On May 6, 2024 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, reported financial and recent operational results for the quarter ending March 31, 2024 (Press release, Rocket Pharmaceuticals, MAY 6, 2024, View Source [SID1234642685]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Rocket has had a strong start to 2024. This quarter has been marked by meaningful progress across all our clinical programs, most notably with the EMA’s review of RP-L102 for Fanconi Anemia, commercial preparations for the potential approval of KRESLADI for severe LAD-I, and ongoing enrollment in the Phase 2, pivotal study of RP-A501 for Danon Disease and the Phase 1 study of RP-A601 for PKP2-ACM," said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharmaceuticals. "As we build on the advancements made this quarter, we remain focused on execution for patients with rare and devastating diseases with limited treatment options."

Recent Pipeline and Operational Updates

•
Advanced Fanconi Anemia (FA) program through key regulatory milestones.

o
In April, Rocket announced that the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for RP-L102 for the treatment of FA. MAA acceptance was based on positive, previously disclosed data from the global RP-L102 Phase 1/2 clinical trial.

o
The Company remains on track to submit the Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) in the first half of 2024.

•
KRESLADI on track for June 30, 2024, PDUFA date.

o
Rocket continues to ramp up enabling activities to support the launch of its lentiviral (LV) vector portfolio beginning with KRESLADI for severe Leukocyte Adhesion Deficiency-I (LAD-I). Qualified Treatment Center initiation, disease education, payer engagement, and field team build-out are all underway.

•
Bolstered finance, investor relations, and corporate communications expertise of company leadership to support evolution towards commercial stage.

o
Rocket appointed Aaron Ondrey as Chief Financial Officer (CFO). Mr. Ondrey brings over 20 years of experience leading commercial-stage financial management, strategic planning, and capital allocation. Mr. Ondrey was previously the CFO at Mirati Therapeutics and has held multiple senior finance leadership positions at Arena Pharmaceuticals, Alexion Pharmaceuticals, and Regeneron Pharmaceuticals.

o
In addition, Meg Dodge was appointed Vice President to lead Investor Relations & Corporate Communications. Ms. Dodge is experienced with engaging stakeholders across investors, media, and other communities in the biotech and financial sectors. Prior to joining Rocket, Ms. Dodge was Head of Investor Relations and Corporate Communications at Krystal Biotech.

•
Celebrated annual Rare Disease Day with multi-faceted awareness campaign.

o
On February 29, 2024, Rocket hosted its annual Rare Disease Day recognition program highlighting the theme, "Leap into Action for Rare." Several hundred attendees gathered at the Liberty Science Center and virtually to hear inspirational stories from the community. Rocket also continued its Light Up for Rare initiative in collaboration with global partners to light up buildings and landmarks in Rare Disease Day colors, including the Empire State Building.

o
Rocket remains highly committed to supporting the rare disease community through patient-focused events, education and advancing science to bring potential treatments to patients with unmet needs.

•
Milestones in 2024 are on track across its pipeline of gene therapies for rare and devastating diseases.

o
Rocket continues to advance three disclosed programs from its adeno-associated virus (AAV) cardiovascular portfolio, including:

■
Phase 2 pivotal study of RP-A501 for Danon Disease,

■
Phase 1 study of RP-A601 for PKP2-arrhythmogenic cardiomyopathy (ACM), and

■
IND-enabling studies for BAG3-associated dilated cardiomyopathy (DCM)

o
In its late-stage LV portfolio, Rocket is working towards initiation of the Phase 2 pivotal study of RP-L301 for Pyruvate Kinase Deficiency (PKD).

•
Data from Rocket’s LV hematology portfolio to be presented at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 27th Annual Meeting.

o
Updated data across Rocket’s LV hematology programs will be highlighted as oral presentations at the ASGCT (Free ASGCT Whitepaper) 27th Annual Meeting taking place May 7-11, 2024, in Baltimore, MD

o
Incremental updates include longer-term data demonstrating the safety and efficacy of Rocket’s Phase 1/2 pivotal studies of KRESLADI for severe LAD-I and RP-L102 for FA, in addition to the Phase 1 study of RP-L301 for PKD.

o
Details for oral presentations are as follows:

Title: Gene Therapy for Adult and Pediatric Patients with Severe Pyruvate Kinase Deficiency: Results from a Global Study of RP-L301
Session: Clinical Trials Spotlight Symposium
Presenter: Julián Sevilla, M.D., Ph.D., Clinical Investigator, Hematología y Hemoterapia, Hematología y Oncología Pediátricas, Hospital Infantil Universitario Niño Jesús
Presentation date and time: Wednesday, May 8, 2024, 8:00 a.m. – 8:15 a.m. ET
Location: Ballroom 1
Presentation number: 4

