On November 4, 2020 Mauna Kea Technologies (Paris:MKEA) (OTCQX:MKEAY) (Euronext: MKEA) inventor of Cellvizio, the multidisciplinary probe and needle-based confocal laser endomicroscopy (pCLE/nCLE) platform, reported the publication of a peer-reviewed meta-analysis and of an international Delphi consensus report based on a systematic, evidence-based review of endoscopic ultrasound (EUS) guided needle-based confocal laser endomicroscopy for pancreatic cystic lesion (PCL) evaluation (Press release, Mauna Kea Technologies, NOV 4, 2020, View Source [SID1234569877]).

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The first publication entitled "Needle-based Confocal Laser Endomicroscopy in Pancreatic Cysts: a Meta-Analysis", was published in the European Journal of Gastroenterology & Hepatology (2020, DOI: 10.1097/MEG.0000000000001728). Ten studies enrolling 536 patients were included and the authors evaluated diagnostic accuracy, pooled sensitivity, specificity, and mean procedural time. The meta-analysis concluded that confocal laser endomicroscopy "clearly outperformed" endoscopic ultrasound fine-needle aspiration (EUS-FNA) in terms of diagnostic accuracy (odds ratio 3.94, 1.58–9.82; P = 0.003) and supports the use of needle-based confocal laser endomicroscopy as a safe and effective tool in the diagnostic algorithm of pancreatic cysts.

The second publication entitled "Confocal Endomicroscopy for the Evaluation of Pancreatic Cystic Lesions: A Systematic Review and an International Delphi Consensus Report", was published in the peer-reviewed journal Endoscopy International Open (2020, DOI: 10.1055/a-1229-4156) and was based on consensus statements from an international panel of 15 experts in pancreatic disease that conducted evidence-based reviews of the applications, outcomes, procedural processes, indications, training, and credentialing of EUS-nCLE in the management of PCLs. The consensus report reflected a high level of agreement pertaining to expert consensus statements and established that EUS-guided nCLE is a minimally invasive procedure that improves evaluation of PCLs and "should be systematically considered when EUS-FNA is indicated for PCL evaluation."

The consensus report also concluded that the use of nCLE as an adjunct to standard EUS-FNA could positively impact patient management and improve healthcare resource utilization by reducing the number of misdiagnoses and preventing redundant follow-up investigations and unnecessary surgery.

"The new meta-analysis and consensus report provide further support that Cellvizio plays a key role in the evaluation of pancreatic cysts and can significantly improve the speed and accuracy of reaching a conclusive diagnosis," said Robert L. Gershon, Chief Executive Officer of Mauna Kea Technologies. "The level of clinical evidence and support suggests that hospitals performing endoscopic evaluation of pancreatic cysts would benefit their patients by incorporating Cellvizio as part of the management of their patients. This represents approximately 75,000 procedures in the US alone. We look forward to the growing adoption of Cellvizio for this important application."

On November 4, 2020 ADC Therapeutics SA (NYSE:ADCT), a late clinical-stage oncology-focused biotechnology company pioneering the development and commercialization of highly potent and targeted antibody drug conjugates (ADCs) for patients with hematological malignancies and solid tumors, reported that eight abstracts have been selected for presentation at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which is being held virtually from December 5-8, 2020 (Press release, ADC Therapeutics, NOV 4, 2020, View Source [SID1234569876]). Presentations will feature data on three of the Company’s pyrrolobenzodiazepine (PBD)-based ADCs – loncastuximab tesirine (Lonca), camidanlumab tesirine (Cami) and ADCT-602.

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"The breadth of data that will be presented at the ASH (Free ASH Whitepaper) Annual Meeting reflects the potential of our product candidates and speaks to our leadership in the development of next-generation ADCs to improve outcomes for patients with blood cancers," said Jay Feingold, MD, PhD, Senior Vice President and Chief Medical Officer of ADC Therapeutics. "Notably, we look forward to sharing subgroup data from the Lonca LOTIS 2 pivotal trial in relapsed or refractory diffuse large B-cell lymphoma, interim results from our LOTIS 3 trial evaluating Lonca in combination with ibrutinib in patients with advanced diffuse large B-cell lymphoma or mantle cell lymphoma, as well as preliminary results from our pivotal Phase 2 trial of Cami in patients with relapsed or refractory Hodgkin lymphoma."

