On May 10, 2021 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported financial results for the quarter ending March 31, 2021 and updates on the Company’s key pipeline developments, business operations, and upcoming milestones (Press release, Rocket Pharmaceuticals, MAY 10, 2021, View Source [SID1234579583]).

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"I am pleased that we began 2021 with sustained momentum across clinical, regulatory and manufacturing activities as reflected in our exciting PKD and LAD-I results, advancement of the pipeline toward regulatory submissions, and continued progress of our GMP manufacturing facility," said Gaurav Shah, M.D., Chief Executive Officer of Rocket. "New data readouts and designations from regulatory agencies continue to validate and reinforce the transformative potential of our gene therapies for patients suffering from rare diseases with no current drug treatments. We continued to strengthen our world class operations and capabilities as we added to our leadership team and fortified our balance sheet."

Dr. Shah continued, "We are encouraged by the positive low dose RP-A501 Phase 1 trial results in Danon Disease disclosed today. We anticipate a one quarter delay in enrollment to address FDA requests for risk mitigation methods in our protocol. We have successfully treated five patients in the low- and high-dose adult cohorts. Today, we announce that new longer-term low-dose data demonstrate durable expression and ongoing improvements in biomarkers, and evidence of a positive risk/benefit profile out to 18-months. In the two patients followed to 18-months, we see improvement in 6MWT in one patient and stabilization/improvement the other, and improvement in NYHA Class from II to I in one patient with stabilization in the other. These updated results increase our confidence in the low dose as a potentially viable dose for patients with Danon Disease. Safety is our top priority as we progress our gene therapy trials. We are diligently working with the agency prior to initiating our low-dose pediatric cohort, which we believe has high potential as a Phase 2 dose given the durable results noted in adult patients. "

Dr. Shah continued, "The balance of the year is shaping up to be a productive period, starting next week with updates from our FA, LAD-I, and PKD programs at ASGCT (Free ASGCT Whitepaper), followed by initial IMO data in the third quarter of 2021. We are focused on strong clinical and operational execution as we continue our development of total cures at the genetic level."

