Enzyvant Announces FDA Acceptance of Biologics License Application (BLA) and Priority Review Status for RVT-802, a Novel Investigational Tissue-Based Regenerative Therapy for Pediatric Congenital Athymia

—RVT-802, a one-time therapy, leverages Enzyvant’s T cell generation
platform designed to treat profound immunodeficiencies—

—Left untreated, congenital athymia is uniformly fatal, with death
typically occurring in first 24 months of life—

—Company to present at Roivant Pipeline Day in New York City on June
6, 2019—

CAMBRIDGE, Mass. & BASEL, Switzerland–(BUSINESS WIRE)–Enzyvant, a biopharmaceutical company focused on developing and
commercializing transformative therapies for patients with rare, often
fatal conditions, today announced that the U.S. Food and Drug
Administration (FDA) has accepted for filing its Biologics License
Application (BLA) for RVT-802, a novel investigational tissue-based
regenerative therapy designed to treat pediatric congenital athymia, and
granted Priority Review. Congenital athymia is a rare and deadly
condition associated with complete DiGeorge Anomaly (cDGA), CHARGE
syndrome, and FOXN1 deficiency. At this time, the FDA is not planning to
hold an Advisory Committee meeting to discuss the application, and
Enzyvant anticipates a regulatory decision in December 2019.

Children with congenital athymia are born without a thymus, resulting in
a severe immunodeficiency due to the inability to produce normally
functioning T cells, which defend against infection and regulate
essential processes in the immune system. Approximately 20 infants are
born each year in the United States with congenital athymia, which is
fatal if untreated. Death typically occurs in the first 24 months of
life due to susceptibility to infection. Currently, there are no
FDA-approved therapies for this condition. RVT-802 stimulates and
facilitates the body’s production of naive, immunocompetent T cells,
with the goal of bolstering the immune system and restoring the body’s
ability to fight infection. Investigational RVT-802 is designed to be
administered as a single treatment.

“We are proud to be advancing RVT-802, a regenerative therapy that
embodies bold, transformative science. The intense urgency to treat
infants and young children who would otherwise succumb to congenital
athymia drew us to forge a partnership with Duke University and
continues to motivate us to advance toward a potential approval with
focus and speed,” said Rachelle Jacques, Chief Executive Officer of
Enzyvant. “The long-term data for RVT-802 as a one-time treatment
reinforces the potentially life-saving value and durable impact of this
therapy. We are committed to working collaboratively with payers to
establish a value-based reimbursement model that accelerates access for
patients.”

The BLA filing for RVT-802 included clinical data that demonstrated
long-term durability of treatment with RVT-802. At the time of the BLA
filing, a total of 93 patients received RVT-802 across multiple clinical
studies, including 85 patients who met the criteria for inclusion in the
efficacy analysis. The Kaplan-Meier estimates of survival [95%
confidence interval] at year one and year two post treatment were 76%
[66 – 84] and 75% [66 – 83], respectively. For patients surviving 12
months post-treatment, there was a 93% probability of surviving 10 years
post-treatment. During clinical development, the most commonly (≥ 5%)
reported RVT-802 related adverse events included thrombocytopenia (11%),
neutropenia (8%), pyrexia (5%), and proteinuria (5%).

“The journey of this therapy has involved the dedication and
contributions of so many and, most notably, the bravery of patients and
their families,” said Dr. Louise Markert, Professor of Pediatrics at
Duke University School of Medicine, whose pioneering work at Duke led to
the development of RVT-802. “It is gratifying to see this therapy
advance a significant step closer to a potential FDA approval. We are
hopeful we can look to a future of continuing to save children’s lives.”

“We congratulate the Enzyvant team on this important milestone, as well
as Dr. Markert and her colleagues at Duke for their remarkable
scientific accomplishments and dedication to athymic patients and their
families,” said Myrtle Potter, Vant Operating Chair at Roivant Pharma,
and Chair of Enzyvant’s Board of Directors. “We look forward to the
potential of RVT-802 becoming available as an approved regenerative
medicine to all families and patients who could benefit from it.”

Ms. Jacques will be presenting at Roivant Pipeline Day in New York City
tomorrow, June 6, at 4:20 p.m. ET. To request access to the webcast or
to learn more about Roivant Pipeline Day, please email [email protected].

About RVT-802

RVT-802 is a novel investigational tissue-based regenerative therapy
designed to treat primary immune deficiency resulting from pediatric
congenital athymia. In a healthy, functioning immune system, T cells
that start as stem cells in bone marrow become fully developed in the
thymus. RVT-802 is designed to replicate this process in the absence of
a thymus.

Derived from infant thymus tissue, RVT-802 is processed and cultured
prior to implantation into a patient’s quadricep muscle. The patient’s
bone marrow stem cells migrate to the implanted tissue product, where
they are trained to become naïve, immunocompetent T cells. With the
renewed ability to generate T cells, immune system function can be
restored.

RVT-802 has been granted Breakthrough Therapy, Regenerative Medicine
Advanced Therapy (RMAT), Rare Pediatric Disease, and Orphan Drug
designations by the FDA.

In 2016, Enzyvant entered into an exclusive worldwide licensing
agreement with Duke University to develop RVT-802. M. Louise Markert,
M.D., Ph.D., Professor of Pediatrics at Duke University School of
Medicine, has led research on the treatment of immunodeficiency in
patients with congenital athymia. The findings of Dr. Markert and her
research team have been published in the New England Journal of
Medicine as well as numerous other peer-reviewed scientific journals
and clinical publications.

About Enzyvant

Enzyvant, a wholly owned subsidiary of Roivant Sciences, is a
biotechnology company focused on developing transformative therapies for
patients with rare diseases. Enzyvant leverages the Roivant platform to
develop therapies that address high unmet medical needs while driving
greater efficiency in research, clinical development, and
commercialization. The FDA has accepted Enzyvant’s Biologics License
Application submission for RVT-802, a novel investigational tissue-based
regenerative therapy for the treatment of congenital athymia and granted
Priority Review. Enzyvant anticipates a regulatory decision in December
2019. The company is also preparing to initiate a clinical trial of
RVT-801, an investigational enzyme replacement therapy for the treatment
of Farber disease. For more information, please visit www.enzyvant.com.

About Roivant

Roivant aims to improve health by rapidly delivering innovative
medicines and technologies to patients. Roivant does this by building
Vants – nimble, entrepreneurial biotech and healthcare technology
companies with a unique approach to sourcing talent, aligning
incentives, and deploying technology to drive greater efficiency in R&D
and commercialization. For more information, please visit www.roivant.com.

About Roivant Pharma

Roivant Pharma is the biopharmaceutical business unit of Roivant
Sciences. Roivant Pharma is focused on end-to-end biopharmaceutical
company creation, launch, and oversight. Roivant Pharma companies
include Altavant, Aruvant, Axovant, Dermavant, Enzyvant, Genevant,
Immunovant, Metavant, Myovant, Respivant, Urovant, and Arbutus.

About Roivant Pipeline Day

Roivant Pipeline Day will be held on Thursday, June 6, 2019 in New York
City. The event will feature presentations and Q&A sessions from
executives across the Roivant family of companies highlighting new
clinical data and investments in technology. The event is scheduled to
begin at 1:00 p.m. ET and will continue until approximately 4:30 p.m.
ET. A live webcast will be available to interested parties. To request
access to the webcast or to learn more about the event, please email [email protected].

Contacts

Media:
Liz Melone
[email protected]