On August 9, 2021 Athersys, Inc. (NASDAQ: ATHX) reported its financial results for the three months ended June 30, 2021 and provided a corporate update (Press release, Athersys, AUG 9, 2021, View Source [SID1234586126]).

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"We are happy about the positive topline results for the ONE-BRIDGE ARDS study in Japan reported by Healios last week," stated Mr. William (B.J.) Lehmann, Jr., Interim Chief Executive Officer of Athersys. "The results are consistent with results from our previous MUST-ARDS trial in the United States and the United Kingdom and provide further support for the potential therapeutic benefit for ARDS patients treated with MultiStem cell therapy."

"Additionally, we were pleased to announce new agreements with Healios to improve our collaboration as the MultiStem therapy approaches commercialization following potential regulatory approvals in Japan," added Mr. Lehmann. "Healios will take on greater responsibility and investment with respect to product supply in Japan, which we will continue to support, while we deepen our focus on advanced manufacturing technologies and higher volume manufacturing. In addition, by expanding the scope of the license in Japan, we have created opportunities for both parties to realize additional returns on their investments in this innovative therapy."

Highlights of the second quarter of 2021 and recent events include:

Partner HEALIOS K.K. (Healios) announced positive topline data from its ONE-BRIDGE clinical trial in Japan evaluating the safety and efficacy of MultiStem cell therapy (HLCM051; invimestrocel) in patients with pneumonia-induced and COVID-induced acute respiratory distress syndrome (ARDS);
Expanded our partnership with Healios to optimize and better align the collaboration structure to drive the commercial success and therapeutic reach for MultiStem therapy in Japan;
Published in Scientific Reports important new data about the relevance of MultiStem’s mechanism of action in critical care applications – specifically, the role of these cells in promoting regulatory T cell (Treg) differentiation and proliferation and modulation of immune response;
Advanced our large-scale bioreactor manufacturing platform and initiated efforts to establish GMP production capacity including necessary supply chain and support capabilities;
Continued to build an experienced, world-class team with important hires in regulatory, supply chain, and operations, including the addition of Mr. James Glover as Senior Vice President of Commercial Manufacturing;
Recognized net loss of $22.6 million, or $0.10 net loss per share, for the quarter ended June 30, 2021; and
Ended the second quarter with $56.7 million of cash and cash equivalents.
"We continue to advance our clinical programs with a priority focus on our MASTERS-2 ischemic stroke trial and on the establishment of core capabilities and partnerships necessary for driving commercial success following approval. With important read-outs ahead of us, including Healios’ TREASURE stroke study and our MASTERS-2 study, we are planning and preparing for success," concluded Mr. Lehmann.

Second Quarter Results

There were no revenues for the three months ended June 30, 2021 compared to $0.1 million for the three months ended June 30, 2020. Our collaboration revenues currently fluctuate from period to period based on the delivery of goods and services under our arrangement with Healios.

Research and development expenses increased to $17.7 million for the three months ended June 30, 2021 from $13.8 million for the comparable period in 2020. The $3.9 million increase is associated with increases in clinical trial and manufacturing process development costs of $2.6 million, personnel costs of $0.8 million, facilities costs of $0.3 million and other costs of $0.2 million. Our clinical development, clinical manufacturing and manufacturing process development expenses vary over time based on the timing and stage of clinical trials underway, manufacturing campaigns for clinical trials and manufacturing process development projects.

General and administrative expenses decreased slightly to $4.2 million for the three months ended June 30, 2021 from $4.4 million in the comparable period in 2020. The $0.2 million decrease was primarily related to decreased stock compensation expense.

Net loss for the second quarter of 2021 was $22.6 million compared to a net loss of $18.4 million in the second quarter of 2020. The difference primarily results from the above variances.

During the six months ended June 30, 2021, net cash used in operating activities was $37.2 million compared to $24.9 million in the six months ended June 30, 2020. At June 30, 2021, we had $56.7 million in cash and cash equivalents, compared to $51.5 million at December 31, 2020.