Key Pipeline and Operational Updates

RP-A501 Danon Disease program paused for additional risk mitigation. No new drug-related safety events have been observed in the low- or high-dose adult cohorts of the Phase 1 trial. The U.S. Food and Drug Administration (FDA) has requested the Company to pause patient dosing and modify the protocol and other supporting documents with revised guidelines for patient selection and management. All follow-up study activities will continue and no additional data are requested. Rocket is continuing its dialogue with the agency to ensure safety measures are updated and harmonized adequately and anticipates additional patient treatment by 3Q2021.
Low Dose (6.7×1013) RP-A501 Treatment Demonstrates Durable Expression and Improvements in Biomarkers. New Data Include:
Patient 1002: 78% IHC (revised from previous 67% due to improved technique), BNP continues to decrease to 200 (baseline 943)
Functional outcomes
Patient 1001: NYHA Class stable (II), 6-minute walk test (MWT) improved at 18-months
Patient 1002: NYHA Class Improved (II to I), 6MWT stable to modestly improved at 18 months
Patient 1005: NYHA Class Stable (II), 6MWT pending at 12 months
Higher Dose (1.1×1014) data will be updated in the fourth quarter
Rocket does not anticipate pursuing doses higher than 1.1×1014 moving forward.
Presented positive clinical updates from RP-L201 Leukocyte Adhesion Deficiency-I (LAD-I) program at the Clinical Immunology Society (CIS) Annual Meeting. The Phase 1/2 data presented in a poster at CIS 2021 are from four pediatric patients with severe LAD-I. RP-L201 was well tolerated with no safety issues reported with treatment or post-treatment. All four patients achieved hematopoietic reconstitution within 5-weeks and demonstrated CD18 expression substantially exceeding the 4-10% threshold associated with survival into adulthood. The first patient with 18-months follow up demonstrated durable CD18 expression of ~40%, peripheral blood vector copy number (VCN) levels of 1.2 at 12-months post-treatment and resolution of skin lesions with no new lesions. The second patient with 9-months of follow up demonstrated CD18 expression of ~28% and peripheral blood VCN levels of 0.75 at 6-months post-treatment with kinetics consistent with those of the first patient. The third and fourth patients demonstrated high CD18 expression of ~70% and ~51%, respectively at 3-months post treatment, and peripheral blood VCN kinetics consistent with those of the first two patients. A link to the full data disclosed is available here: View Source More comprehensive Phase 2 results will be presented at ASGCT (Free ASGCT Whitepaper).
Announced updated positive preliminary clinical data from Phase 1 trial of RP-L301 for the treatment of Pyruvate Kinase Deficiency (PKD). The updated preliminary Phase 1 RP-L301 data are from two patients that showed sustained safety and tolerability 6- and 3-months after treatment, respectively. The two patients demonstrated durable normalization of hemoglobin levels from an average baseline of ~7.4 grams (g)/deciliter (dL) to 13.9 g/dL at 6-months post treatment in the first patient and from a baseline of ~7.0 g/dL to 13.8 g/dL at 3-months post treatment in the second patient. The two patients both demonstrated significant improvements in bilirubin 6- and 3-months after treatment, which had been substantially elevated prior to study enrollment. The Phase 1 trial continues to enroll patients with longer-term data on track for the fourth quarter of 2021, with a near-term update at ASGCT (Free ASGCT Whitepaper).
Dosed first patient with RP-L401 in Infantile Malignant Osteopetrosis Phase I trial. Patient treatment has commenced in the Phase I trial of RP-L401, Rocket’s LVV-based gene therapy for infantile malignant osteopetrosis(IMO). The first patient is being treated at UCLA Children’s Hospital. With this significant milestone patients are now being treated in all five gene therapy programs across the company’s pipeline. Preliminary results are expected in the third quarter of this year.
Received regulatory designations for LAD-I program from the FDA and European Medicines Agency (EMA). The LAD-I program received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA and Priority Medicines (PRIME) designation from the EMA, completing the full complement of all U.S. and EU accelerated regulatory designations for the program.
Further strengthened Rocket leadership. In March 2021, Gayatri Rao, MD, JD was promoted to Senior Vice President, Chief Development Officer of Rocket’s LVV pipeline. Since joining Rocket from the FDA in 2018, Dr. Rao has led global product development of the Company’s four clinical-stage LVV programs, as well as Regulatory Policy and Patient Advocacy. Going forward, she will be responsible for the integrated development of Rocket’s LVV pipeline and LVV Global Product Teams. In late 2020, Jose Trevejo, M.D., Ph.D. was appointed Senior Vice President, Chief Development Officer of Rocket’s AVV pipeline. Dr. Trevejo brings significant clinical development leadership to Rocket from roles at Genentech, Vertex Pharmaceuticals, and others, most recently serving as Chief Executive Officer of non-viral gene therapy company SmartPharm. The Company’s establishing of two CDOs across AAV/LVV reflect the company’s commitment to developing world-class capabilities and treatments across the spectrum of gene therapy.
Strengthened balance sheet with redemption of existing convertible notes. On April 26, 2021, Rocket completed a redemption of its outstanding 6.25% Convertible Senior Notes due 2022. The company redeemed at an aggregate redemption price equal to 100% for each $1,000 principal amount of such notes, plus accrued and unpaid interest, removing the $38.35 million of the 6.25% Convertible Senior Notes from the consolidated balance sheet. Approximately $5.15 million aggregate principal amount of the 5.75% Convertible Senior Notes due 2021 remain outstanding.
Anticipated Milestones

Fanconi Anemia (RP-L102)
Updated "Process B" data (Q2 2021)
LAD-I (RP-L201)
Longer-term Phase 2 data (Q3 2021)
Danon Disease (RP-A501)
Longer-term Phase 1 data (Q4 2021)
PKD (RP-L301)
Longer-term Phase 1 data (Q4 2021)
IMO (RP-L401)
Initial Phase 1 data (Q3 2021)
Upcoming Investor Conferences

BofA Securities’ 2021 Virtual Health Care Conference, May 13, 2021
UBS Global Healthcare Virtual Conference, May 26, 2021
First Quarter Financial Results

