Full Press Release Details
Type: Q1 2021 Earnings Call
Abeona Therapeutics, Inc.
W. Gin - Vice President-Investor Relations & Corporate Communications
Amoroso - President & Chief Executive Officer
G. Carr - Chief Accounting Officer & Principal Financial Officer
day, ladies and gentlemen and welcome to the Abeona First Quarter Earnings Call. At this time, all participants are on a listen-only
mode. We will open up the floor for questions and comments after the presentation. It is now my pleasure to turn the floor over to your
host Gregory Gin, Vice President of investor Relations and Corporate Communications at Abeona. Please go ahead.
you, Holly. Good morning, everyone, and welcome and thank you for joining us on our first quarter 2021 conference call. Press release
announcing the first quarter results and recent operational progress is available on our website at www.abeonatherapeutics.com. On the
call today with prepared remarks are Michael Amoroso, Chief Executive Officer of Abeona; and Ed Carr, Chief Accounting Officer. After
the prepared remarks, we will host a Q&A session where we will be joined by Dr. Juan Ruiz, Vice President of Abeona who heads up
Clinical Development for our MPS programs; and Dr. Brian Kevany, Lead Research Scientist working on our preclinical eye programs.
we start, I will review our Safe Harbor statement. Remarks made during today's call may contain
and forward-looking statements regarding future events. Forward-looking statements are made pursuant to the Safe Harbor provisions of
the federal securities laws. These forward-looking statements are based on current expectations and are subject to change and actual
results may differ materially from those expressed or implied in the forward-looking statements.
factors that could cause actual results to differ include, but are not limited to, those identified under the section entitled Risk Factors
in the company's Annual Report on Form 10-K and quarterly reports on Form 10-Q filed by the company with the SEC. These documents are
available on our website at www.abeonatherapeutics.com.
with that said, I will now turn the call over to Michael. Michael?
you, Greg. Good morning to our investor community. We're excited to spend some time with you this morning. Thank you for joining us.
As I approach the midpoint of my first 100 days in the job as CEO, I wanted to stop by reviewing three key strategic priorities for Abeona
priority, being to bolster the relevant operational experience on our management team and board. Making sure we have the right coaches
in place, the right experience to meet our goals going forward.
delivering operational excellence, both timely and fiscally disciplined, to further advance our in-clinic programs toward meaningful
milestones for patients in need.
third, to further prioritize, execute, and advance our preclinical eye programs toward the clinic. We've made substantial progress on
these priorities, and we're off to a fast start in 2021. I want to thank the team for their efforts.
priority one, continuing forward under the right leadership. We continue to focus on building the right talent and experience on our
team which positions us well to execute against operational goals. I'm very excited just this morning to have announced the appointment
of Dr. Vishwas Seshadri, a Senior Vice President, and our new Head of Research and Clinical Development.
as he goes by, brings more than 20 years of experience across academia, followed by various senior and executive level leadership roles
within the life sciences industry, overseeing product development, regulatory strategy and teams for submissions, and commercialization
for novel therapies, including most recently, personalized autologous cell therapies in the CAR T space for Celgene and BMS.
has significant experience across early and late-stage development from first patient in all the way through successful commercial launches.
He understands the tradeoff decisions and necessary discipline for evidence-based drug development.
is also a proven coach in developing people across functions. Through his project leadership in working with clinical, regulatory, medical
and commercial teams at Celgene, we're thrilled to have him join our team in early June and we look forward to introducing Vish to you
on future quarterly calls. Welcome Vish to the Abeona team.
in the first quarter, we have strengthened our Board of Directors with the appointment of four new independent members as well as myself.
The new members have experience sitting on boards of public and private companies and bring relevant operational leadership experience
within the life science industry, including areas such as clinical development, manufacturing of cell and gene therapy products, and
corporate and financial compliance. The new members were selected based on their relevant and diverse experience, making them the right
partners to the current management team in our pursuit of both near and long-term objectives.
like to take a minute to review the new members and their critical experiences for Abeona. First, Dr. Leila Alland, a pediatric hematologist,
oncologist and physician scientist. Leila spent over 20 years in the biopharmaceutical industry focusing on bringing novel therapies
to patients with rare diseases, and is currently the Chief Medical Officer at PMV Pharmaceuticals. We're delighted to have Leila and
she'll be partnering closely with Vish's team.