Conference Call

Members of the management will host a conference call today to review the results as follows:

We encourage shareholders to listen using the webcast link above. If you would like to dial in using the phone to ask a question, please register for the conference call ahead of time using the call registration link above. Once registered, you will receive the toll-free number, a direct entry passcode and a registrant ID.

A replay of the event will be available on the webcast link at www.athersys.com under the investors’ section approximately two hours after the call has ended. Shareholders may also call in for on-demand listening approximately three hours after the completion of the call until 11:59 PM Eastern Time on August 16, 2021, by dialing (800) 585-8367 or (416) 621-4642 and entering the conference code 5972392.

On August 9, 2021 Inhibrx, Inc. (Nasdaq: INBX), a biotechnology company with four clinical programs in development, reported financial results for the second quarter of 2021 (Press release, Inhibrx, AUG 9, 2021, View Source [SID1234586125]).

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Financial Results

Cash and Cash Equivalents. As of June 30, 2021, Inhibrx had cash and cash equivalents of $125.7 million, compared to $128.7 million as of December 31, 2020.

R&D Expense. Research and development expenses were $17.9 million during the second quarter of 2021, compared to $19.0 million during the second quarter of 2020. This overall decrease was primarily due to the timing of work performed by Inhibrx’s contract development and manufacturing organization partners for the formulation and manufacturing of certain of its therapeutic candidates, offset in part by an increase in headcount and personnel-related costs due to the continued expansion of its organization.

G&A Expense. General and administrative expenses were $2.9 million during the second quarter of 2021, compared to $1.5 million during the second quarter of 2020. This increase was primarily due to an increase in personnel-related costs and other expenses associated with operating as a public company following its initial public offering in August 2020.

Net Loss. Net loss was $20.7 million during the second quarter of 2021, or $0.55 per share, compared to $17.9 million during the second quarter of 2020, or $0.99 per share.
About the Inhibrx sdAb Platform

Inhibrx utilizes diverse methods of protein engineering in the construction of therapeutic candidates that can address the specific requirements of complex target and disease biology. A key tool for this effort is the Inhibrx proprietary sdAb platform, which enables the development of therapeutic candidates with attributes superior to other monoclonal antibody and fusion protein approaches. This platform allows the combination of multiple binding units in a single molecule, enabling the creation of therapeutic candidates with defined valency or multiple specificities that can achieve enhanced cell signaling or conditional activation. An additional benefit of this platform is that these optimized, multi-functional entities can be manufactured using the established processes that are commonly used to produce therapeutic proteins.

On August 9, 2021 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company developing first-in-class antibody product candidates focused on emerging immune control mechanisms applicable to inflammation and immuno-oncology indications, reported operating results for the second quarter ended June 30, 2021 and provided pipeline updates (Press release, AnaptysBio, AUG 9, 2021, View Source [SID1234586124]).

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"We continue to make progress in advancing our wholly-owned pipeline and look forward to multiple clinical data readouts over the upcoming 18 months," said Hamza Suria, president and chief executive officer of AnaptysBio. "The recent approval of JEMPERLI, which is the first AnaptysBio-generated antibody to be approved in the US and EU, validates AnaptysBio’s antibody discovery platform and provides additional revenues to support our capital-efficient business model."