Cash position. Cash, cash equivalents and investments as of March 31, 2021 were $466.4 million.
R&D expenses. Research and development expenses were $28.5 million for the three months ended March 31, 2021, compared to $17.0 million for the three months ended March 31, 2020, due to an increase in compensation and benefits expense resulting from increased R&D headcount, an increase in non-cash stock compensation expense, an increase in manufacturing and development costs and an increase in clinical trials expense.
G&A expenses. General and administrative expenses were $10.7 million for the three months ended March 31, 2021, compared to $7.2 million for the three months ended March 31, 2020, due to an increase in non-cash stock compensation expense and an increase in compensation and benefits expense due to increased G&A headcount.
Net loss. Net loss was $40.2 million or $0.65 per share (basic and diluted) for the three months ended March 31, 2021, compared to $24.7 million or $0.45 per share (basic and diluted) for the three months ended March 31, 2020.
Shares outstanding. 61,987,799 shares of common stock were outstanding as of March 31, 2021.
Financial Guidance

Rocket expects its balance in cash, cash equivalents and investments of $466.4 million as of March 31, 2021 to fund its operations into the second half of 2023, including the buildout and initiation of AAV cGMP manufacturing capabilities at our Cranbury, New Jersey R&D and manufacturing facility and advancement of our five clinical programs.
Conference Call Details

Rocket management will host a conference call today at 4:30 PM EDT. To access the call and webcast, please visit the events section of the website. The webcast replay will be available on the Rocket website following the completion of the call.

Investors may listen to the call by dialing (866) 939-3921 from locations in the United States or +1 (678) 302-3550 from outside the United States. Please refer to conference ID number 50162863.

On May 10, 2021 IMV Inc. (Nasdaq: IMV; TSX: IMV) ("IMV" or the "Company"), a clinical-stage biopharmaceutical company pioneering a novel class of cancer immunotherapies, reported that its lead compound, maveropepimut-S (DPX-Survivac) will be investigated in patients with hormone receptor positive/HER2-negative (HR+/HER2-) breast cancer. HR+/HER2- tumors represent an unmet clinical need with relatively poor responses to neoadjuvant endocrine treatment1 (Press release, IMV, MAY 10, 2021, View Source [SID1234579602]). This investigator-initiated clinical study will be conducted at the Providence Cancer Institute in Portland, Oregon, and is expected to begin during summer 2021.

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"We are excited to launch the next clinical proof of concept study of maveropepimut-S, which has already demonstrated positive clinical benefit in other cancer indications," said Frederic Ors, Chief Executive Officer at IMV. "Through targeted T-cell therapy, we believe maveropepimut-S presents a compelling option for the treatment of breast cancer. Our lead compound has demonstrated to enhance the innate tumor-fighting response which has resulted in efficacy in solid and liquid tumors without significant toxicity."

Kristina H. Young, M.D., Ph.D., Assistant Member, Tumor Microenvironment Lab in the Earle A. Chiles Research Institute, a division of Providence, commented, "Survivin upregulation is strongly associated with a subpopulation of breast cancer patients that are resistant to aromatase inhibitors. We believe that IMV’s survivin-targeted T cell therapy may improve the sensitivity to neoadjuvant aromatase inhibitors and, therefore, holds the potential to overcome resistance to treatment."

This three-arm Phase 1B trial is designed to assess the combination of maveropepimut-S and aromatase inhibitor with/without radiotherapy or cyclophosphamide (CPA) prior to surgery. Across the three arms of this study, IMV’s lead compound will be evaluated in 18 subjects with resectable, non-metastatic HR+/HER2- breast cancer.

The primary objective is to evaluate the safety of neoadjuvant combination of maveropepimut-S with the aromatase inhibitor and with/without radiation, or CPA and immunogenicity in each arm. Survivin-specific T cells in the resected tumor will be evaluated as a secondary objective. Extensive translational studies will be conducted as exploratory analyses to characterize maveropepimut-S’ mechanism of action in the tumor and the tumor’s immune environment.

"Understanding how HR+HER2- breast cancer responds to immunotherapy, particularly prior to surgery, remains an active area of investigation across the world," said Sasha E. Stanton, M.D., Ph.D., Assistant Member, Cancer Immunoprevention Lab at Providence. "Examining the role of this immune therapy targeting Survivin with the aromatase inhibitor letrozole, as well as the addition of radiation and low dose immune modulating chemotherapy, is important to improve neoadjuvant therapy response in these women."

