On May 5, 2022 Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) reported financial and operational results for the first quarter ended March 31, 2022 (Press release, Vanda Pharmaceuticals, MAY 5, 2022, View Source [SID1234613722]).

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"We are excited with our progress in improving patient access for HETLIOZ, especially for Medicaid beneficiaries with Non-24 and SMS diagnoses," said Mihael H. Polymeropoulos, M.D., Vanda’s President, CEO and Chairman of the Board. "Further, we now anticipate completing enrollment of the Fanapt bipolar disorder study by the end of this year and look forward to expanding our psychiatry franchise. Today we are sharing details of our advanced analysis of data from the tradipitant clinical program in gastroparesis, which support the efficacy of tradipitant, and we look forward to discussing our planned New Drug Application with the FDA."

Financial Highlights

Total net product sales from HETLIOZ and Fanapt were $60.2 million in the first quarter of 2022, a 4% decrease compared to $62.7 million in the first quarter of 2021.
HETLIOZ net product sales were $37.0 million in the first quarter of 2022, a 6% decrease compared to $39.3 million in the first quarter of 2021, due in part to the continued reimbursement challenges for prescriptions for patients with Non-24.
Fanapt net product sales were $23.2 million in the first quarter of 2022, a 1% decrease compared to $23.3 million in the first quarter of 2021.
Net loss was $6.4 million in the first quarter of 2022 compared to net income of $8.7 million in the first quarter of 2021.
Cash, cash equivalents and marketable securities (Cash) was $435.2 million as of March 31, 2022, representing an increase to Cash of $57.0 million compared to March 31, 2021 and an increase to Cash of $2.4 million compared to December 31, 2021.
Key Operational Highlights

HETLIOZ (tasimelteon)

Clinical trials for HETLIOZ in delayed sleep phase disorder (DSPD) and symptoms of autism spectrum disorder (ASD) are currently enrolling patients.
Since November 2021, more than 15 states have revised or agreed to revise their Medicaid prior authorization criteria to broaden access to HETLIOZ for patients with Non-24 and nighttime sleep disturbances in Smith-Magenis Syndrome (SMS).
In January 2022, Vanda settled its HETLIOZ patent litigation against one of the defendants. The trial for the consolidated lawsuit against the remaining defendants was held in March 2022. A decision is expected from the court in the second half of 2022.
Tradipitant

Tradipitant is a neurokinin-1 (NK-1) receptor antagonist in development for the treatment of idiopathic and diabetic gastroparesis. Earlier in the year, Vanda reported results of a clinical study and initial exploratory analysis. Vanda has now completed a pooled analysis of two clinical studies of tradipitant in gastroparesis consisting of 342 patients with relevant clinical endpoints. We believe these studies are adequate and well controlled and support substantial evidence of efficacy of tradipitant. Figure 1 and Table 1 show the results of such pooled analysis of all patients randomized in the two studies (intent to treat population, ITT) and Figure 2 and Table 2 show the results for the same parameters in the population of patients who were judged as compliant to treatment based on analysis of drug exposure (treatment compliant population).

Consistently, both pooled analyses show tradipitant to be superior to placebo in key clinical parameters including improvement in DD-Nausea (primary endpoint parameter), percent Nausea Free Days, Patient Global Impression scale change (PGI-C), Overall Benefit Score and Gastroparesis Cardinal Symptom Index (GCSI) score.

Vanda is continuing to conduct an open-label study of safety for tradipitant in gastroparesis and continues to receive requests from patients reaching out to gain access to tradipitant through the Expanded Access program which has multiple patients continuing to take tradipitant for more than a year.
Vanda has scheduled a pre-NDA meeting with the U.S. Food and Drug Administration (FDA) to discuss the planned New Drug Application submission for tradipitant in the short-term treatment of nausea in gastroparesis.
The Phase III study of tradipitant in the treatment of motion sickness has restarted enrollment and is already over 15% enrolled. A prior Phase II study of tradipitant in the treatment of motion sickness observed a significantly lower incidence of vomiting in tradipitant-treated patients as compared to placebo-treated patients.
Fanapt (iloperidone)

A Phase III study of Fanapt in acute manic episodes in patients with bipolar disorder is over 75% enrolled and expected to complete enrollment by the end of 2022.
GAAP Financial Results

Net loss was $6.4 million in the first quarter of 2022 compared to net income of $8.7 million in the first quarter of 2021. Diluted net loss per share was $0.11 in the first quarter of 2022 compared to diluted net income per share of $0.15 in the first quarter of 2021.