Title: Lentiviral-Mediated Gene Therapy (RP-L102) for Fanconi Anemia [Group A] is Associated with Polyclonal Integration Patterns in the Absence of Conditioning
Session: Cell Therapy and Cell-Based Gene Therapy Trials
Presenter: Agnieszka Czechowicz, M.D., Ph.D., Center for Definitive and Curative Medicine, Department of Pediatrics, Division of Hematology/ Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University School of Medicine, Lucile Packard Children’s Hospital Stanford
Presentation date and time: Friday, May 10, 2024, 2:45 p.m. – 3:00 p.m. ET
Location: Ballroom 1
Presentation number: 245

Title: Autologous Ex-Vivo Lentiviral Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I Provides Sustained Efficacy with a Favorable Safety Profile
Session: Cell Therapy and Cell-Based Gene Therapy Trials
Presenter: Donald B. Kohn, M.D., Distinguished Professor of Microbiology, Immunology & Molecular Genetics (MIMG), Pediatrics and Molecular & Medical Pharmacology; Director of the UCLA Human Gene and Cell Therapy Program, University of California, Los Angeles
Presentation date and time: Friday, May 10, 2024, 3:00 p.m. – 3:15 p.m. ET
Location: Ballroom 1
Presentation number: 246

Upcoming Investor Conference

•
Bank of America Global Healthcare Conference 2024: May 16, 2024

First Quarter Financial Results

•
Cash position. Cash, cash equivalents and investments as of March 31, 2024, were $330.3 million.

•
R&D expenses. Research and development expenses were $45.2 million for the three months ended March 31, 2024, compared to $46.4 million for the three months ended March 31, 2023. The decrease in R&D expenses was primarily driven by decreases in manufacturing and development costs and direct materials of $5.8 million. Decreases were partially offset by increases in costs for compensation and benefits expense of $1.4 million due to increased R&D headcount, professional fees of $1.1 million, laboratory supplies of $0.9 million, non-cash stock compensation expense of $0.8 million, and clinical trial costs of $0.6 million.

•
G&A expenses. General and administrative expenses were $22.1 million for the three months ended March 31, 2024, compared to $15.8 million for the three months ended March 31, 2023. The increase in G&A expenses was primarily driven by increased commercial preparation expenses which consists of commercial strategy, medical affairs, market development and pricing analysis of $3.3 million, legal expenses of $1.5 million, and non-cash stock compensation expense of $0.5 million.

•
Net loss. Net loss was $62.1 million or $0.66 per share (basic and diluted) for the three months ended March 31, 2024, compared to $58.3 million or $0.73 (basic and diluted) for the three months ended March 31, 2023.

•
Shares outstanding. 90,646,590 shares of common stock were outstanding as of March 31, 2024.

Financial Guidance

•
Cash position. As of March 31, 2024, Rocket had cash, cash equivalents and investments of $330.3 million. Rocket expects such resources will be sufficient to fund its operations into 2026, including producing AAV cGMP batches at the Company’s Cranbury, N.J. R&D and manufacturing facility and continued development of its six clinical and/or preclinical programs.

On May 6, 2024 Ratio Therapeutics Inc. (Ratio), an emerging pharmaceutical company employing innovative technologies to develop best-in-class radiopharmaceuticals for cancer treatment and monitoring, reported the appointment of Marc Becker as the company’s Chief Financial Officer and Chief Operating Officer (Press release, Ratio Therapeutics, MAY 6, 2024, View Source [SID1234642684]). This appointment will reinforce Ratio’s financial and operational leadership by bringing significant experience in biotech and pharma to guide growth strategies and ensure continued success.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are honored to welcome Marc to the Ratio team," commented Dr. Jack Hoppin, Chairman and Chief Executive Officer of Ratio. "Building on the recent expansion of our leadership team and board, the appointment of our new CFO/COO is essential as we navigate the dynamic and rapidly evolving radiopharmaceutical space, which is currently experiencing significant growth and activity. Marc’s experience in financial management and strategic planning makes him a valuable asset as we continue to grow and develop our pipeline of radiopharmaceuticals."

"It’s a privilege to become part of Ratio’s team during this critical juncture, especially with the escalating momentum surrounding the development of targeted radiopharmaceuticals within our industry," said Marc Becker, Chief Financial Officer and Chief Operating Officer of Ratio. "I look forward to applying my financial expertise and knowledge of the biopharmaceutical sector to help shape Ratio’s strategic direction and ensure our growth is robust and sustainable."