About Maveropepimut-S (DPX-Survivac)

Maveropepimut-S is the lead candidate in IMV’s new class of immunotherapy that generates targeted and sustained cancer cell killing capabilities in vivo. Maveropepimut-S is evaluated in three phase 2 trials: in association with low dose cyclophosphamide (CPA) in advanced, recurrent ovarian cancer, and in combination with Merck’s Keytruda and low dose CPA in hematological and solid cancers (DLBCL, bladder, hepatocellular and MSI-high tumor cancers). Treatment with maveropepimut-S in association with CPA have demonstrated a favorable safety profile across all clinical studies.

Maveropepimut-S, consists of survivin-based peptides formulated in IMV’s proprietary delivery platform (DPX) which is designed to generate a sustained cytotoxic T cell response against cancer cells presenting survivin peptides on their surface.

Survivin, recognized by the National Cancer Institute (NCI) as a promising tumor-associated antigen, is broadly over-expressed in most cancer types, and plays an essential role in antagonizing cell death, supporting tumor-associated angiogenesis, and promoting resistance to chemotherapies. IMV has identified over 20 cancer indications in which survivin can be targeted by maveropepimut-S.

Maveropepimut-S has received Fast Track designation from the FDA as maintenance therapy in advanced ovarian cancer, as well as Orphan Drug designation status from the FDA and the European Medicines Agency (EMA) in the ovarian cancer indication.

On May 10, 2021 Compugen Ltd. (Nasdaq: CGEN), a clinical-stage cancer immunotherapy company and a leader in predictive target discovery, reported that it has expanded its research collaboration with Johns Hopkins University to include studies investigating the biology of a novel myeloid target that was computationally-discovered by Compugen (Press release, Compugen, MAY 10, 2021, View Source [SID1234579623]). Initial preclinical studies demonstrate the potential of this target to serve as a novel myeloid immunomodulator, with significant tumor growth inhibition observed upon genetic deletion in in-vivo studies.

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The research program, headed by Jelani Chinelo Zarif, M.S., Ph.D., Assistant Professor of Oncology and Drew Pardoll, M.D., Ph.D., Professor of Oncology, both at Johns Hopkins University School of Medicine, will explore the biological function and mechanism of this novel target, which is expressed on myeloid cells and macrophages in various cancers. The expanded research plan is intended to further evaluate and validate the role of the target in various tumors.

"We continue to invest in our early-stage programs, which serve as our pipeline growth engine and remain a high priority for Compugen," said Anat Cohen-Dayag, Ph.D., President and CEO of Compugen. "Our collaboration with Johns Hopkins has played an important role in the development of our clinical stage asset, COM701, and we are thrilled to expand this collaboration to deepen our biological understanding of this novel myeloid target selected from our early-stage pipeline. We hope that these studies will provide a strong biological foundation to support the future development of a new, potentially first-in-class, therapeutic program, which may diversify our immuno-oncology pipeline programs and offer a new treatment opportunity for cancer patients."

On May 10, 2021 BioXcel Therapeutics, Inc. ("BioXcel" or the "Company") (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, reported its quarterly results for the first quarter ended March 31, 2021 and provided an update on key strategic and operational initiatives (Press release, BioXcel Therapeutics, MAY 10, 2021, View Source [SID1234579521]).

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"This quarter, we have remained committed to executing on our vision for our neuroscience franchise, as we continue to advance the development of BXCL501 for treatment of agitation associated with dementia and prepare for its potential approval in acute agitation associated with schizophrenia and bipolar disorders," stated Vimal Mehta, Chief Executive Officer of BioXcel. "We are pleased to have recently received Breakthrough Therapy designation for BXCL501 in agitation associated with dementia, highlighting the urgent need for new therapy options, as well as this candidate’s potential to provide a solution across treatment settings. While we solidify our plans for a pivotal program in this condition, we are committed to building the commercial infrastructure needed to successfully launch BXCL501 in the first two indications, in addition to follow-on indications in the future."

Dr. Mehta continued, "Importantly, we are moving forward with our strategic geographic expansion strategy, with plans to file a Marketing Authorization Application to the European Medicines Agency for BXCL501 for the acute treatment of agitation associated with schizophrenia and bipolar disorders in the second half of 2021. As we continue to advance our innovative neuroscience program, we remain committed to delivering transformative medicines to patients, expanding our regulatory footprint and creating value for our stockholders."