2022 Financial Guidance

Vanda expects to achieve the following financial objectives in 2022:

Conference Call

Vanda has scheduled a conference call for today, Thursday, May 5, 2022, at 4:30 PM ET. During the call, Vanda’s management will discuss the first quarter 2022 financial results and other corporate activities. Investors can call 1-866-688-9426 (domestic) or 1-409-216-0816 (international) and use passcode number 1355275. A replay of the call will be available on Thursday, May 5, 2022, beginning at 7:30 PM ET and will be accessible until Thursday, May 12, 2022 at 7:30 PM ET. The replay call-in number is 1-855-859-2056 for domestic callers and 1-404-537-3406 for international callers. The passcode number is 1355275.

The conference call will be broadcast simultaneously on Vanda’s website, www.vandapharma.com. Investors should click on the Investors tab and are advised to go to the website at least 15 minutes early to register, download, and install any necessary software or presentations. The call will also be archived on Vanda’s website for a period of 30 days.

On May 5, 2022 Cardiff Oncology, Inc. (Nasdaq: CRDF), a clinical-stage biotechnology company leveraging PLK1 inhibition to develop novel therapies across a range of cancers, reported recent company highlights and financial results for the first quarter ended March 31, 2022 (Press release, Cardiff Oncology, MAY 5, 2022, View Source [SID1234613721]).

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"With a strong balance sheet and clinical data highlighting onvansertib’s potential to improve patient outcomes by combining synergistically with the standard-of-care, we believe we are well positioned for success," said Mark Erlander, Ph.D., chief executive officer of Cardiff Oncology. "Phase 1b/2 data from our lead KRAS-mutated metastatic colorectal cancer (mCRC) program show clear improvements in treatment response and durability compared to historical controls. These results also clinically demonstrate onvansertib’s KRAS-agnostic mechanism of action, setting it apart from competing agents that target only the G12C variant."

Dr. Erlander added, "We are also highly encouraged by recent data from our Phase 2 prostate cancer trial showing clinically meaningful disease control rates in patients who showed signs of progression on prior therapy, and preclinical studies that showed onvansertib-PARP inhibitor combination therapy leading to statistically significant survival benefits in patient-derived xenograft models of PARP-inhibitor-resistant ovarian cancer. These results further highlight how onvansertib’s ability to inhibit PLK1 positions it to target tumor vulnerabilities and overcome treatment resistance across a spectrum of indications. We look forward to building on these results as we move towards the second half of the year."

Program highlights for the quarter ended March 31, 2022, and recent business updates include:

KRAS-mutated mCRC Program:

Reported new Phase 1b/2 data showing treatment with onvansertib plus FOLFIRI/bevacizumab leading to an objective response rate (ORR) and median progression-free survival (mPFS) that substantially exceed those recorded in historical control trials

The data were announced on a webcast and conference call hosted by Cardiff Oncology, and a subset were presented by Heinz-Josef Lenz, M.D., FACP, principal investigator, USC Norris Comprehensive Cancer Center, in a poster at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO GI). Highlights from the webcast and conference call include:

Efficacy data in evaluable patients:

Among patients treated per protocol with onvansertib at the recommended Phase 2 dose (RP2D; 15 mg/m2) in combination with FOLFIRI/bevacizumab:
34% (12 of 35) achieved an initial complete response (CR) or partial response (PR)
29% (10 of 35) achieved a confirmed CR or PR (awaiting confirmatory scan for 1 patient)
Historical control trials evaluating different drug combinations, including the standard-of-care of FOLFIRI with bevacizumab, in similar patient populations have shown ORRs of 5-13%1-4
Patients evaluable for response treated at onvansertib dose levels 12 mg/m2, 15 mg/m2, and 18 mg/m2
35% (17 of 48) achieved an initial CR or PR
27% (13 of 48) achieved a confirmed CR or PR (awaiting confirmatory scan for 1 patient)
mPFS, biomarker, and safety data:

mPFS not yet reached in patients treated per protocol at the RP2D
mPFS across all response-evaluable patients (n = 48) is 9.4 months (95% confidence interval: 7.1 – not yet reached)
mPFS of ~4.5-5.7 months has been reported in historical control trials1-4
Complete or partial responses were observed across seven different KRAS mutation variants, including the 3 most commonly observed in colorectal cancer (G12D, G12V, G13D)
Onvansertib in combination with FOLFIRI/bevacizumab has been well-tolerated with only 11% (84/788) of reported treatment-emergent adverse events (TEAEs) being G3/G4
Metastatic Castration-resistant Prostate Cancer (mCRPC) Program:

Announced updated clinical and new biomarker data from Phase 2 trial evaluating onvansertib in combination with abiraterone/prednisone in mCRPC patients showing initial abiraterone resistance

Results from the trial, which were presented in a poster at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, showed clinically meaningful disease control rates that rose with increasing onvansertib dose density. Additional highlights from the announcement include:

75% (15 of 20) evaluable patients in Arm C, which represents the trial’s most dose dense treatment schedule, showed disease control by radiographic stable disease (SD/PR) at 12-weeks, compared to 9 of 17 (53%) and 11 of 19 (58%) in the less dose-dense Arms A and B, respectively
Treatment response (SD/PR) was positively associated with mutations in PTEN and MTOR, key genes in the PI3K signaling pathway
Gene signatures correlating with treatment response included those corresponding to the ERG+ and Notch pathways, which are involved in cell-invasion, epithelial-mesenchymal transition, and metastasis
Genes related to mitochondrial and immune functions were downregulated in patients achieving SD or a PR compared to those showing progressive disease
Onvansertib in combination with abiraterone/prednisone has been well tolerated in the trial
Each arm in the trial has evaluated a different dosing schedule of onvansertib alongside abiraterone and prednisone administered throughout the respective treatment cycle. Arm A evaluated 24 mg/m2 onvansertib on Days 1-5 of 21-day cycles, Arm B evaluated 18 mg/m2 onvansertib on Days 1-5 of 14-day cycles, and Arm C is evaluating 12 mg/m2 onvansertib on Days 1-14 of 21-day cycles.

Preclinical Highlights:

Reported new preclinical data that show onvansertib combining with a PARP inhibitor to overcome PARP inhibitor (PARPi) resistance in BRCA1-mutant and wild-type patient-derived xenograft ovarian cancer models

Preclinical studies featured in a poster presentation at the AACR (Free AACR Whitepaper) Annual Meeting evaluated onvansertib in combination with the PARPi olaparib in three olaparib-resistant patient-derived xenograft (PDX) ovarian cancer models. Two of the three PDX models studied were cisplatin-sensitive with a mutated BRCA1 gene, while the third was cisplatin-resistant with wild type BRCA1. Results showed that treatment with onvansertib plus olaparib led to a statistically significant survival benefit compared to treatment with either agent alone in each of the three evaluated PDX models. The combination regimen was also shown to be well tolerated in mice.

First Quarter 2022 Financial Results:

Liquidity and cash burn

As of March 31, 2022, Cardiff Oncology had approximately $129.4 million in cash, cash equivalents, and short-term investments.

Net cash used in operating activities for the first quarter of 2022 was approximately $10.2 million, an increase of approximately $4.3 million from $5.9 million for the same period in 2021.

The overall increase in research and development expenses was primarily due to costs associated with an increase in outside service costs related to chemistry, manufacturing, and controls ("CMC") and pharmacology for the development of our lead drug candidate, onvansertib. Salaries and staff costs increased due to a higher headcount in the current period, as compared to the prior period. The increase in stock-based compensation is due to additional stock option grants to employees granted subsequent to the prior period.