Imsidolimab (Anti-IL-36 Receptor) Program

Following an end-of-Phase 2 meeting with the FDA in Q2, we publicly disclosed trial designs for our imsidolimab generalized pustular psoriasis (GPP) Phase 3 trials, called GEMINI-1 and GEMINI-2. We anticipate initiating GEMINI-1 in Q3 2021. The primary endpoint of our Phase 3 program is the proportion of patients achieving clear or almost clear skin as determined by a Generalized Pustular Psoriasis Physician’s Global Assessment (GPPPGA) score of zero or 1 at week 4 of GEMINI-1, while GEMINI-2 is designed for 6 months of safety follow-up assessment.
We continue to enroll GPP patients in our worldwide registry of GPP patients, called RADIANCE, which is designed to improve our understanding of GPP patient journeys and support enrollment of our GEMINI Phase 3 trials. Medical claims analyses recently conducted by IQVIA indicate approximately 37,000 unique patients were diagnosed with GPP at least once, and approximately 15,000 unique patients were diagnosed with GPP at least twice, in the United States by a physician between 2017 and 2019 using the International Classification of Diseases 10th Revision (ICD-10) billing code pertaining to GPP (L40.1).
Full data from our completed Phase 2 GALLOP trial of imsidolimab in GPP, including efficacy and safety of imsidolimab treatment through week 16, will be disclosed in an oral presentation at the European Academy of Dermatology and Venereology (EADV) Congress on October 2nd, 2021.
We are continuing to advance imsidolimab through Phase 2 clinical trials in multiple additional indications associated with IL-36 signaling dysfunction. Our 120-patient placebo-controlled ACORN Phase 2 trial of imsidolimab in moderate-to-severe acne is anticipated to read out top-line data in the first half of 2022. Imsidolimab is also being tested versus placebo in hidradenitis suppurativa, where our 120-patient HARP trial is anticipated to generate top-line data in the second half of 2022. We continue to enroll our 45-patient placebo-controlled EMERGE Phase 2 trial of imsidolimab in EGFR/MEK-mediated skin toxicities, where we anticipate an interim analysis by the end of 2021. We also continue to enroll patients in our INSPIRE Phase 2 trial in ichthyosis where top-line data is anticipated during 2022.
Rosnilimab (Anti-PD-1 Agonist) Program

We anticipate top-line data in Q4 2021 from our ongoing Phase 1 healthy volunteer clinical trial of rosnilimab, our wholly-owned PD-1 agonist antibody, designed to assess the safety, pharmacokinetics and pharmacodynamics of rosnilimab in single and multiple ascending dose cohorts.
We plan to initiate a placebo-controlled Phase 2 clinical trial of rosnilimab in alopecia areata in Q4 2021.
ANB032 (Anti-BTLA Modulator) Program

We are advancing ANB032, our wholly-owned BTLA modulator antibody, in a healthy volunteer Phase 1 single and multiple ascending dose clinical trial where top-line data is anticipated during the first half of 2022.
GSK Partnered Programs

JEMPERLI (dostarlimab), our proprietary anti-PD-1 antagonist antibody, was approved by the FDA and the European Medicines Agency (EMA) during April 2021 for treatment of advanced or recurrent mismatch repair deficient endometrial cancer. This is the first AnaptysBio-generated antibody, of eight currently under clinical development, to obtain regulatory approval. GSK has recently disclosed peak annual sales estimates of £1-£2 billion for JEMPERLI, and AnaptysBio will earn 8-25% royalties on global net sales of JEMPERLI. We received $20 million and $10 million milestone payments upon FDA and EMA approval of JEMPERLI, respectively. We anticipate earning an additional $20 million milestone payment upon a second FDA BLA approval for JEMPERLI in pan-deficient mismatch repair tumors during the second half of 2021. AnaptysBio is due an additional $15 million and $165 million upon certain JEMPERLI regulatory and commercial milestones, respectively.
Second Quarter Financial Results