First Quarter 2021 and Recent Highlights

BXCL501-Neuroscience Program

BXCL501 is an investigational, proprietary, orally dissolving thin film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist, designed for the treatment of agitation and opioid withdrawal symptoms. The Company believes BXCL501 may directly target a causal agitation mechanism.

Reported positive topline results from the Phase 1b/2 TRANQUILITY trial of BXCL501 for the acute treatment of agitation associated with dementia.
Granted Breakthrough Therapy designation by the U.S. Food and Drug Administration ("FDA") to BXCL501 for the acute treatment of agitation associated with dementia.
End of Phase 2 meeting with the FDA planned in 2Q 2021 to discuss registrational trial.
Recruitment in 40 mcg supplemental dose cohort of BXCL501 in TRANQUILITY expansion study is progressing well.
Completed its New Drug Application ("NDA") submission to the FDA for BXCL501 for the acute treatment of agitation associated with schizophrenia and the acute treatment of agitation associated with bipolar disorders I and II.
Plan to submit Marketing Authorization Application ("MAA") with European Medicines Agency ("EMA") using SERENITY I and II data for the acute treatment of agitation associated with schizophrenia and bipolar disorders I and II in 2H 2021.
Reported results from the Phase 1b/2 study of BXCL501 for the treatment of opioid withdrawal symptoms. Primary safety endpoints were met, and numerically improved retention rates, a secondary endpoint, were demonstrated in multiple BXCL501 dose cohorts.
PLACIDITY enrollment was voluntarily paused to assess challenges posed in opening relevant clinical sites and enrolling delirium patients in the ICU setting, including as a result of the burden COVID-19 has placed on ICUs.
Strengthened BXCL501 IP portfolio with issuance of two Japanese patents – Patent No. 6868698, directed to methods of treating agitation, which will expire no earlier than 2037, and Patent No. 1681960, directed to film design, which will expire no earlier than 2041.
Commercial Highlights

Launched "Boiling Point" educational campaign to raise awareness about agitation associated with schizophrenia and bipolar disorder and its impact on patients and clinicians.
Completed U.S. market assessment initiatives and design of sales force size and structure.
Medical Affairs Initiatives

Deployed the Medical Science Liaison and Medical Manage Care teams and have begun scientific and medical-to-medical exchange with healthcare professionals and payers, respectively.
Presented two posters covering data from the pivotal Phase 3 SERENITY trials in patients with acute agitation associated with schizophrenia and bipolar disorders I and II at the American Psychiatric Association ("APA") Annual Meeting.
BXCL701-Immuno-Oncology Program

BXCL701 is an orally-delivered small molecule, immunomodulator designed to inhibit dipeptidyl peptidase ("DPP") 8/9 and block immune evasion by targeting Fibroblast Activation Protein ("FAP"). It has shown single agent anti-tumor activity in melanoma and safety has been evaluated in more than 700 healthy subjects and cancer patients.

The adenocarcinoma cohort of the ongoing Phase 1b/2 trial of BXCL701 and pembrolizumab (KEYTRUDA) in aggressive forms of prostate cancer has met its efficacy bar to move to stage two. The trial will now continue to full enrollment and more complete efficacy data are expected to be presented at a scientific conference later this year.
Granted orphan drug designation by the FDA to BXCL701 for the treatment of soft tissue sarcoma.
Corporate Highlights

Appointed June Bray to the Company’s Board of Directors. Ms. Bray brings over forty years of extensive U.S. and global regulatory experience across all therapeutics areas, including psychiatry and neurology.
Appointed Javier Rodriguez as Chief Legal Officer and Corporate Secretary. Mr. Rodriguez brings over 20 years of extensive strategic and legal experience within the biopharmaceutical industry.
First Quarter 2021 Financial Results

Research and Development Expenses: Research and development expenses were $14.7 million during the first quarter of 2021, as compared to $12.4 million for the same period in 2020. The higher expenses were primarily attributable to an increase in personnel and related costs necessary to enlarge our development and medical teams. In addition, we experienced increased professional fees in conjunction with higher consulting fees and CMC costs related to BXCL501, as well as increased costs related to our RELEASE clinical trial. These increases were offset in part by a decrease in SERENITY I and II clinical trial costs.

General and Administrative Expenses: General and administrative expenses were $11.6 million for the first quarter of 2021, as compared to $2.6 million for the same period in 2020. The increase was primarily due to higher personnel related costs as well as costs related to our expansion in preparation of the potential commercial launch of BXCL501 in the U.S., and increased legal, professional fees, and insurance costs.