The overall increase in selling, general and administrative expense was primarily due to costs associated with outside services and stock-based compensation. The increase in outside services is related to strategic valuation consulting related to our lead drug candidate, onvansertib. The increase in stock-based compensation is due to additional stock option grants to employees and directors granted subsequent to the prior period.

On May 5, 2022 Nektar Therapeutics (Nasdaq: NKTR) reported financial results for the first quarter ended March 31, 2022 (Press release, Nektar Therapeutics, MAY 5, 2022, View Source [SID1234613720]).

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Cash and investments in marketable securities at March 31, 2022 were approximately $704.4 million as compared to $798.8 million at December 31, 2021, which is expected to support operations into 2025.

"The new strategic plan that we recently announced refocuses our company around specific investment into our most important pipeline programs – NKTR-358, NKTR-255, and core preclinical candidates," said Howard W. Robin, President and CEO of Nektar. "In addition, we have now implemented a cost restructuring plan which extends our cash runway through the first half of 2025. We believe our pipeline in auto-immune disease and oncology provides a path to bringing important therapeutics to patients and creating value for our shareholders."

Summary of Financial Results
Revenue in the first quarter of 2022 was $24.8 million as compared to $23.6 million in the first quarter of 2021.

Total operating costs and expenses in the first quarter of 2022 were $141.4 million as compared to $133.0 million in the first quarter of 2021. Operating costs and expenses increased primarily as a result of an increase in R&D expense.

R&D expense in the first quarter of 2022 was $107.3 million as compared to $95.6 million for the first quarter of 2021. R&D expense increased primarily due to increases in expense for bempegaldesleukin, NKTR-255 and NKTR-358.

G&A expense was $27.3 million in the first quarter of 2022 and $31.7 million in the first quarter of 2021.

Net loss for the first quarter of 2022 was $90.4 million or $0.49 basic and diluted loss per share as compared to a net loss of $123.0 million or $0.68 basic and diluted loss per share in the first quarter of 2021.

On April 25, 2022, Nektar announced new strategic and cost restructuring plans (View Source) and conducted a call with analysts and investors to present those plans. On that call, the company provided annual financial guidance for 2022, and because of that, the company stated it would not hold its regular quarterly conference call conducted in conjunction with release of Q1 2022 financial results.

On May 5, 2022 Antengene Corporation Limited ("Antengene" SEHK: 6996.HK), a leading innovative, commercial-stage global biopharmaceutical company dedicated to discovering, developing and commercializing first-in-class and/or best-in-class therapeutics in hematology and oncology, reported that the Company will be participating virtually in three upcoming investor conferences (Press release, Antengene, MAY 5, 2022, View Source [SID1234613719]).

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CICC Healthcare Industry Forum 2022

BTIG Virtual China Biotech Day

Citi’s 3rd Pan-Asia Regional Investor Conference 2022

On May 5, 2022 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, reported financial results for the quarter ending March 31, 2022, and updates from the Company’s key pipeline developments, business operations and upcoming milestones (Press release, Rocket Pharmaceuticals, MAY 5, 2022, View Source [SID1234613718]).

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"I am pleased with our first quarter progress as we maintained our strong focus on execution across each of our clinical programs, in-house AAV manufacturing readiness and overall business operations," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. "We are looking forward to this month’s ASGCT (Free ASGCT Whitepaper) meeting, where we will present updates from all four clinical programs. We also remain on track to deliver full data readouts through the rest of the year as planned."

Dr. Shah continued, "In the first quarter, we also bolstered our Company leadership team with the appointments of Fady Malik, M.D., Ph.D., an internationally recognized physician-scientist, to our Board of Directors, global commercial executive Carlos Martin to Chief Commercial Officer and capital markets expert Jessie Yeung to Vice President, Investor Relations and Corporate Finance. Furthermore, we continued to scale up our in-house manufacturing facility and remain on track for AAV cGMP manufacturing initiation in Q2."

"Finally, we extended our cash runway from the second half of 2023 into the first half of 2024 and have a strong balance sheet with liquidity through potential regulatory filings and approvals," said Dr. Shah. "Taken together, we have generated tremendous momentum with our best-in-class gene therapies, utilizing both ex-vivo lentiviral and in-vivo AAV platforms, in our pursuit of cures for patients facing these rare and truly devastating diseases."