Mr. Becker brings 20+ years of experience in biotech and pharma to his role as CFO/COO at Ratio. With a proven track record of success in strategic planning, finance transactions, and operational scaling, he will play a crucial role in driving forward the company’s vision and business objectives. Prior to his most recent position as CFO, Growth Companies, at Flagship, Mr. Becker served as CFO for Concert Pharmaceuticals, Inc., where he raised several rounds of financing and led a successful M&A process culminating in a sale to Sun Pharma in March 2023. He also previously served as CFO at CRISPR Therapeutics, where he led a successful IPO. Additionally, he held the position of CFO and Senior Vice President at rEVO Biologics and served as Vice President at Genzyme Corporation. Mr. Becker holds a BS in Accounting from the University of Massachusetts, an MBA from Babson College and was licensed as a CPA.

On May 6, 2024 Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company developing an innovative drug delivery technology designed to leverage a patient’s own biology to deliver rare disease therapeutics, reported that Dirk Thye, M.D., Quince’s Chief Executive Officer and Chief Medical Officer, will present at The Citizens JMP Life Sciences Conference on Monday, May 13, 2024 beginning at 1:30 p.m. Eastern Time (Press release, Quince Therapeutics, MAY 6, 2024, View Source [SID1234642683]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A live webcast of the presentation will be accessible on the Events page under the News & Events heading of Quince’s Investor Relations website at ir.quincetx.com. An archive of the webcast will be available shortly following the end of the live event.

On May 6, 2024 Pliant Therapeutics, Inc. (Nasdaq: PLRX), a late-stage clinical biotechnology company and leader in the discovery and development of novel therapeutics for the treatment of fibrotic diseases, reported a corporate update and announced first quarter 2024 financial results (Press release, Pliant Therapeutics, MAY 6, 2024, View Source [SID1234642682]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"In the first quarter, we made significant progress across our portfolio, highlighted by the positive safety and exploratory efficacy data from our Phase 2a INTEGRIS-PSC trial, as well as completion of a Phase 2a PET imaging trial evaluating bexotegrast’s effects on total lung collagen and lung function in IPF patients," said Bernard Coulie, M.D., Ph.D., President and Chief Executive Officer of Pliant Therapeutics. "Our strong execution has also allowed us to accelerate the ongoing BEACON-IPF Phase 2b trial through the implementation of an adaptive Phase 2b/3 design which will reduce the time to Phase 3 data by up to two years."
First Quarter and Recent Highlights
Bexotegrast Highlights
•Acceleration of bexotegrast development plan with implementation of BEACON-IPF as a pivotal, adaptive Phase 2b/3 trial in patients with idiopathic pulmonary fibrosis (IPF). The adaptive design implementation, based on acceptance by the European Union (EU) and other global health authorities, is expected to significantly shorten bexotegrast’s late-stage development compared to standalone Phase 2b and Phase 3 trials. As part of the adaptive Phase 2b/3 implementation, the Phase 2b portion of BEACON-IPF was upsized from 267 patients to 360 patients. Enrollment is progressing and we expect to complete enrollment of the upsized Phase 2b study expected in the first quarter of 2025.
•Completion of a 12 week Phase 2a PET imaging trial, measuring the effects of bexotegrast on total lung collagen and forced vital capacity (FVC) in IPF patients. This randomized, double-blind, placebo-controlled trial (NCT05621252) was conducted at Massachusetts General Hospital. The trial’s primary endpoint is the change in total lung collagen in 10 participants with IPF following once-daily treatment with bexotegrast at 160 mg for 12 weeks. Collagen will be evaluated with PET imaging, utilizing a collagen-binding probe. The secondary endpoint is safety and tolerability. Exploratory endpoints include change in FVC, cough severity and fibrosis biomarkers. Conduct of this trial has been completed, with topline data expected in the coming weeks.
•Completion of INTEGRIS-PSC Phase 2a trial in patients with primary sclerosing cholangitis (PSC) with data readout expected mid-year. As previously announced, at a once-daily dose of 320 mg, bexotegrast was well tolerated over 12 weeks of treatment with no drug-related severe or serious adverse events. At the 320 mg dose, bexotegrast reduced both Enhanced Liver Fibrosis (ELF) scores and PRO-C3 levels and showed improvements in hepatocyte function and bile flow by contrast MRI imaging relative to placebo at Week 12. The final 24-week data readout from the 320 mg dose group is expected in mid-2024.
Pipeline Programs
•Muscular dystrophy program cleared for conduct of Phase 1 study. PLN-101325 is a monoclonal antibody designed to act as an allosteric agonist of integrin α7β1. The Company recently received regulatory clearance from Australia’s Research Ethics Committee (HREC) for the conduct of a first-in-human Phase 1 clinical study of

