.
Cash amounted to SEK 867.3 million and SEK 728.6 million as of March 31, 2021 and 2020, respectively.
Significant events during Q1 2021, in summary
In January 2021, Calliditas announced a positive readout of the Phase 1 study with setanaxib, which enables clinical trials with higher dosing levels.
In January 2021, Calliditas shared the clinical development plan for setanaxib, including planned trials in Primary biliary cholangitis (PBC) and head and neck cancer, and additional data from Part A of NeflgArd study at its R&D Day.
In March 2021, Calliditas announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Nefecon in patients with primary IgA Nephropathy (IgAN).
Significant events after the end of reporting period, in summary
In April 2021, Calliditas was granted accelerated assessment procedure by the European Medicine Agency’s (EMA) Committee for Human Medicinal Products (CHMP) reducing the maximum timeframe for review of the application for marketing authorization. If approved, Nefecon could be available to patients in Europe in first half of 2022.
In April 2021, Calliditas announced that the FDA accepted the submission and granted Priority Review for the NDA for Nefecon. The FDA have set a Prescription Drug User Fee Act (PDUFA) goal date of September 15, 2021. Subject to approval, this would enable commercialization of Nefecon in the US in Q4, 2021.
Investor presentation May 18, 14:30 CET
Audio cast with teleconference, Q1 2021, May 18, 2021, 14:30 (Europe/Stockholm)

Webcast: View Source

Teleconference: SE: +46850558366 UK: + 443333009271 US: 18335268381

Financial calendar
Interim Report for the period January 1 – June 30, 2021 August 19, 2021

Interim Report for the period January 1 – September 30, 2021 November 18, 2021

Year-end Report for the period January 1 – December 31, 2021 February 24, 2022

On May 18, 2021 Almac Discovery, the independent research driven drug discovery company dedicated to the development of novel and innovative therapeutics, reported that it is delighted to share ground-breaking new research which has identified nearly thousands of gene pairs that represent ‘Achilles Heels’ or cancer vulnerabilities in analysis of more than 700 different cancer models (Press release, Almac, MAY 18, 2021, View Source [SID1234580179]). In the future, this could lead to new ways to stop cancer cells in their tracks by using existing drugs, as well as proposing new targets for drug development. These drugs could even be used to combat cancers that do not respond to the current standard treatments.

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The research was led by Mark Wappett, Head of Bioinformatics at Almac Discovery and Honorary Lecturer at Queen’s University Belfast, in collaboration with the Overton and McDade groups in the Patrick G Johnston Centre for Cancer Research (PGJCCR) at Queen’s University Belfast and the Department of Biochemistry at Vanderbilt University, USA.

Pivotal to the research is a completely new computational method for discovering cancer synthetic lethality from clustered regularly interspaced short palindromic repeats (CRISPR) and gene expression data using a website called ‘SynLeGG’. This site analyses large and complex datasets to identify changes in genes that occur in certain cancers that may make them more addicted to other similar genes and therefore susceptible to treatments targeting these partners. It was published today (17 May) in the journal Nucleic Acids Research and can be accessed here.

Study lead Mark Wappett, Almac Discovery, commented "Synthetic Lethality with Genetics and Genomics, or SynLeGG for short, provides the wider scientific community access to key datasets generated by cutting edge molecular biology technologies, such as CRISPR, and a toolkit with which to analyse this data. Ultimately we hope that by increasing the reach of this data we can expedite more targeted and effective cancer treatments."

Dr Ian Overton, Senior Lecturer at the PGJCCR explains: "Understanding the molecular fingerprints of cancer can pinpoint ways to target drugs precisely to those patients where they will be most effective. Our work makes a step towards more effective and personalised cancer treatments, ultimately saving lives.

"We make our results available on the SynLeGG web server, opening a window to share these rich resources with researchers across the scientific community – in order to accelerate progress in cancer research."

Dr Simon McDade, Senior Lecturer from the PGJCCR said: "We anticipate that this resource will seed detailed investigation of a number of specific vulnerabilities we have identified, ultimately identifying novel treatment strategies that will translate into significant benefits for cancer patients in the long term."