Key Pipeline and Operational Updates

Danon, FA, LAD-I and PKD trials have continued to progress and remain on track. All 2022 milestones remain on track including potential top-line readouts for Phase 2 trials in LAD-I and FA in Q2 and Q3, respectively, pediatric efficacy readout for the Phase 1 Danon Disease trial in Q3, and the Phase 1 readout for PKD in Q4.
Updated clinical data to be presented at ASGCT (Free ASGCT Whitepaper). Updates from all four clinical programs to be presented at the 2022 Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) at the Walter E. Washington Convention Center in Washington, D.C., May 16-19.
Details for oral presentations are as follows:

Title: Extended Results from First-In-Human Clinical Trial of RP-A501 (AAV9:LAMP2B) Gene Therapy
Treatment For Danon Disease
Session: Cardiovascular and Pulmonary Diseases
Presenter: Barry Greenberg, M.D., FHFSA, University of California, San Diego Medical Center, La Jolla, CA
Date: Monday, May 16, 2022
Session Time: 10:15 a.m. – 12:00 p.m. ET
Presentation Time: 10:45 a.m. – 11:00 a.m. ET
Location: Room 206
Abstract Number: 24

Title: Ex vivo Lentiviral-mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials
Session: Hematopoietic Stem Cell Gene Therapy
Presenter: Agnieszka Czechowicz, M.D., Ph.D., Center for Definitive and Curative Medicine, Stanford University School of Medicine, Stanford, CA
Date: Monday, May 16, 2022
Session Time: 3:45 p.m. – 5:30 p.m. ET
Presentation Time: 4:15 p.m. – 4:30 p.m. ET
Location: Room 202
Abstract Number: 108

Title: Interim Results from an ongoing Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)
Session: Clinical Trials Spotlight Symposium
Presenter: Donald B. Kohn, M.D., Broad Stem Cell Research Center, UCLA, Los Angeles and recipient of ASGCT (Free ASGCT Whitepaper)’s 2022 Outstanding Achievement Award
Date: Thursday, May 19, 2022
Session Time: 8:00 a.m. – 9:45 a.m. ET
Presentation Time: 8:00 a.m. – 8:15 a.m. ET
Location: Ballroom C
Abstract Number: 1188

Details for the poster presentation are as follows:

Title: Changing the Treatment Paradigm for Pyruvate Kinase Deficiency with Lentiviral Mediated Gene
Therapy: Interim Results from an Ongoing Global Phase 1 Study
Presenter: Ami Shah, M.D., Center for Definitive and Curative Medicine, Stanford University School of Medicine, Stanford, CA
Date: Monday, May 16, 2022
Time: 5:30 p.m. – 6:30 p.m. ET
Location: Hall D
Abstract Number: 357

