Benzinga | May 13, 2021 07:05AM EDT

Rocket Pharma Presents Clinical Data From Fanconi Anemia, Leukocyte Adhesion Deficiency-I, Pyruvate Kinase Deficiency Programs at Meeting of American Society of Gene and Cell Therapy: Showed Evidence Of Prelim. Engraftment In At Least 6 Of 9 Patients

--RP-L102 for Fanconi Anemia Shows Evidence of Preliminary Engraftment in at Least Six of Nine Patients--

-- RP-L201 for LAD-I Leads to Durable CD18 Expression, Improved Skin Lesions, and Consistent Peripheral Vector Copy Numbers Up to 18-Months Post-treatment--

--Sustained Hemoglobin Improvements Up to 9-Months Post-treatment with RP-L301 for PKD --

-- Positive Benefit/Risk Profile Observed Across LVV Programs as Rocket Advances Towards Regulatory Submissions--

CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces positive clinical data from its Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), and Pyruvate Kinase Deficiency (PKD) gene therapy programs presented at the 24th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.

"We are very excited to report positive clinical results from three of our lentiviral-based gene therapy programs at this year's ASGCT, which show the great potential of these therapies to successfully treat FA, LAD-I and PKD. In the case of RP-L102 for FA and RP-L201 for LAD-I, the new data advance us closer to regulatory submissions," said Gaurav Shah, M.D., Chief Executive Officer of Rocket. "At least six out of nine patients in our FA Phase 1 and 2 trials now show evidence of engraftment, further supporting the potential of RP-L102 to serve as a hematologic treatment option for FA in the absence of cytotoxic conditioning. Although preliminary, four out of the five patients anticipated necessary for a positive trial outcome have initially met the minimum 10% MMC resistance threshold in the bone marrow on at least one occasion, including two patients at 6-months post-treatment."

Dr. Shah continued, "In our Phase 1/2 trial for LAD-I, all four patients with follow-up ranging from 3 to 18 months had CD18 expression that substantially exceeded the 4-10% threshold associated with survival into adulthood and consistent peripheral blood vector copy number, further demonstrating the potential of RP-L201 to yield durable clinical benefit. All of these patients have been free of serious infections since hospital discharge following RP-L201 therapy. Lastly, data from our Phase 1 trial of RP-L301 for PKD show that both patients' hemoglobin levels have safely normalized, with neither patient requiring red blood cell transfusions after hematopoietic reconstitution while demonstrating improving hemolysis markers. We are proud of the progress we have made across all three programs and look forward to further advancing our investigational gene therapies to offer curative treatments to patients with these devastating diseases."

Gene Therapy for Fanconi Anemia [Group A]: Preliminary Results of Ongoing RP-L102 Clinical Trials

The data described in the presentation are from nine pediatric patients treated with RP-L102, Rocket's ex vivo lentiviral gene therapy candidate for FA.

* RP-L102 demonstrated a highly favorable safety profile with all subjects being treated without conditioning and with no sign of dysplasia or other concerning features. One patient experienced a Grade 2 transient infusion-related reaction.

* Increasing evidence of engraftment was observed in two patients with at least 15-months of follow-up and four patients with at least 6-months of follow-up as indicated by peripheral blood VCN. Patient 2, who was further along in bone marrow failure and also had complications due to a previously disclosed influenza B infection contracted during the early stage of treatment, was withdrawn from the trial at 18-months post-treatment..

* At 24-months post-treatment, Patient 1 demonstrated a 16% BM progenitor resistance to 10 nM MMC and peripheral blood VCN levels of 0.32.

* At 15-months post-treatment, Patient 3 demonstrated a 29% BM progenitor resistance to 10 nM MMC and peripheral blood VCN levels of 0.12.

* At 6-months post-treatment, Patients 4 and 5 had early evidence of BM progenitor resistance with 25% and 14% respectively to 10 nM MMC, consistent with the BM VCNs observed at this timepoint. Patient 4 and 5 also demonstrated peripheral blood VCN levels of 0.07 and 0.06, respectively.

* Patient 6 demonstrated peripheral blood VCN levels of 0.08 at 4-months post-treatment and Patient 7 demonstrated peripheral blood VCN levels of 0.04 at 6-months post-treatment. BM assessments in Patients 6 and 7 were deferred due to COVID-19 travel concerns.

* Patients 8 and 9 were treated more recently with less than 6-months follow-up, and one demonstrated detectable VCN.

Presentation Details:Session: Hematologic and Immunologic Diseases

Presenter: Agnieszka Czechowicz, M.D., Ph.D., Assistant Professor of Pediatrics, Division of Stem Cell Transplantation, Stanford University School of Medicine

Date: Tuesday, May 11, 2021

Time: 8:00-10:00 a.m. EDT

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results

The data presented in the oral presentation are from four pediatric patients with severe LAD-I, as defined by CD18 expression of less than 2%, who were treated with RP-L201, Rocket's ex-vivo lentiviral gene therapy candidate. The safety profile of RP-L201 appears favorable with all infusions well tolerated and no drug product-related serious adverse events (SAEs) .

