At the American Society of Hematology (ASH) Annual Meeting in December 2021, AlloVir reported positive preliminary data from the open-label Phase 2 study assessing the safety and efficacy of posoleucel for the prevention of multiple viruses in high-risk allogeneic hematopoietic cell transplant (allo-HCT) patients. The preliminary data set demonstrated a substantial reduction in the expected rate of clinically significant viral infections or diseases and facilitates an accelerated path to Phase 3 development, which has been agreed to in principle by the U.S. Food and Drug Administration (FDA). The Phase 3 registrational study is expected to initiate in the first half of 2022.

In December 2021, AlloVir initiated a global Phase 3 registrational study of posoleucel for the treatment of AdV infections in adult and pediatric allo-HCT patients. The study is the second registrational trial of posoleucel to initiate. AdV-associated viral disease is among the leading causes of life-threatening complications following allo-HCT, often involving multiple vital organs, including the brain, lungs and liver, and occurs in 32% of pediatric and 6% of adult allo-HSCT patients.

The FDA granted posoleucel Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of AdV infections, based on Phase 2 data and in recognition of the potential for posoleucel to address an unmet medical need. The FDA previously granted RMAT and orphan drug designations (ODD) to posoleucel for the treatment of hemorrhagic cystitis (HC) caused by BKV in adults and children following allo-HCT. The European Medicines Agency has granted posoleucel PRIority Medicines (PRIME) designation for the treatment of serious infections with AdV, BKV, CMV, EBV and HHV-6, and Orphan Medicinal Product (OMP) designation as a potential treatment of viral diseases and infections in patients undergoing HCT.

Following a review at the end of last year of initial, blinded safety data from the ongoing posoleucel proof-of-concept (POC) study for BK viremia treatment in adult kidney transplant recipients, the study has been amended to begin enrolling patients with higher viral loads. BKV is detected in up to 20% of kidney transplant patients and can lead to graft loss and a return to end-stage renal disease and dialysis. There are no approved or effective BKV treatments.

A Phase 1/2 POC study of ALVR106, an investigational multi-respiratory VST therapy designed to target infections and diseases caused by human metapneumovirus, influenza, parainfluenza virus and respiratory syncytial virus in autologous and allo-HCT recipients initiated in December 2021.

Data on ALVR109, a SARS-CoV-2 targeted VST available via compassionate use, further validated the potential of AlloVir’s VSTs for the treatment of respiratory viruses. A case report detailing the use of ALVR109 in an immunocompromised heart transplant patient with severe refractory COVID-19 was published in the American Journal of Transplantation in December 2021; rapid clinical and virologic improvement occurred following the administration of ALVR109. Recent preclinical testing has demonstrated that ALVR109 retains potent antiviral activity against a broad range of variant strains including Omicron.

AlloVir established regional clinical distribution capabilities for its VSTs in Europe and Asia Pacific, thereby opening these geographies to participation in its clinical trials.

AlloVir conducted a technology transfer of its VST manufacturing platform to ElevateBio’s BaseCamp facility. In doing so, AlloVir added capacity and redundancy to its external manufacturing network of contract development and manufacturing organizations.

Global enrollment is ongoing in registrational studies of posoleucel for the treatment of virus-associated HC and for the treatment of AdV infections, both in adult and pediatric allo-HCT patients. Enrollment of the HC trial is expected to be complete in the first half of 2023.

A Phase 3 registrational trial for multi-virus prevention, using the dose, dosing interval and key endpoints from the Phase 2 study, is expected to initiate in the first half of this year, pending FDA review of the final study protocol. Multi-virus prevention has the potential to be clinically transformative, while also expanding the addressable patient population for posoleucel.

The company expects to report initial data from the ongoing posoleucel POC study for BKV treatment in KT recipients at a scientific meeting in the first half of 2022. The study is the first to evaluate the use of posoleucel in solid organ transplant (SOT) recipients; results from this study will also inform plans for a multi-virus prevention study in SOT patients. SOT represents a large opportunity to significantly expand the addressable patient population for posoleucel.

Final results, including persistence data, from the posoleucel Phase 2 study for multi-virus prevention are expected to be presented at a scientific meeting in the second half of 2022.

Enrollment in the Phase 1/2 POC study of ALVR106 for the treatment of multiple respiratory viral infections is ongoing.

Preclinical and IND-enabling studies of ALVR107 to treat and cure hepatitis B will be completed in 2022 to support advancement into a POC study.

Research and development expenses were $120.7 million for the year ended December 31, 2021, compared with $49.7 million for the year ended December 31, 2020. The increase year-over-year is attributable to costs related to the development of the company’s product candidates, increased activity in outsourcing of manufacturing, and an increase in headcount and external consultants in support of research activities.

General and administrative expense was $49.1 million for the year ended December 31, 2021, compared with $21.6 million for the year ended December 31, 2020. The increase year-over-year was primarily attributable to increased headcount and professional fees for legal and accounting associated with operating as a public company.

Stock-based compensation expense was $44.0 million and $9.4 million for the years ended December 31, 2021, and 2020, respectively.

As of December 31, 2021, AlloVir had cash, cash equivalents and marketable securities of $248.1 million, compared with cash, cash equivalents and marketable securities of $356.3 million as of December 31, 2020.

For the year ended December 31, 2021, net loss was $172.0 million or $2.74 per share, compared with a net loss of $69.8 million or $2.59 per share for the year ended December 31, 2020.

For fiscal year 2022, AlloVir expects operating expenses to be in the range of $130 million to $145 million, excluding non-cash stock compensation expenses.

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