• |
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 (6.7x1013) RP-A501 Treatment Demonstrates Durable Expression and Improvements in Biomarkers. New Data Include:
|
o |
Patient 1002: 78% IHC (revised from previous 67% due to improved technique), BNP continues to decrease to 200 (baseline 943)
|
o |
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
|
o |
Higher Dose (1.1x1014) data will be updated in the fourth quarter
|
o |
Rocket does not anticipate pursuing doses higher than 1.1x1014 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: https://rocketpharma.com/CIS. More comprehensive Phase 2 results will be presented at ASGCT.
|
• |
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.
|
• |
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.
|
• |
Fanconi Anemia (RP-L102)
|
o |
Updated 'Process B' data (Q2 2021)
|
• |
LAD-I (RP-L201)
|
o |
Longer-term Phase 2 data (Q3 2021)
|
• |
Danon Disease (RP-A501)
|
o |
Longer-term Phase 1 data (Q4 2021)
|
• |
PKD (RP-L301)
|
o |
Longer-term Phase 1 data (Q4 2021)
|
• |
IMO (RP-L401)
|
o |
Initial Phase 1 data (Q3 2021)
|
• |
BofA Securities' 2021 Virtual Health Care Conference, May 13, 2021
|
• |
UBS Global Healthcare Virtual Conference, May 26, 2021
|
• |
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.
|
• |
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.
|
Three Months Ended March 31,
| ||||||||
2021
|
2020
| |||||||
Operating expenses:
| ||||||||
Research and development
|
$
|
28,542
|
$
|
16,957
| ||||
General and administrative
|
10,680
|
7,163
| ||||||
Total operating expenses
|
39,222
|
24,120
| ||||||
Loss from operations
|
(39,222
|
)
|
(24,120
|
)
| ||||
Research and development incentives
|
500
|
-
| ||||||
Interest expense
|
(1,729
|
)
|
(1,573
|
)
| ||||
Interest and other income net
|
911
|
967
| ||||||
(Amortization of premium) accretion of discount on investments - net
|
(639
|
)
|
62
| |||||
Total other expense, net
|
(957
|
)
|
(544
|
)
| ||||
Net loss
|
$
|
(40,179
|
)
|
$
|
(24,664
|
)
| ||
Net loss per share attributable to common stockholders - basic and diluted
|
$
|
(0.65
|
)
|
$
|
(0.45
|
)
| ||
Weighted-average common shares outstanding - basic and diluted
|
61,574,405
|
54,883,120
|
March 31,
|
December 31,
| |||||||
2021
|
2020
| |||||||
Cash, cash equivalents and investments
|
466,353
|
482,719
| ||||||
Total assets
|
576,484
|
590,824
| ||||||
Total liabilities
|
96,485
|
87,305
| ||||||
Total stockholders' equity
|
479,999
|
503,519
|
SVP, Strategy & Corporate Development
investors@rocketpharma.com
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Disclaimer
Rocket Pharmaceuticals Inc. published this content on 10 May 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 12 May 2021 12:51:09 UTC.