Precigen, Inc.

Good day and welcome to Precision Fourth Quarter and Year-End 2020 Financial Results Conference Call. All participants will be in listen-only mode. Should you need assistance, please signal a conference specialist by pressing the star key followed by zero. After today's presentation, there will be an opportunity to ask questions. To ask a question, you may press star then one on your touchtone phone. If you would like to withdraw your question, please press star, then two. Please note this event is being recorded. I would now like to turn the conference over to Steve Harrison. Please go ahead.

Thank you, Operator, and thank you all for joining us today. With me are Dr. Helen Sabzavary, President and CEO of Precision, and Tom Samuelson, Head of Financial Strategy. Helen will provide an update on our pipeline and technologies after which Tom will review our fourth quarter 2020 financial results. Following the prepared remarks, we will open the call to Q&A. Before we begin, let's briefly review our forward-looking statements. During today's call, we will make various forward-looking statements. Investors are cautioned that these statements are based on current expectations and are subject to risks and uncertainties that could cause actual results or outcomes to differ materially from those indicated by our forward-looking statements. Please read the State Farber statement contained in this presentation, as well as the risk factors contained in Presagen's most recent SEC filings, for a more complete discussion of these risks and uncertainties. I will now turn the call over to Dr. Zabzavary.

Thanks, Steve, and thank you to everyone taking the time to listen in today. I hope this call finds all of our stakeholders and their families safe and healthy. It is great to connect with all of you today to review our 2020 highlights and financial results. Precision continues to execute and work to achieve our mission of delivering novel treatment options to patients with unmet medical needs. I'm very proud of our team's achievement during 2020. It truly was a transformative year for us in several ways. First and foremost, we implemented rigorous health and safety measures to ensure research and clinical trial continuity. As a result, we were able to meet all of our stated clinical milestones during a very challenging year. Second, the company successfully transitioned to a health-focused company. Throughout the year, we made major strides in optimizing our operations, to become more nimble and focused, advancing our exciting portfolio of innovative therapeutic candidates. Third, we fortified our financial position through a capital raise and a fiscal discipline. Tom will provide additional details on our quarterly numbers later in the call. Finally, we made meaningful advances across our portfolio completing important clinical and manufacturing milestones that position us for an exciting 2021. Now, moving to a recap of our portfolio progress. The first, our transformative UltraCar T platform. As we started the year in 2020, our main goal for this platform was to validate overnight decentralized manufacturing and demonstrate in vivo expansion and persistence of CAR T cells. Based on the data we presented during our update call in December, we demonstrated our ability to successfully manufacture our ultra-CAR T cells across multiple sites and have shown robust expansion and persistence in vivo even without the need for lymphodepletion in both the solid and hematological tumor settings. Furthermore, we demonstrated encouraging clinical activity in our lowest dose cohorts and excellent safety profile with no dose limiting or neurotoxicities to date in either trial. In addition, We aimed at advancing the platform towards becoming a scalable and commercially viable therapeutic option. The introduction of Ultraoperator, our semi-closed, high-throughput system, is intended to be a viable scale-up and commercialization solution for decentralized ultra-car team manufacturing. Ultraoperator includes hardware and software solutions and potentially represents a major advancement over current electroporation devices by significantly reducing the process time and contamination risk. This system was cleared for clinical manufacturing use by FDA, and we have started dosing patients with ultracar T cells manufacturing using ultraproider for both our hematological and solid tumor CAR T trials. As for the trials, PRGN-3005 targeting unshed MUX16, a phase 1, 1B trial in patients with ovarian cancer. Initial data presented in December for PRGN-3005 ultracar T cells showed encouraging expansion and persistence after low-dose IP infusion without lymphodepletion. In addition, 50% of patients treated, three out of six, at either dose level one or dose level two with low doses between six and 21 million ultracar T cells with no lymphodepletion experienced regression in total target tumor burden. And two out of these six patients achieved a stable disease according to research criteria at their restaging evaluation. Dose expansion continues in the IP arm, and we are looking forward to initiating the expansion phase in the second half of 2021. We have now received clearance from the FDA to initiate dosing in IV arm of PRGN 3005 phase one trial concurrently with IP arm. And I'm happy to announce that we have successfully dosed the first patient in the IV arm. And for PRGN 3006, we announced encouraging data from the ongoing Phase 1-1B clinical study in patients with relapsed or refractory acute myeloid leukemia, AML, and higher risk myelodysplastic syndrome at the ASH annual meeting in December. As presented at ASH by our PI, Dr. Solomon, PRGN3006 cells showed encouraging expansion and persistence in peripheral blood after low-dose infusion. Between 1 and 29 million ultracar T cells in both the lymphodepletion and non-lymphodepletion