Lonca Presentations

Efficacy and Safety of Loncastuximab Tesirine (ADCT-402) in Relapsed/Refractory Diffuse Large B-Cell Lymphoma
Abstract: 1183
Date and Time: Saturday, December 5, 2020, 7 a.m. – 3:30 p.m. PST
Session: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)—Results from Prospective Clinical Trials: Poster I
Presenter: Paolo F. Caimi, MD, Case Comprehensive Cancer Center, Case Western Reserve University

Characteristics and Treatment Patterns of Patients with Relapsed/Refractory Diffuse Large B-Cell Lymphoma Who Received ≥3 Lines of Therapies
Abstract: 1651
Date and Time: Saturday, December 5, 2020, 7 a.m. – 3:30 p.m. PST
Session: 905. Outcomes Research—Malignant Conditions (Lymphoid Disease): Poster I
Presenter: Jipan Xie, MD, PhD, Analysis Group, Inc.

Interim Results of Loncastuximab Tesirine Combined with Ibrutinib in Diffuse Large B-Cell Lymphoma or Mantle Cell Lymphoma (LOTIS-3)
Abstract: 2099
Date and Time: Sunday, December 6, 2020, 7 a.m. – 3:30 p.m. PST
Session: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)—Results from Prospective Clinical Trials: Poster II
Presenter: Julien Depaus, MD, CHU UCL Namur

Safety and Antitumor Activity Study Evaluating Loncastuximab Tesirine and Rituximab Versus Immunochemotherapy in Diffuse Large B-Cell Lymphoma
Abstract: 2107
Date and Time: Sunday, December 6, 2020, 7 a.m. – 3:30 p.m. PST
Session: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)—Results from Prospective Clinical Trials: Poster II
Presenter: Yuliya Linhares, MD, Miami Cancer Institute, Baptist Health South Florida

Cami Presentations

Preliminary Results of a Phase 2 Study of Camidanlumab Tesirine (Cami), a Novel Pyrrolobenzodiazepine-Based Antibody-Drug Conjugate, in Patients with Relapsed or Refractory Hodgkin Lymphoma
Abstract: 474
Date and Time: Sunday, December 6, 2020, 3 p.m. PST
Session: 624. Hodgkin Lymphoma and T/NK Cell Lymphoma—Clinical Studies: Clinical Studies in Hodgkin Lymphoma
Presenter: Alex Herrera, MD, City of Hope Medical Center

Pharmacokinetic and Pharmacodynamic Correlates from the Phase 1 Study of Camidanlumab Tesirine (Cami) in Patients with Relapsed or Refractory Hodgkin Lymphoma and Non-Hodgkin Lymphoma
Abstract: 2981
Date and Time: Monday, December 7, 2020, 7 a.m. – 3:30 p.m. PST
Session: 624. Hodgkin Lymphoma and T/NK Cell Lymphoma—Clinical Studies: Poster III
Presenter: Joseph Boni, ADC Therapeutics

Combination of Camidanlumab Tesirine, a CD25-Targeted ADC, with Gemcitabine Elicits Synergistic Anti-Tumor Activity in Preclinical Tumor Models
Abstract: 1178
Date and Time: Saturday, December 5, 2020, 7 a.m. – 3:30 p.m. PST
Session: 625. Lymphoma: Pre-Clinical—Chemotherapy and Biologic Agents: Poster I
Presenter: Francesca Zammarchi, ADC Therapeutics

ADCT-602 Presentation

Analysis of Adct-602 Pre-Clinical Activity in B-Cell Lymphoma Models and Identification of Potential Biomarkers for Its Activity
Abstract: 3011
Date and Time: Monday, December 7, 2020, 7 a.m. – 3:30 p.m. PST
Session: 625. Lymphoma: Pre-Clinical—Chemotherapy and Biologic Agents: Poster III
Presenter: Francesco Bertoni, MD, Institute of Oncology Research, Università della Svizzera italiana

Online Publication in November Supplemental Issue of Blood
Symptoms, Health-Related Quality of Life, and Tolerability of Loncastuximab Tesirine in Patients with Relapsed or Refractory Diffuse Large B Cell Lymphoma
Abstract Number: 3919

The abstracts are available through the ASH (Free ASH Whitepaper) online meeting program and will be published in the November supplemental issue of Blood.