Cash, cash equivalents and investments totaled $396.3 million as of June 30, 2021, compared to $411.2 million as of December 31, 2020, for a decrease of $14.9 million. The decrease relates primarily to cash used for operating activities.
Collaboration revenue was $30 million and $41.3 million for the three and six months ended June 30, 2021. The $30 million earned during the second quarter relates to milestone revenue for the US and EU approval of JEMPERLI (dostarlimab), compared to zero and $15 million of milestone revenue for the three and six months ended June 30, 2020.
Research and development expenses were $25.3 million and $49.5 million for the three and six months ended June 30, 2021, compared to $17.9 million and $38.9 million for the three and six months ended June 30, 2020. The increase was due primarily to continued advancement of the Company’s clinical programs.
General and administrative expenses were $5.2 million and $10.7 million for the three and six months ended June 30, 2021, compared to $4.7 million and $9 million for the three and six months ended June 30, 2020. The increase was due primarily to personnel-related expenses, including share-based compensation.
Net loss was $0.4 million and $18.6 million for the three and six months ended June 30, 2021, or a net loss per share of $0.02, and $0.68, compared to a net loss of $21.5 million and $29.8 million for the three and six months ended June 30, 2020, or a net loss per share of $0.79 and $1.09.
Financial Guidance

AnaptysBio expects its net cash burn in 2021 will be less than $100 million. We anticipate that our cash, cash equivalents and anticipated revenues will fund our current operating plan at least into 2024.

On August 9, 2021 Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs), reported financial results for the second quarter and six months ended June 30, 2021 and highlighted recent corporate progress (Press release, Avidity Biosciences, AUG 9, 2021, View Source [SID1234586123]).

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"The FDA clearance to proceed with our AOC 1001 Phase 1/2 MARINA trial in adults with myotonic dystrophy (DM1) is a huge milestone for Avidity and our AOC platform. AOC 1001 will be the first program based on our novel technology to enter clinical development. This also marks an important step forward for the DM1 community who have no approved therapies and so desperately needs therapeutic options," said Sarah Boyce, president and chief executive officer. "In addition, over the past quarter we made significant advances in our pipeline including nominating AOC 1044 as the clinical development candidate for our lead DMD program. We remain on track for both AOC 1044 and our AOC FSHD program to advance into the clinic in 2022."

"We are well funded with $280 million at the end of Q2’21, along with an additional $155 million in estimated net proceeds from our successful financing in August 2021. Our strong financial position allows us to further progress our late stage programs while continuing to invest in our skeletal muscle pipeline and our AOC platform," said Mike MacLean, chief financial officer.

AOC Platform and Pipeline Highlights

Received FDA clearance to proceed with clinical studies for AOC 1001 in adults with DM1.

Avidity recently received clearance to proceed with the Phase 1/2 MARINA trial of AOC 1001 in adults with DM1, under its Investigational New Drug application (IND). Avidity continues to be on track to initiate the Phase 1/2 MARINA clinical trial this year. In the second half of 2022, the Company plans to conduct a preliminary assessment of safety, tolerability and key biomarkers.

Detailed information on the MARINA study was presented during Volume 2 of Avidity’s virtual investor and analyst event series. Volume 2 featured presentations on AOC 1001, the MARINA study and a presentation on the clinical impact of DM1 by Nicholas E. Johnson, MD, MSCI, FAAN, an associate professor, division chief of neuromuscular, and vice chair of research in the department of neurology at Virginia Commonwealth University. The event also featured a live Q&A session with Avidity’s management team and Dr. Johnson. Volume 2 in the series follows Volume 1 which was focused on the years of engineering underpinning Avidity’s AOC platform and AOC 1001. Replays of Volume 1 and Volume 2 can be found on the events page in the investors section of the Avidity website.

In July, the FDA granted Orphan Drug Designation for AOC 1001 for the treatment of DM1. The FDA grants Orphan Drug Designation to novel drugs that seek to treat a rare disease or condition and, if the drug is approved for the designated orphan indication, provides 7 years of market exclusivity, along with certain financial incentives, including tax credits, opportunities for grant funding towards clinical trial costs and FDA user-fee waivers.