In-house AAV current Good Manufacturing Practice (cGMP) manufacturing initiation anticipated in Q2. The Company’s state-of-the-art, 103,720 ft2 manufacturing facility in Cranbury, N.J. is being scaled up to manufacture AAV drug product for a planned Phase 2 study in Danon Disease.
Broadened cardiovascular experience of Rocket’s Board of Directors. The Company announced the appointment of Fady Malik, M.D., Ph.D. to its Board of Directors. Dr. Malik brings nearly 25 years of experience as an internationally recognized cardiovascular physician-scientist and highly successful biopharmaceutical executive. Dr. Malik is Executive Vice President of Research and Development at Cytokinetics, a late-stage biopharmaceutical company, where he has worked in a variety of positions since he joined the founders to launch the company.
Named Chief Commercial Officer. In March 2022, Carlos Martin was appointed to Senior Vice President, Chief Commercial Officer. Mr. Martin brings over 20 years of global commercial leadership gained at Novartis, Schering Plough and Eli Lilly. Mr. Martin will lead the go-to-market entry models in the U.S. and EU and build-out of key commercial capabilities to pave the way for potential patient access, reimbursement and treatment of Rocket’s gene therapies.
Appointed Vice President, Investor Relations & Corporate Finance. In March 2022, Jessie Yeung joined the Company as Vice President, Investor Relations. Ms. Yeung brings more than 15 years of investor relations, corporate finance and capital market experience across industries including the biopharmaceutical and financial sectors. Prior to Rocket, Ms. Yeung was the Head of Corporate Finance and Investor Relations at Legend Biotech. Ms. Yeung also has experience as an equity research analyst at Bank of America Merrill Lynch, Wells Fargo, and J.P. Morgan.
Published peer-reviewed expanded categorization of severe PKD. "Who should be eligible for gene therapy clinical trials in red blood cell Pyruvate Kinase Deficiency (PKD)?: Toward an expanded definition of severe PKD" was published in the American Journal of Hematology. The publication summarizes the natural history of the disease and proposes an expanded categorization of severe PKD to help identify the most optimal patients for ongoing gene- and cell-based evaluations.
Recognized Rare Disease Day with an event at NASDAQ Tower in New York City. On February 28, 2022, Rocket hosted its annual Rare Disease Day celebration highlighting the theme, "Rare, But Not Alone." More than 250 members of the global rare disease community and Rocket team gathered in person and virtually to hear about the impact of rare disease and clinical research from patients, families, advocacy groups and scientific collaborators and innovators – including Dr. Moris Danon, founder of Danon Disease. The event concluded with the lighting of the Empire State Building, as well as other global landmarks, in Rare Disease Day colors.
Anticipated 2022 Milestones

RP-A501 for Danon Disease (AAV)

Report data from pediatric patient cohort of Phase 1 trial – Q3 2022
Initiate pivotal Phase 2 trial activities – Q4 2022
RP-L201 for Leukocyte Adhesion Deficiency-I (LVV)

Report top-line data from pivotal Phase 2 trial – Q2 2022
RP-L102 for Fanconi Anemia (LVV)

Report top-line data from pivotal Phase 2 trial – Q3 2022
RP-L301 for Pyruvate Kinase Deficiency (LVV)

Report preliminary Phase 1 data – Q4 2022
Initiate pivotal Phase 2 trial activities – Q4 2022
Manufacturing Facility in Cranbury, New Jersey

Achieve in-house AAV current Good Manufacturing Practice (cGMP) manufacturing readiness – Q2 2022
Upcoming Investor Conference

Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer, will deliver an in-person company presentation at the Bank of America Securities Healthcare Conference on Wednesday, May 11 at 2:20 p.m. ET at the Encore Hotel in Las Vegas.
First Quarter Financial Results

Cash position. Cash, cash equivalents and investments as of March 31, 2022, were $346.6 million.
R&D expenses. Research and development expenses were $30.8 million for the three months ended March 31, 2022, compared to $28.3 million for the three months ended March 31, 2021. The increase in research and development expense was primarily driven by an increase in compensation and benefits expense due to increased R&D headcount, an increase in laboratory supplies and manufacturing development expenses, offset by a decrease in non-cash stock compensation expense.
G&A expenses. General and administrative expenses were $11.7 million for the three months ended March 31, 2022, compared to $10.9 million for the three months ended March 31, 2021. The increase in general and administrative expenses was primarily driven by an increase in compensation and benefits expense due to increased G&A headcount, and an increase in commercial preparation expenses, offset by a decrease in non-cash stock compensation expense.
Net loss. Net loss was $43.0 million or $0.67 per share (basic and diluted) for the three months ended March 31, 2022, compared to $40.2 million or $0.65 per share (basic and diluted) for the three months ended March 31, 2021
Shares outstanding. 64,522,057 shares of common stock were outstanding as of March 31, 2022
Financial Guidance

Cash position. As of March 31, 2022, we had cash, cash equivalents and investments of $346.6 million. In April 2022, the Company sold 1.3 million shares of common stock for net proceeds of $17.3 million under the at-the-market facility. With the at-the-market facility proceeds and other efficiencies, the Company expects such resources will be sufficient to fund its operating expenses and capital expenditure requirements into the first half of 2024, 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 four clinical programs as well as future pipeline programs.