About Loncastuximab Tesirine (Lonca)

Loncastuximab tesirine (Lonca, formerly ADCT-402) is an antibody drug conjugate (ADC) composed of a humanized monoclonal antibody directed against human CD19 and conjugated through a linker to a pyrrolobenzodiazepine (PBD) dimer cytotoxin. Once bound to a CD19-expressing cell, Lonca is designed to be internalized by the cell, following which the warhead is released. The warhead is designed to bind irreversibly to DNA to create highly potent interstrand cross-links that block DNA strand separation, thus disrupting essential DNA metabolic processes such as replication and ultimately resulting in cell death. CD19 is a clinically validated target for the treatment of B-cell malignancies.

In September 2020, ADC Therapeutics submitted a Biologics License Application to the U.S. Food and Drug Administration seeking accelerated approval for Lonca for the treatment of relapsed or refractory diffuse large B-cell lymphoma (DLBCL). Lonca is being evaluated in LOTIS 2, a pivotal Phase 2 clinical trial in patients with relapsed or refractory DLBCL, LOTIS 3, a Phase 1/2 clinical trial in combination with ibrutinib in patients with relapsed or refractory DLBCL or mantle cell lymphoma, and LOTIS 5, a Phase 3 confirmatory clinical trial in combination with rituximab in patients with relapsed or refractory DLBCL.

About Camidanlumab Tesirine (Cami)

Camidanlumab tesirine (Cami, formerly ADCT-301) is an antibody drug conjugate (ADC) comprised of a monoclonal antibody that binds to CD25 (HuMax-TAC, licensed from Genmab A/S), conjugated to the pyrrolobenzodiazepine (PBD) dimer payload, tesirine. Once bound to a CD25-expressing cell, ADCT-301 is internalized into the cell where enzymes release the PBD-based warhead killing the cell. This applies to CD25-expressing tumor cells, and also to CD25-expressing Tregs. The intra-tumoral release of its PBD warhead may also cause bystander killing of neighboring tumor cells and PBDs have also been shown to induce immunogenic cell death. All of these properties of Cami may enhance immune-mediated anti-tumor activity. Cami is being evaluated in a pivotal Phase 2 clinical trial in patients with relapsed or refractory Hodgkin lymphoma (HL), as well as in a Phase 1a/1b clinical trial in patients with relapsed or refractory HL and non-Hodgkin lymphoma and a Phase 1b clinical trial as monotherapy and in combination with pembrolizumab in solid tumors.

On November 4, 2020 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported that updated data from the ongoing Phase 1 clinical study of GDA-201, an investigational, natural killer (NK) cell-based cancer immunotherapy for the treatment of patients with non-Hodgkin lymphoma, will be presented in an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) 62nd Annual Meeting, which is being held virtually from December 5-8, 2020. NK cell immunotherapies are thought to offer tremendous potential for transforming the care of hematologic malignancies. Gamida Cell is pioneering a novel approach that harnesses the power of its cell expansion technology, which uniquely improves antibody-dependent cellular cytotoxicity (ADCC) and tumor targeting of NK cells.

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Additionally, new data from the ongoing Phase 1/2 study of omidubicel in patients with severe aplastic anemia will be shared in a poster presentation during the meeting. Omidubicel is an investigational advanced cell therapy in development as a potential life-saving treatment option for patients in need of a bone marrow transplant.

Omidubicel is also being evaluated in a Phase 3 study in patients with hematologic malignancies. Earlier this year, Gamida Cell reported that its Phase 3 study of omidubicel met its primary endpoint, demonstrating a highly statistically significant reduction in time to neutrophil engraftment, a key milestone in recovery from a stem cell transplant. Last month, Gamida Cell also reported that all three secondary endpoints for the study demonstrated statistical significance. The secondary endpoints in the study include outcomes for: platelet engraftment, infections and hospitalizations.