In May, Avidity reported results from its IND-enabling toxicology study of AOC 1001. Results from the study showed the highest dose tested was the maximum feasible dose and was the no-observed adverse effect level (NOAEL). The Company did not observe any treatment-related histopathologic toxicity or any changes in safety pharmacology parameters (cardiac, respiratory and neurological). All dose levels in the study produced a greater than 80% reduction in the expression of dystrophy myotonic protein kinase (DMPK) across multiple skeletal muscles, demonstrating that pharmacology was essentially saturated even at the lowest dose tested.
Progressed Skeletal Muscle Pipeline including nominating AOC 1044 to move into IND-enabling studies.

AOC 1044 was recently nominated as the clinical development candidate for the DMD program targeting Exon 44. AOC 1044 is entering into IND-enabling studies and is on track with plans to advance into the clinic in 2022.

Data from the AOC FSHD program targeting facioscapulohumeral muscular dystrophy (FSHD) was presented at the 28th Annual FSHD Society International Research Congress (IRC) in June. The data demonstrated promising preclinical activity in both in vitro and in vivo experiments in the cells of patients with FSHD.
Second Quarter 2021 Financial Results

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $279.5 million as of June 30, 2021, compared to $328.1 million as of December 31, 2020.
Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity’s partnership with Eli Lilly and Company and totaled $2.6 million for the second quarter of 2021 compared with $1.5 million for the second quarter of 2020, and $5.3 million for the first six months of 2021 compared with $2.9 million for the first six months of 2020.
Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were $22.7 million for the second quarter of 2021 compared with $9.0 million for the second quarter of 2020, and $43.4 million for the first six months of 2021 compared with $14.5 million for the first six months of 2020. The increases were primarily driven by the advancement of AOC 1001, AOC 1044 and our AOC FSHD program, as well as costs related to the expansion of our overall research capabilities.
General and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs, and patent filing and maintenance fees. These expenses were $6.3 million for the second quarter of 2021 compared with $2.9 million for the second quarter of 2020, and $12.2 million for the first six months of 2021 compared with $4.9 million for the first six months of 2020. The increases were primarily due to higher personnel costs (including noncash stock-based compensation), professional fees and insurance costs related to being a public company.

On August 9, 2021 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported that financial results for the quarter ending June 30, 2021 and updates on the Company’s key pipeline developments, business operations, and upcoming milestones (Press release, Rocket Pharmaceuticals, AUG 9, 2021, View Source [SID1234586122]).

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"We are grateful to the FDA for its support and for working with us on our Danon program toward resuming our trial, which we believe will occur in the third quarter," said Gaurav Shah, M.D., chief executive officer of Rocket Pharma. "Further, we have observed durable clinical benefit in the low dose adult cohort, which we believe is supportive of its potential as a viable Phase 2 dose. As of July 2021, we see improvement in two of three low dose patients in NYHA class. In these two patients, we also observed substantial improvement of a key marker of heart failure, BNP, which decreased from a pretreatment baseline by 75 percent in one patient and 79 percent in the other as well as improvement in cardiac output by 35 percent in one patient and 62 percent in the other as measured by invasive hemodynamics. The third patient has demonstrated stabilization of NYHA class and BNP. Given the positive benefit/risk profile in the low dose, and additionally to mitigate safety concerns observed at the higher dose, in agreement with FDA we will no longer treat Danon patients with the higher dose (1.1e14). With our full focus on the low dose, we look forward to progressing our trial on behalf of Danon patients devastated by this disease."

Dr. Shah continued, "We also presented positive data across three of our lentiviral-based gene therapy programs at ASGCT (Free ASGCT Whitepaper) in May, which we believe support the growing potential of these programs to treat Fanconi Anemia (FA), LAD-1 and PKD patients. Based on these results, we continue our momentum toward advancing these programs. In the case of our FA program, two recent publications reinforce the natural history, clinical design and methods being utilized in our Phase 1/2 FA trial of RP-L102. We look forward to providing updates on all five of our programs in the fourth quarter of 2021."