Preliminary efficacy was evident in all four patients, including two patients with at least 9-months of follow-up. All four patients demonstrated CD18 expression consistent with the reversal of severe LAD-I phenotype.

* 18-months post-treatment, Patient 1001 demonstrated durable CD18 expression of ~40% and resolution of skin lesions with no new lesions reported; 12-months post-treatment, peripheral blood VCN levels were 1.2.

* 9-months post-treatment, Patient 1004 demonstrated CD18 expression of 28%; 6-months post-treatment, peripheral blood VCN levels were 0.75 with kinetics consistent with those of Patient 1.

* 6-months post-treatment, Patient 2006 demonstrated CD18 expression of 74%; 3-months post-treatment, peripheral blood VCN levels were 2.3 with kinetics consistent with those of Patient 1.

* 3-months post-treatment, Patient 2005 demonstrated CD18 expression of 51%; 2-months post-treatment, peripheral blood VCN levels were 0.8 with kinetics consistent with those of Patient 1.

Most importantly, each of these patients were able to leave the hospital in the weeks following RP-L201 therapy, and all have been at home without any serious or severe infections following hospital discharge.

Presentation Details:Session: Genetic Blood and Immune Disorders

Presenter: Donald Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los Angeles

Date: Tuesday, May 11, 2021

Time: 6:15-6:30 p.m. EDT

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Updated Results of a Global Phase 1 Study for Adult and Pediatric Patients

The data presented in the oral presentation are from two adult patients with significant anemia and transfusion requirements. The patients were treated with RP-L301, Rocket's ex vivo lentiviral gene therapy candidate for PKD. RP-L301 continued to be well tolerated, with no serious safety issues or infusion-related complications observed up to 9-months post-treatment.

Preliminary efficacy was evident in both patients during the initial 9-months and 3-months post-treatment, respectively.

* Patient 1 received a CD34+ cell dose of 3.9x106 cells/kilogram (kg). At 9-months post-treatment the patient demonstrated normalized hemoglobin levels of 13.1 g/dL, compared to an average pre-treatment baseline of ~7.4 grams (g)/deciliter (dL) . Peripheral blood VCN levels were 2.14 at 6-months post-treatment.

* Patient 2 received a CD34+ cell dose of 2.4x106 cells/kg. At 6-months post-treatment the patient demonstrated normalized hemoglobin levels of 14.4g/dL, compared to a pre-treatment baseline of ~7.0 g/dL. Peripheral blood VCN levels were 2.55 at 3 months post-RP-L301 infusion.

Presentation Details:Session: Gene Therapies for Hemoglobinopathies

Presenter: Jos? Luis L?pez Lorenzo, M.D., Hospital Universitario Fundaci?n Jim?nez D?az, Madrid, Spain

Date: Wednesday, May 12, 2021

Time: 6:45-7:00 p.m. EDT

In addition, the following presentations at this year's conference also detail results from Rocket Pharma clinical studies:

Title: Gene Therapy in Fanconi Anemia: Follow-Up of a Phase I/II Gene Therapy Trial in Patients with Fanconi Anemia, Subtype A

Session: Genetic Blood and Immune Disorders

Presenter: Juan A. Bueren, Ph.D., Head of the Hematopoietic Innovative Therapies Division at the Centro de Investigaciones Energ?ticas, Medioambientales y Tecnol?gicas (CIEMAT) in Spain / CIBER-Rare Diseases / IIS-Fundaci?n Jim?nez D?az

Date: Tuesday, May 11, 2021

Time: 5:30-5:45 a.m. EDT

Select results from Dr. Bueren's presentation will also be highlighted by Paula Rio, Ph.D. Details for this Invited Presentation are as follows:

Title: Gene Therapy in Fanconi Anemia: Current Strategies to Enable the Correction of HSCs

Session: International Focus on Stem Cell Gene Therapy

Presenter: Paula R?o, Ph.D., Senior Researcher, Hematopoietic Innovative Therapies Division at CIEMAT in Spain / CIBER-Rare Diseases / IIS-Fundaci?n Jim?nez D?az

Date: Thursday May 13, 2021

Time: 10:00-11:45 a.m. EDT

Title: LV-Mediated Gene Therapy of Pyruvate Kinase Deficiency

Session: Cutting Edge Gene and Cell Therapy Research in Europe (Organized by ESGCT)

Presenter: Jose-Carlos Segovia, Head of the Differentiation and Cytometry Unit, Hematopoietic Innovative Therapies Division at CIEMAT in Spain / CIBER-Rare Diseases / IIS-Fundaci?n Jim?nez D?az

Date: Wednesday May 12, 2021

Time: 10:52-11:18 a.m. EDT