cohort, as well as the ability to traffic, expand, and persist in bone marrow. Furthermore, PRGM-3006 treatment indicated clinical activity as evidenced by reduction in AML tumor blast levels. The potential strength of this platform was highlighted at ASH 2020 case study of patients with multiple prior treatment failures where ultracar T cells persisted for more than seven months after a very low dose only a 24 million total ultracar T cell infusion without prior lymphodepletion. This patient showed a decline in blast levels in blood and bone marrow concomitant with ultracar T expansion and persistent and had a stable disease. For our update in December, a patient who received PRGN 3006 at dose level 1 with approximately 9 million cells with lymphodepletion, had an objective response and achieved CRI per ELN criteria. I am now pleased to report that this patient has subsequently received a hematopoietic stem cell transplant and is doing well, which according to Dr. Solomon is very encouraging outcomes. We are simultaneously enrolling in the dose escalation phase of both the lympho and non-lympho depletion arms and are on track to initiate the expansion phase in the second half of 2021. We look forward to our investigators providing clinical updates on these ongoing trials at upcoming medical conferences. As we continue to provide clinical validation of the Ultracar-T platform, we believe we are on the way to creating a tool for precision medicine. Our goal is to develop and validate a library of non-viral plasmids to target tumor-associated antigens. Based on the patient's cancer indication and biomarker profile, one or more non-viral plasmids would be selected from the library to build a personalized ultracar T treatment. After initial treatment, this approach has the potential to allow for redosing of the ultracar T targeting the same or a new tumor-associated antigen based on the treatment response and the changes in antigen expression of the patient's tumors. We believe this is the only platform that has the flexibility to generate multiple autologous ultracar T's for patients. Redosing, if needed, at the same time has the potential to do so at the lower cost. This is a very exciting prospect for advancement of personalized medicine. Now, let's start to adenoverse immunotherapy platform, which is based on our gorilla adenovector library. It has a high payload capacity and provides the ability to redose both advantages to competing approaches. Now moving to our first-in-class PRGN 2009, which is in a phase one, phase two trial, to treat HPV-positive solid tumors under a cradle with the NCI. In August, we announced the first patient was dosed, and in January, we announced the completion of enrollment in a phase one monotherapy dose escalation arm of the trial. All six patients enrolled in the phase one monotherapy arm have received multiple PRGN2009 doses, and to date, the repeated administration has been well-tolerated with no dose-limiting toxicities. Preliminary correlative analysis showed that 100%, three out of three of patients treated at dose level one demonstrated an increase in HPV16 and or HPV18-specific T cell responses post-PRGN 2009 administration. Furthermore, repeat administration of PRGN 2009 resulted in an increase in magnitude and breadth of HPV-specific immune response. This highlights the potential differentiation of adenovirus platform compared to existing treatments. We were very encouraged with the preliminary findings and look forward to providing further updates in the coming months. We have initiated dosing in a combo portion of the trial, which includes PRGN 2009 and M7824. We anticipate an interim phase one readout in the second half of 2021, and to initiate the phase two portion of the trial in the same timeframe. Now, moving on to PRGN 2012, which is our first adenovirus program targeting infectious disease. PRGN 2012 is an investigational off-the-shelf immunotherapy for the treatment of recurrent respiratory papillomatosis, or RRP. In January 2021, We announced that the FDA had cleared the IMD application to initiate the Phase I clinical trial, and we expect initial dosing first half of 2021. Finally, in January, we presented preclinical data for PRGN 2013, our adenovirus therapy for hepatitis B HBV infection. This therapy incorporates novel HPV antigen design in a gorilla adenovactor. The data showed that PRGM 2013 induced superior cytotoxic T cell responses against more HPV epitopes in mice than a competitor vaccine candidate and decreased plasma hepatitis B surface antigen levels, the key marker for chronic HPV infection. We are very encouraged by this data and the opportunity to advance this program toward the clinic in infectious disease. Now moving to a Phase 1b-2a trial of AG019, which is based on our Actobiotics platform. We have completed enrollment in AG019 for treatment of T1D in the phase 2A combo arm of the trial. In December, we provided positive data from the phase 1B-2A portion of the trial, showing an encouraging trend in insulin C peptide levels and ability to induce antigen-specific immune modulation following only one treatment oral cycle of AG019 as a monotherapy or combination. We expect to provide 12-month follow-up data in the adult arm in the Phase IIa portion during the first half of 2021 and in the adolescent arm in the Phase IIa portion during the second half of 2021. We are engaged with the regulatory agencies for the next phase trial design with the goal to rapidly advance this program toward the BLA. Finally, for INXN-4001, our phase one trial for heart failure. We have completed 12-month follow-up in the phase one study with a solid safety profile and encouraging clinical activity. And as we speak, we are actively involved in partnering discussions. Now I will turn the call over to Tom for an overview of our financial results. Tom?