Details about the ASH (Free ASH Whitepaper) presentations are as follows:

Title: Results of a Phase 1 Trial of GDA-201, Nicotinamide-Expanded Allogeneic Natural Killer (NK) Cells in Patients with Refractory Non-Hodgkin Lymphoma (NHL) and Multiple Myeloma
Abstract Number: 63
Lead Author: Veronika Bachanova, M.D., Ph.D., Division of Hematology, Oncology and Transplantation, University of Minnesota, Minneapolis, MN
Time: Saturday, December 5, 2020, 7:30 a.m. – 9:00 a.m. PT (session time) and 7:30 a.m. PT (presentation)

Title: Rapid Engraftment, Immune Recovery, and Resolution of Transfusion Dependence in Treatment-Refractory Severe Aplastic Anemia Following Transplantation with Ex Vivo Expanded Umbilical Cord Blood (Omidubicel)
Abstract Number: 1531
Lead Author: Mohamed Samour, M.D., Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD
Time: Saturday, December 5, 2020, 7:00 a.m. – 3:30 p.m. PT

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In both Phase 1/2 and Phase 3 clinical studies (NCT01816230, NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

About GDA-201

Gamida Cell applied the capabilities of its NAM-based cell expansion technology to develop GDA-201, an innate natural killer (NK) cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. GDA-201 is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.1

GDA-201 is an investigational therapy, and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On November 4, 2020 Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of cancer and autoimmune disease, reported that data from the ongoing Phase 1 dose-escalation study of vibecotamab (XmAb14045), a CD123 x CD3 bispecific antibody, in patients with relapsed/refractory acute myeloid leukemia (AML) will be presented in an oral session at the 2020 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting on Sunday, December 6, 2020 (Press release, Xencor, NOV 4, 2020, View Source [SID1234569874]).

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"Data from the Phase 1 study of vibecotamab suggest that patients with AML having low baseline disease burden and specific T-cell signatures may be more likely to respond to treatment with vibecotamab. The primary toxicity, CRS, is generally mild-to-moderate in severity when observed and is manageable," said Allen Yang, M.D., Ph.D., senior vice president and chief medical offer at Xencor. "We continue to optimize dosing regimen in this study, and along with our partner Novartis, we are planning our next clinical trials to develop vibecotamab in patients, for whom an intermittently dosed, CD123-targeting antibody could be a needed therapeutic option."

Key Highlights from the Abstract

The accepted abstract is available on the ASH (Free ASH Whitepaper) conference website.

At data cut off for submitting the abstract, 104 patients with AML, one patient with B cell acute lymphoblastic leukemia and one patient with chronic myeloid leukemia had received vibecotamab. Patients had a median age of 63 years and were heavily pretreated, having a median of three prior therapies, and 30% (n=32/106) had undergone prior allogeneic stem cell transplantation.
Patients received doses of vibecotamab ranging from 0.003 mcg/kg to 12 mcg/kg. The recommended initial priming dose was determined to be 0.75 mcg/kg. A maximum tolerated dose (MTD) was not reached.
Cytokine release syndrome (CRS) was the most common toxicity occurring in 58% of patients (n=62), and 8% of patients (n=9) experienced CRS at Grade 3 or higher. The majority of CRS was observed on the first dose and was generally manageable with premedication. Additional adverse events consistent with CRS but not reported as such, including chills, fever, tachycardia and hypotension, were reported in an additional 24% of patients. No myelosuppression requiring dose modification or evidence of tumor lysis syndrome was observed.
At dose levels of at least 0.75 mcg/kg (n=51), two patients achieved complete remission (CR), three patients achieved a CR with incomplete hematologic recovery, and two patients or morphologic leukemia-free state (ORR=14%).
Patients with responses were characterized by lower disease burden and specific T-cell subtypes.
Presentation Details