Dr. Shah added, "Finally, and importantly, we are deeply saddened that the first patient dosed in our Phase 1 IMO trial has passed way from pulmonary hemorrhage related to thrombocytopenia following conditioning therapy and also related to underlying osteopetrosis. This event was considered likely not related to RP-L401 gene therapy. Consistent with the trial protocol, enrollment has been temporarily paused pending a comprehensive evaluation in collaboration with the Independent Data Monitoring Committee, which will include a review of the conditioning regimen and other potential safety measures to mitigate the impact of underlying disease on treatment. This outcome underscores the need to find cures for this devastating disease and has furthered our commitment and dedication to patients and our mission to develop curative gene therapies for rare disease."

Key Pipeline and Operational Updates

Danon Disease:

Progressed toward agreement with FDA on changes to the Phase 1 clinical trial protocol in Danon Disease. Rocket anticipates trial may resume this quarter. The U.S. Food and Drug Administration (FDA) had previously requested Rocket pause patient dosing in the Phase 1 clinical trial of RP-A501 and modify the protocol and other supporting documents with revised guidelines for patient selection and management. No new drug-related safety events were observed in the low- or higher-dose adult cohorts; the previously disclosed SAE of thrombotic microangiopathy, which has since resolved, was reclassified as a SUSAR. All follow-up study activities continue. Longer-term results from the low (6.7e13) and higher dose (1.1e14) adult cohorts will be reported in the fourth quarter. In agreement with FDA, Rocket will no longer continue dosing patients at the higher dose (1.1e14).
Fanconi Anemia (FA):

Presented positive clinical updates from RP-L102 Fanconi Anemia (FA) program at ASGCT (Free ASGCT Whitepaper). Preliminary results from the Phase 1 and 2 trials presented in a poster at ASGCT (Free ASGCT Whitepaper) are from nine pediatric patients. For RP-L102, Rocket’s ex vivo lentiviral gene therapy candidate for FA, increasing evidence of engraftment was observed in at least six of the nine patients, including two patients with at least 15-months of follow-up and four patients with at least 6-months of follow-up. A highly favorable tolerability profile was also observed with all subjects being treated without conditioning and with no reports of dysplasia. One patient experienced a Grade 2 transient infusion-related reaction. The full data presented are available here.
Published two peer-reviewed studies supporting the natural history and clinical design of FA clinical trial. "Natural gene therapy by reverse mosaicism leads to improved hematology in Fanconi anemia patients" was published in the American Journal of Hematology. Data strengthen the natural history of Fanconi Anemia and indicate that reverse mosaicism is a good prognostic factor in FA and is associated with more favorable long-term clinical outcomes. FA mosaicism in hematopoietic cells is a biologic and clinical proof-of principle for autologous gene therapy in FA patients and results provide a compelling rationale for continued clinical evaluation of autologous gene therapy. Additionally, "Improved Collection of Hematopoietic Stem Cells and Progenitors from Fanconi Anemia Patients for Gene Therapy Purposes" was published in Molecular Therapy: Methods & Clinical Development. Results demonstrate the safety and efficacy of filgrastim and plerixafor for mobilization of hematopoietic stem and progenitor cells (HSPCs) and collection by leukapheresis in FA patients, offering crucial information for the enrollment of FA patients for gene therapy studies.
Infantile Malignant Osteopetrosis (IMO):

First patient dosed in the RP-L401 Infantile Malignant Osteopetrosis (IMO) Phase 1 clinical trial passed away from likely non-RP-L401 gene therapy related pulmonary complications. IMO is a bone marrow-derived disorder associated with severe bone and hematologic manifestations leading to death in the first decade of life, frequently within the first two years of life, without an allogenic hematopoietic stem cell transplant (HSCT).