Net Loss: BioXcel reported a net loss of $26.4 million for the first quarter of 2021, compared to a net loss of $14.9 million for the same period in 2020.

The first quarter 2021 results include approximately $5.6 million in non-cash stock-based compensation costs, compared to non-cash stock-based compensation of $776,000 for the same period in 2020.

As of March 31, 2021, cash and cash equivalents totaled approximately $194 million.

Conference Call

BioXcel will host a conference call and webcast today at 8:30 a.m. ET. To access the call, please dial 877-407-2985 (domestic) and 201-378-4915 (international). A live webcast of the call will be available on the Investors sections of the BioXcel website and a replay of the call will be available through at least May 24, 2021. BioXcel Therapeutics website is available at www.bioxceltherapeutics.com.

BioXcel may use its website as a distribution channel of material information about the Company. Financial and other important information regarding the Company is routinely posted on and accessible through the Investors sections of its website at www.bioxceltherapeutics.com. In addition, you may automatically receive email alerts and other information about the Company when you enroll your email address by visiting the "Email Alerts" option under the News / Events menu of the Investors section of its website at www.bioxceltherapeutics.com.

On May 10, 2021 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported first quarter 2021 financial results (Press release, Precigen, MAY 10, 2021, View Source [SID1234579584]).

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"In the first quarter of 2021, our portfolio, including our most advanced clinical programs, has progressed consistent with guidance," said Helen Sabzevari, PhD, President and CEO of Precigen. "Our UltraCAR-T trials for PRGN-3005 in ovarian cancer and PRGN-3006 in acute myeloid leukemia as well as the PRGN-2009 trial in HPV-associated cancers continue to progress according to plan. We were pleased to initiate the first-in-human Phase 1 study of PRGN-2012, Precigen’s first off-the-shelf AdenoVerse immunotherapy targeting infectious disease to enter the clinic, and receive orphan drug designation from the US FDA in patients with RRP. We look forward to upcoming presentations of new clinical data for AG019 at FOCIS and the current trial status for PRGN-2009 at ASCO (Free ASCO Whitepaper) and we anticipate multiple data readouts from our portfolio in the coming months, meeting our stated milestones."

Business Highlights:

PRGN-3005 UltraCAR-T

Overview: PRGN-3005 UltraCAR-T is a first-in-class investigational therapy under evaluation in a Phase 1/1b clinical trial for the treatment of advanced, recurrent platinum resistant ovarian, fallopian tube or primary peritoneal cancer. Study subjects receive the PRGN-3005 infusion either via intraperitoneal (IP) (Arm A) or intravenous (IV) (Arm B) infusion (clinical trial identifier: NCT03907527). The study is being conducted in collaboration with the University of Washington and Fred Hutchinson Cancer Research Center. Preliminary Phase 1 data reported from the lowest two dose levels of the IP arm showed a favorable safety profile with no dose-limiting toxicities (DLTs), neurotoxicity or cytokine release syndromes (CRS); encouraging expansion and persistence without lymphodepletion; and clinical activity as evidenced by regression in total target tumor burden.
Enrollment Status: Dose escalation in both IP and IV arms of the Phase 1 trial is ongoing concurrently. Enrollment was completed in dose level 3 of the IP arm and dosing was initiated in dose level 4 of the IP arm. Escalation to higher doses is made possible as a result of the implementation of the UltraPorator system for on-site rapid manufacturing. The first patient was dosed in the IV arm after the US Food and Drug Administration (US FDA) cleared dosing of patients in the dose escalation phase of the IV arm concurrently with the IP arm. The US FDA has cleared enrollment of patients in dose level 3 in the IV arm without the need to follow 3+3 dose escalation through dose levels 1 and 2.
Upcoming Milestones: The Company anticipates the presentation of interim data from the IP arm of the Phase 1 dose escalation trial as well as the initiation of the expansion phase of the IP arm in the second half of 2021.
PRGN-3006 UltraCAR-T