Abstract: 460
Title: Complete Responses in Relapsed/Refractory Acute Myeloid Leukemia (AML) Patients on a Weekly Dosing Schedule of Vibecotamab (XmAb14045), a CD123 x CD3 T Cell-Engaging Bispecific Antibody; Initial Results of a Phase 1 Study
Session: 613. Acute Myeloid Leukemia: Potpourri of Potential Practice Changing Studies
Date & Time: Sunday, December 6, 2020, 2:30 p.m. PST
About Vibecotamab

Vibecotamab (XmAb14045) is a tumor-targeted antibody that contains both a CD123 binding domain and a cytotoxic T-cell binding domain (CD3) in a Phase 1 clinical trial for the treatment of acute myeloid leukemia (AML) and other CD123-expressing hematologic malignancies. An XmAb Bispecific Fc domain serves as the scaffold for these two antigen binding domains and confers long circulating half-life, stability and ease of manufacture on vibecotamab. CD123 is highly expressed on AML cells and leukemic stem cells, and it is associated with poorer prognosis in AML patients. Engagement of CD3 by vibecotamab activates T cells for highly potent and targeted killing of CD123-expressing tumor cells.

On November 4, 2020 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported financial results for the quarter that ended September 30, 2020, along with an update on the Company’s key pipeline developments, business operations and upcoming milestones (Press release, Rocket Pharmaceuticals, NOV 4, 2020, View Source [SID1234569873]).

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"We are incredibly pleased with the steady progress we made across all five of our clinical programs this quarter. The data from our RP-L201 trial for LAD-I demonstrated the potential for a very robust and attractive profile for our ‘Process B’ lentiviral gene therapy pipeline. We also treated our first higher dose patient in our Phase 1 study of RP-A501 for the treatment of Danon Disease, and presented compelling preclinical data in IMO," said Gaurav Shah, M.D., President and Chief Executive Officer of Rocket. "In addition, I am proud that against the backdrop of a global pandemic, while there have been some delays in patient follow up and data collection, we continued to enroll and treat patients in our LVV and AAV gene therapy programs as well as expand our clinical trials internationally. Finally, we made strong progress in the build-out of Rocket’s AAV R&D and manufacturing facility, with the ability to produce GMP product anticipated in 2021."

Dr. Shah continued, "We are looking forward to sharing additional updates as we enter the fourth quarter. These data announcements include an update on the Phase 1 and 2 trials for FA ‘Process B’, and for the first time, Phase 1 clinical data from our two largest indications: RP-L301 for PKD and RP-A501 for Danon Disease. We believe we are advancing closer to our goal of taking drug products from the discovery phase to BLA submission and launch so we may help improve the lives of patients facing these rare and devastating childhood diseases."