The first patient in the Phase 1 study, a six-year-old child with severe IMO-related anemia and bone abnormalities, was infused with RP-L401 without immediate complications. During the initial weeks after therapy, the patient died of pulmonary complications, most likely pulmonary hemorrhage related to thrombocytopenia following conditioning therapy and also related to underlying osteopetrosis. Pulmonary hemorrhage is a rare but documented complication of HSCT, and pulmonary complications, including life-threatening and fatal complications, have been observed to occur with high frequency in osteopetrosis patients undergoing allogeneic HSCT procedures.

The patient death is not considered to be RP-L401-related by study investigators and as corroborated by autopsy findings. In accordance with the trial protocol, enrollment has been temporarily paused pending a comprehensive evaluation in collaboration with the Independent Data Monitoring Committee.
Leukocyte Adhesion Deficiency-I (LAD-I):

Presented positive clinical updates from RP-L201 Leukocyte Adhesion Deficiency-I (LAD-I) program at ASGCT (Free ASGCT Whitepaper). Phase 1/2 data presented in an oral presentation at ASGCT (Free ASGCT Whitepaper) are from four pediatric patients with severe LAD-I. RP-L201, Rocket’s ex-vivo lentiviral gene therapy candidate showed preliminary activity in all four patients, including one patient with 18-months of follow-up and one patient with 9-months of follow-up. CD18 expression substantially exceeded the 4-10% threshold in all four patients, which is associated with survival into adulthood and consistent with the reversal of severe LAD-I phenotype. Most importantly, all four patients were able to leave the hospital in the weeks following RP-L201 therapy. The full data presented are available here.
Pyruvate Kinase Deficiency (PKD):

Presented positive clinical updates from RP-L301 Pyruvate Kinase Deficiency (PKD) program at ASGCT (Free ASGCT Whitepaper). Updated preliminary Phase 1 data presented in an oral presentation at ASGCT (Free ASGCT Whitepaper) are from two patients with significant anemia and transfusion requirements that showed sustained tolerability. Preliminary activity, measured by peripheral blood VCN levels, was observed in both patients during the initial 9-months and 3-months post-treatment, respectively. Durable normalization of hemoglobin levels were observed, from an average baseline of ~7.4 grams (g)/deciliter (dL) to 13.1 g/dL at 9-months post treatment in the first patient and from a baseline of ~7.0 g/dL to 14.4 g/dL at 6-months post treatment in the second patient. The Phase 1 trial continues to enroll patients with longer-term data expected in the fourth quarter. The full data presented are available here.
Anticipated Milestones

Fanconi Anemia (RP-L102)
Updated "Process B" data (Q4 2021)
LAD-I (RP-L201)
Longer-term Phase 2 data (Q4 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)
Phase 1 clinical update (Q4 2021)
Upcoming Investor Conference

Citi’s 16th Annual BioPharma Virtual Conference, Sept. 8-10, 2021
Second Quarter Financial Results

Cash position. Cash, cash equivalents and investments as of June 30, 2021 were $426.8 million.
R&D expenses. Research and development expenses were $24.8 million for the three months ended June 30, 2021, compared to $16.7 million for the three months ended June 30, 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 $9.3 million for the three months ended June 30, 2021, compared to $6.8 million for the three months ended June 30, 2020, due to an increase in non-cash stock compensation expense, an increase in compensation and benefits expense due to increased G&A headcount and an increase in office and administrative costs.
Net loss. Net loss was $34.5 million or $0.55 per share (basic and diluted) for the three months ended June 30, 2021, compared to $25.0 million or $0.45 per share (basic and diluted) for the three months ended June 30, 2020.
Shares outstanding. 63,448,069 shares of common stock were outstanding as of June 30, 2021.
Financial Guidance

Rocket expects its balance in cash, cash equivalents and investments of $426.8 million as of June 30, 2021 to fund its operations into the second half of 2023, including the continued buildout and initiation of AAV cGMP manufacturing capabilities at our Cranbury, New Jersey R&D and manufacturing facility and continued development of our five clinical programs.
Conference Call Details

Rocket management will host a conference call today at 4:30 p.m. ET. 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 access the conference 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 50210581.