Overview: PRGN-3006 UltraCAR-T is a first-in-class investigational therapy currently under evaluation in a Phase 1/1b clinical trial for the treatment of patients with relapsed or refractory (r/r) acute myeloid leukemia (AML) or higher-risk myelodysplastic syndromes (MDS). Study subjects receive the PRGN-3006 infusion either without prior lymphodepletion (Cohort 1) or following lymphodepleting chemotherapy (Cohort 2) (clinical trial identifier: NCT03927261). The study is being conducted in collaboration with the H. Lee Moffitt Cancer Center & Research Institute. PRGN-3006 UltraCAR-T has been granted Orphan Drug Designation in patients with AML by the US FDA. Preliminary Phase 1 data reported for the two lowest dose levels in Cohort 1 and the lowest dose level in Cohort 2 showed a favorable safety profile with no DLTs or neurotoxicity; encouraging expansion and persistence of PRGN-3006 UltraCAR-T in both cohorts; and clinical activity as evidenced by reduction in AML tumor blast levels. One of the patients treated with PRGN-3006 at the lowest dose level with lymphodepletion (Cohort 1), with approximately nine million UltraCAR-T cells, achieved complete remission with incomplete hematologic recovery (CRi) per European Leukemia Net (ELN) criteria.
Enrollment Status: The dose escalation phase of both the lymphodepletion and non-lymphodepletion cohorts of the Phase 1 trial is ongoing concurrently. Enrollment was completed in dose level 3 of the non-lymphodepletion cohort and dose level 2 of the lymphodepletion cohort.
Upcoming Milestones: The Company anticipates the presentation of interim Phase 1 data as well as the initiation of the dose expansion phase in the second half of 2021.
AG019 ActoBiotics

Overview: AG019 ActoBiotics is a first-in-class, orally administered, investigational therapy designed to address the underlying cause of Type 1 diabetes (T1D) and is currently under evaluation in a Phase 1b/2a clinical trial for the treatment of early-onset T1D (clinical trial identifier: NCT03751007; EudraCT 2017-002871-24). Interim data from both the Phase 1b monotherapy and Phase 2a combination arms showed a favorable safety profile with no dose-related adverse events or serious adverse events; an encouraging trend in insulin C-peptide levels, a biomarker for T1D disease progression; an increase in preproinsulin (PPI)-specific Type 1 regulatory (Tr1) cells; and a decrease in PPI specific CD8+ T cells.
Enrollment Status: Enrollment and dosing is complete in the Phase 1b and Phase 2a portions of the study.
Clinical Data Presentation at FOCIS 2021: Interim data from the AG019 Phase 1b/2a clinical trial will be presented on June 10, 2021 at 2:05 PM PT as an oral presentation at the Federation of Clinical Immunology Societies (FOCIS) 2021 Virtual Annual Meeting. The abstract entitled, "Lactococcus lactis producing Proinsulin and IL-10 therapy increases Antigen Specific Regulatory T-cells in Monotherapy and in Combination with an anti-CD3 Monoclonal Antibody (Teplizumab) in newly diagnosed T1D patients" will be presented by Kevan Herold, MD, CNH Long Professor of Immunobiology and of Medicine (Endocrinology) at the Yale School of Medicine.
PRGN-2009 AdenoVerse Immunotherapy