Key Pipeline Developments and Operational Updates

Positive clinical update for the Company’s Leukocyte Adhesion Deficiency Program (LAD-I) presented at the European Society for Immunodeficiencies (ESID) 2020 Meeting along with preclinical data from the Company’s Infantile Malignant Osteopetrosis (IMO) program.
An oral presentation provided positive longer-term follow-up data from the Phase 1/2 clinical trial of RP-L201 for LAD-I. The data presented in the oral presentation are from two pediatric patients with severe LAD-I, as defined by CD18 expression of less than 2%. Both patients were treated with RP-L201, Rocket’s ex-vivo lentiviral gene therapy candidate. Patient L201-003-1001 was 9 years of age at treatment and has been followed for 12 months and Patient L201-003-1004 was 3 years of age at treatment and has been followed for 4 months. Treatments were well tolerated, and no safety issues were reported with infusion or post-treatment. Both subjects achieved hematopoietic reconstitution in less than 4 weeks. Patient L201-003-1001 demonstrated durable CD18 expression of 40%, peripheral blood VCN levels of 1.3, visible signs of improvement in existing skin lesions and no new infections reported 12 months post-treatment. Patient L201-003-1004 demonstrated CD18 expression of 28% and early peripheral blood VCN trending similarly to the first patient, reported 4 months post-treatment.
An e-poster highlighted preclinical trial data on RP-L401 for IMO. Preclinical data on IMO indicate that a modest level of engraftment corrects the disease phenotype in vivo, with increased long-term survival, tooth eruption, weight gain and normalized bone resorption. Results support acceleration into clinical development for RP-L401.
First patient treated in the higher dose cohort of the Phase 1 dose-escalation clinical trial of RP-A501 for the treatment of Danon Disease. The first patient was treated at the higher dose level of 1.1×1014 genome copies/kilogram after clearance from the U.S. Food and Drug Administration (FDA) and the Independent Data Safety Monitoring Committee (IDSMC) to move to the higher dose cohort of the study. Preliminary Phase 1 data are anticipated in December assuming no further delays in patient data collection due to COVID-19.
Opened the Research & Development (R&D) and Chemistry, Manufacturing and Controls (CMC) facility in Cranbury, New Jersey. Approximately half of the newly constructed 103,720 square foot facility will be dedicated to adeno-associated virus (AAV) Current Good Manufacturing Practice (cGMP) manufacturing. As previously guided, the first cGMP clinical product release is expected in 2021.
Received additional regulatory designations for IMO and Danon Disease programs from U.S. Food and Drug Administration. RP-L401 gene therapy for IMO received Fast Track Designation and RP-A501 for Danon Disease received Rare Pediatric Disease Designation. The FDA’s Fast Track program facilitates the development of products intended to treat serious conditions that have the potential to address unmet medical needs. The designation enables greater access to the FDA for the purpose of expediting the product’s development, review and potential approval. The FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the U.S.
Expanded clinical sites for Fanconi Anemia (FA), Danon, LAD-I and IMO trials, adding to global centers of excellence. The newly added centers, which include some of the leading gene therapy research programs in the world, include: Great Ormond Street Hospital (GOSH), Children’s Hospital of Philadelphia (CHOP), the University of Minnesota and the University of California, Los Angeles (UCLA). We anticipate these additional sites will expand patient access to Rocket’s clinical trials worldwide.
Hosted third Pyruvate Kinase Deficiency (PKD) Day in October. Continuing its commitment to the patient communities it serves, Rocket facilitated the first virtual edition of PKD Day. The event, intended for patients with PKD and their families, gave attendees an introduction to PKD, gene therapy and Rocket’s clinical program and provided time for a Q&A session with experts in the field and Rocket team members.
Anticipated Milestones

FA (RP-L102)
Preliminary "Process B" data (12/20)
Updated "Process B" data (1H21)
Danon Disease (RP-A501)
Preliminary Phase 1 data (12/20)
Updated Phase 1 data (2H21)
LAD-I (RP-L201)
Phase 2 data (1H21)
PKD (RP-L301)
Preliminary Phase 1 data (12/20)
Phase 1 data update (2H21)
IMO (RP-L401)
Phase 1 data (2H21)
Upcoming Investor Conferences

Piper Sandler 32nd Annual Healthcare Conference, December 1, 2020
Evercore ISI 3rd Annual HealthCONx Conference, December 2, 2020
Third Quarter Financial Results

Cash position. Cash, cash equivalents and investments as of September 30, 2020 were $228.7 million.
Debt. Our balance sheet includes $52.0 million of fully convertible notes.
R&D expenses. Research and development expenses were $21.7 million for the three months ended September 30, 2020, compared to $14.8 million for the three months ended September 30, 2019, primarily due to increases in manufacturing and development costs, clinical trial expenses, license fees, and compensation and benefit expenses due to increased R&D headcount.
G&A expenses. General and administrative expenses were $5.7 million for the three months ended September 30, 2020, compared to $4.3 million for the three months ended September 30, 2019, primarily due to increases in non-cash stock compensation expense and an increase in compensation and benefit expenses due to increased G&A headcount.
Net loss. Net loss was $29.0 million or $0.53 per share (basic and diluted) for the three months ended September 30, 2020, compared to $19.3 million or $0.38 per share (basic and diluted) for the three months ended September 30, 2019.
Shares outstanding. 55,204,127 shares of common stock were outstanding as of September 30, 2020.
Financial Guidance

Cash position. As of September 30, 2020, we had cash, cash equivalents and investments of $228.7 million. Rocket expects such resources will be sufficient to fund its operations into the second quarter of 2022.