Abeona Therapeutics Inc.

Good day, and welcome to the Abiona Therapeutics First Quarter 2024 Conference Call. At this time, all participants are on a listen-only mode. After management's prepared remarks, there will be a question-and-answer session. I would now like to turn the call over to your host, Greg Chin, Vice President of Investor Relations and Corporate Communications at Abiona. Please go ahead.

Thank you, Kelly. Good morning, and thank you for joining us. on our first quarter 2024 conference call. During this call, we will refer to the press release issued this morning announcing the first quarter results, which is available on our corporate website at www.abionatherapeutics.com. I would like to note that remarks made during today's call may contain projections and forward-looking statements. 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 subject to change, and actual results may differ materially from those expressed or implied in the forward-looking statements. Various factors that could cause actual results to differ include, but are not limited to, those identified under the risk factor section in our Form 10-K and periodic reports filed with the SEC. These documents are available on our website at www.abionatherapeutics.com. On the call today with prepared remarks are Dr. Vish Seshadri, Chief Executive Officer Dr. Mada Vizantafada, Chief Commercial Officer and Head of Business Development, and Joe Vizano, Chief Financial Officer. Joining us for the Q&A session as well will be Dr. Brian Keveny, our Chief Technical Officer. And with that, I'll now turn the call over to Vish Seshadri to lead us off. Vish?

Thank you, Greg. We appreciate everybody joining the call this morning. In order to continue to progress our PZ-Cell program well beyond anticipated regulatory milestones, and through commercial launch, Abiona must be well capitalized. The leadership team has recently been focused on addressing our funding needs. In early May, we completed an underwritten offering with institutional investors that raised $75 million in gross proceeds. Participants in the offering included existing and new investors who are some of the most respected blue chip institutional healthcare investors. This was a significant development for Abiona and we're grateful to our investors who have demonstrated their support. Importantly, financing not only has extended our cash runway into 2026, which is well beyond anticipated significant regulatory milestones and commercial launch of PzCell, but we believe it further validates the great potential for PzCell. We now stay resolutely focused on working with the FDA to address the CMC deficiencies noted in the CRL, and making the BLA resubmission toward bringing PZ-Cell to ordered patients as soon as possible. As a brief recap of our regulatory update call in late April, we received a CRL from the FDA based on the need for additional CMC information before the PZ-Cell BLA can be approved. In general, the information needed to satisfy the CMC requests pertains to validation requirements for certain manufacturing and release testing methods, The CRL did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA, and the FDA did not request any new clinical trials or clinical data to support the approval of PZ Cell. Since the update call, we have already made progress towards addressing the CMC deficiencies noted in the CRL. We have now completed the matrix interference study to validate the replication-competent retrovirus, or RCR, assay, We believe the outcome of this study will satisfy the agency's ask for RCA or assay validation based on our discussion with the FDA in late March. We have also completed the container closure integrity testing for the RVV and the validation reports are in place. We continue to interact with the FDA through informal meetings to gain the agency's alignment on our approach, especially on topics where interpretation of existing guidance in the context of our therapy is required. Looking ahead to the coming weeks and months, we're working on completing deliverables that would address all remaining deficiencies noted in the CRL to enable resubmission of the BLA. We anticipate completion of all these work streams in the late Q2 to early Q3 timeframe and anticipate completing the BLA resubmission in the second half of 2024. We do plan to request a Type A meeting with the FDA in early Q3. The goal of this meeting would be to gain alignment with the agency on the sufficiency of our data to address the outstanding, substantive, and critical CMC items. As always, we will continue to be transparent with the outcomes of the meeting after completion. I'll now turn the call over to our Chief Commercial Officer, Madhav Vasanthavada, to provide an update on our commercialization readiness activities. Thank you, Vish.