Overview: PRGN-2009 is a first-in-class, off-the-shelf (OTS) investigational immunotherapy utilizing the AdenoVerse platform designed to activate the immune system to recognize and target HPV-positive (HPV+) solid tumors. PRGN-2009 is currently under evaluation in a Phase 1/2 clinical trial as a monotherapy or in combination with bintrafusp alfa (M7824) in patients with HPV-associated cancers (clinical trial identifier: NCT04432597). The study is being conducted under a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI). Preliminary Phase 1 data from the monotherapy arm of the Phase 1 trial showed that all patients (n=6) received multiple PRGN-2009 administrations and repeated administration of PRGN-2009 treatment was well-tolerated with no DLTs. Preliminary correlative analysis from patients treated with PRGN-2009 monotherapy at dose level 1 (n=3) demonstrated an increase in HPV 16 and/or HPV 18-specific T-cell response post PRGN-2009 administration in 100% (3 of 3) of patients and an increase in the magnitude and breadth of immune response was seen with repeated administrations of PRGN-2009.
Enrollment Status: The Phase 1 monotherapy arm has completed enrollment. Subsequently, the Phase 2 trial in patients with newly diagnosed stage II/III p16-positive oropharyngeal cancer or patients with newly diagnosed operable stage II/III/IVA/IVB HPV+ sinonasal squamous cell cancer was initiated. The first patient in the Phase 2 trial was dosed.
Preclinical Data Publication: Preclinical data for PRGN-2009 was published in the Journal of Clinical Investigation entitled, "Characterization of recombinant gorilla adenovirus HPV therapeutic vaccine PRGN-2009," which provides the first evaluation of PRGN-2009 and shows promising preclinical antitumor efficacy and induction of HPV-specific T cells, along with the rationale for its evaluation in clinical trials.
Trial-in-Progress Presentation at ASCO (Free ASCO Whitepaper) 2021: A trial-in-progress update on the PRGN-2009 study will be provided at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 annual meeting. The abstract entitled, "First-in-human Phase 1/2 trial of PRGN-2009 vaccine as monotherapy or with bintrafusp alfa in patients with recurrent/metastatic (R/M) human papillomavirus (HPV)-associated cancers (HPVC) and as neoadjuvant/induction therapy in locoregionally advanced (LA) HPV oropharyngeal (OP) and sinonasal (SN) squamous cell cancer (SCC)" will be presented as a poster presentation by Charalampos S. Floudas, MD, DMSc, MS, Assistant Research Physician, Genitourinary Malignancies Branch at the Center for Cancer Research at the NCI.
Upcoming Milestones: The Company anticipates the presentation of interim Phase 1 data in the second half of 2021.
PRGN-2012 AdenoVerse Immunotherapy

Overview: PRGN-2012 is a first-in-class, investigational OTS AdenoVerse immunotherapy designed to elicit immune responses directed against cells infected with HPV 6 or HPV 11 for treatment of recurrent respiratory papillomatosis (RRP). A Phase 1 clinical trial of PRGN-2012 AdenoVerse immunotherapy in adult patients with RRP is ongoing (clinical trial identifier: NCT04724980). In preclinical studies, PRGN-2012 showed robust HPV 6 and HPV 11-specific T-cell responses in RRP patient samples in vitro.
Orphan Drug Designation: In March 2021, the Company received US FDA Orphan Drug Designation for PRGN-2012 AdenoVerse immunotherapy in patients with RRP.
Enrollment Status: In March 2021, the first patient was dosed in the Phase 1 study.
First Quarter 2021 Financial Highlights

Net cash used in operating activities of $16.4 million in 2021 compared to $27.7 million in 2020;
Cash, cash equivalents, short-term and long-term investments totaled $209.3 million as of March 31, 2021; and
Total revenues of $24.5 million in 2021 compared to $29.8 million in 2020.
First Quarter 2021 Financial Results Compared to Prior Year Period
Research and development expenses decreased $0.8 million, or 7%, from the quarter ended March 31, 2020. Salaries, benefits, and other personnel costs decreased $0.7 million in 2021 as the Company scaled down certain research and development functions in the first quarter of 2020 as a result of certain programs being previously deprioritized. Selling, general and administrative expenses decreased $2.8 million, or 13%. Salaries, benefits, and other personnel costs decreased $1.0 million in 2021 primarily due to a reduced headcount as the Company scaled down its corporate functions to support its more streamlined organization and reduced stock compensation costs for previously granted awards that became fully vested in early 2021. Professional fees decreased $1.0 million primarily due to a decrease in legal fees associated with certain litigation matters that were settled in the second half of 2020. Net loss from continuing operations was $21.8 million, or $(0.11) per basic share, of which $9.7 million was for non-cash charges in 2021 compared to net loss from continuing operations of $20.8 million, or $(0.13) per basic share, of which $7.7 million was for non-cash charges in 2020.

Total revenues decreased $5.3 million, or 18%, from the quarter ended March 31, 2020. Collaboration and licensing revenues decreased $10.7 million primarily due to the recognition of previously deferred revenue upon the mutual termination of one of its collaboration agreements in February 2020. Product and service revenues generated by Trans Ova and Exemplar increased $5.4 million primarily due to higher customer demand for Trans Ova’s products and services as a result of stronger beef and dairy industries in the current year, as well as increased services provided by Exemplar to new and existing customers. Gross margin on products and services improved as a result of the increased revenues, a change in pricing structure for certain customers, and operational efficiencies that have been gained through reductions in workforce and improved inventory management.