Don Wuchterl. Having over 29 years of experience in the life science industry with senior roles in operations and CMC. Don is currently
the Chief Manufacturing Officer at T-knife Therapeutics. He has significant experience building and leading CGMP manufacturing organizations
and facilities so Don will provide essential feedback and guidance to our teams in Cleveland. Welcome, Don. We're really excited to have
is Faith Charles, a corporate transaction and security partner at Thompson Hine with over 30 years of legal experience and lead Thompson
Hine's life science practice providing valuable insights to companies and capital markets, corporate governance, and strategic development
such as M&A, licensing transactions, and strategic collaborations. Welcome to Faith.
last but never least, Mark Alvino. Mark has provided leadership and experience in the areas of financial management and business strategy
as a member of the Board of Directors of multiple life science companies including previously Abeona. Mark knows Abeona, our people,
and our products well and his capital market experience will be invaluable as well as his longitudinal perspective on our organization.
We're excited to have Mark back onboard.
I wanted to express my excitement to announce the addition of these respected leaders, experienced both to the management team and our
board. I am confident that we have the right collective leadership in place that will help us to accomplish our objectives at this critical
point in Abeona's lifecycle. Excuse me a moment.
me take an additional minute to comment on our board's aimed to enhance and evolve our governance. At our annual meeting of stockholders
to be held tomorrow, our board recommends that stockholders vote for Proposal 2 to approve the amendment of our restated certificate
of incorporation to declassify the board and eliminate three-year terms for directors in favor of one-year terms. Thus, further aligning
with investor interests and increasing accountabilities of our directors to shareholders.
board believes that annual director elections are in keeping with sound corporate governance practices and promotes additional accountability
to shareholders. If you have questions or need help voting for this very important proposal, please contact Greg Gin, our Head of Investor
moving on to priority 2, advancing our clinical programs to our key milestones for patients. I'll start with an update on EB-101, our
lead pivotal program for recessive dystrophic epidermolysis bullosa or as we refer to as RDEB. Regarding enrollment in EB-101 VITAL trial,
we remain on track for activating a second study site in Northeast to complement Stanford on the West Coast in the second half of 2021.
Patient physician interest in the study continues to be high and we're observing a greater willingness to travel on what we believe is
the growing percentage of the population starting to receive vaccines for COVID-19.
as discussed our last call, a fifth patient had been biopsied for the start of treatment. This patient's cells yielded product that didn't
meet required release criteria as per the pivotal trial protocol. As a result, the clinical team and the patient had opted to re-biopsy
and begin a new cell therapy product generation ongoing. We are hopeful to treat this patient in the coming weeks.
in parallel, additional patients have been identified and are being pre-screened to determine their eligibility for the VITAL trial entry
criteria. As a reminder, the target for this pivotal trial is the treatment of approximately 35 large chronic wounds across 10 to 15
the execution of our comparability plan is ongoing through our tech ops teams in Cleveland as well as ongoing communication with the
FDA. Thus far in 2021 overall, progress for the EB-101 program has been significant and we continue toward our goal of completing enrollment
in the VITAL study by year end. Thank you to the teams and our patients.
us next turned our attention to our adeno-associated virus, AAV-based gene therapy programs starting with our ABO-102 program in Sanfilippo
Syndrome Type A or MPS IIIA. We are preparing for the FDA Type B meeting scheduled for next month in June. As a reminder, we plan to
discuss the data to-date from the ABO-102 Transpher A study and next steps for a potential BLA path with the FDA.
intend to discuss with the FDA whether the Transpher A data set could serve as the basis for BLA submission with natural history data
as a viable control arm. We believe historical controls are critically important for MPS treatment and drug development globally in this
disease as these children are being irreversibly impaired - irreversibly impaired and it's not feasible to give them a placebo.
are excited and hopeful to review the data with the FDA at our upcoming meeting and partner on a viable plan forward for patients. Also,
the FDA feedback from our upcoming meeting will continue to guide and enhance our development plans and pathway to marketing authorization
to our third in-clinic program EB-101, the Transpher B study in Sanfilippo Syndrome Type B or MPS IIIB. Product stability testing discussed
in the last call for the clinical product from Nationwide Children's Hospital is now ongoing. To expedite timing, we brought some assay
testing in-house. I want to thank our teams in Cleveland for their adaptability there.
completion of stability testing, we'll assess our options forward for treating the additional patients remaining for Transpher B. During
the first quarter in recent months, we also shared important clinical updates from both our Transpher A and Transpher B studies at multiple
medical meetings and congresses. This is highlighted by positive data at the 17th Annual WORLDSymposium in February 2021, showing neurocognitive
development of young MPS IIIA patients was preserved up to three years following treatment with ABO-102.
addition, presented results from the Transpher B study continued to show signals of biologic effect with reduction in disease specific
biomarkers in cerebrospinal fluid plasma and urine and reduction in liver volumes. We look forward to ongoing and additional updates
of neurocognitive milestones.
on to our third priority. Prioritizing and advancing our preclinical programs. We're focusing resources on ophthalmic indications within