PLN-101325 which is being developed as a potential treatment for muscular dystrophies, including Duchenne muscular dystrophy (DMD).
•Phase 1 trial of PLN-101095 in solid tumors is progressing with dosing of the third of five cohorts. PLN-101095 is an oral, small molecule, dual selective inhibitor of αvβ8 and αvβ1 integrins designed to block TGF-β activation in the tumor microenvironment. The Company is currently enrolling the third of five cohorts in the Phase 1 open label, dose-escalation trial of PLN-101095 as monotherapy and in combination with pembrolizumab in patients with solid tumors that are resistant to immune checkpoint inhibitors. Preliminary data could be available as early as late 2024.
Corporate Highlights
•Loan facility with Oxford Finance LLC (Oxford), upsized by $50 million to a total of $150 million of available non-dilutive capital. To date, the Company has drawn a total of $30 million from the facility. The remaining funds available under this facility, along with the Company’s March 31, 2024 cash and cash equivalents and short-term investments of $483.9 million are expected to fund Pliant’s anticipated operating expenses and capital expenditure requirements through 2026.
First Quarter 2024 Financial Results
•Research and development expenses were $37.1 million, as compared to $29.3 million for the prior-year quarter. The increase was due primarily to higher employee-related expenses and increased clinical and manufacturing-related costs associated with our lead program, bexotegrast, partially offset by a decrease in preclinical manufacturing costs for our pipeline product candidates.
•General and administrative expenses were $15.2 million, as compared to $14.2 million for the prior-year quarter. The increase was due to higher employee-related expenses.
•Net loss of $47.0 million as compared to $37.5 million for the prior-year quarter. The increase was due to higher operating expenses coupled with a decrease in collaboration revenues under the Novartis agreement during the quarter.
•As of March 31, 2024, the Company had cash, cash equivalents and short-term investments of $483.9 million.

On May 6, 2024 ORIC Pharmaceuticals, Inc. (Nasdaq: ORIC), a clinical stage oncology company focused on developing treatments that address mechanisms of therapeutic resistance, reported financial results and operational updates for the quarter ended March 31, 2024 (Press release, ORIC Pharmaceuticals, MAY 6, 2024, View Source [SID1234642681]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"In the first quarter of 2024, we continued making steady progress across our clinical and preclinical programs, while also strengthening our cash position and runway," said Jacob M. Chacko, M.D., president and chief executive officer. "Most recently, we announced the selection of provisional recommended phase 2 doses for ORIC-114 that confirm its wide therapeutic index, and we initiated three expansion cohorts in patients with EGFR/HER2 mutated non-small cell lung cancer, including those with active, untreated CNS metastases. For ORIC-944, we presented clinical and preclinical data that further reinforce its promise as a potential best-in-class treatment option for prostate cancer based upon its superior drug properties and clinical half-life versus competitor PRC2 inhibitors. We are laser focused on flawless execution as we continue to advance these two programs towards the initiation of registrational studies, which we anticipate in the second half of 2025."

First Quarter 2024 and Other Recent Highlights

ORIC-114: a brain penetrant, orally bioavailable, irreversible EGFR/HER2 inhibitor

Announced the completion of the dose escalation portion of the Phase 1b trial of ORIC-114 and the selection of the provisional recommended phase 2 doses.
Announced first patients dosed across three expansion cohorts in the Phase 1b trial of ORIC-114 in patients with mutated non-small cell lung cancer (NSCLC), including EGFR exon 20 insertion (EGFR exon 20 inhibitor naïve), HER2 exon 20 insertion, and EGFR atypical mutations.
Initiated an extension cohort to evaluate ORIC-114 for the treatment of patients with first-line, treatment-naïve EGFR exon 20 insertion NSCLC.
Expect to report updated Phase 1b data in the first half of 2025.
ORIC-944: a potent and selective allosteric inhibitor of PRC2

Reported initial Phase 1b monotherapy data for ORIC-944 in metastatic prostate cancer supporting advancement into combination development and demonstrating the potential as a best-in-class PRC2 inhibitor, including a clinical half-life of ~20 hours, no signs of CYP autoinduction that was observed with first-generation PRC2 inhibitors, robust target engagement, and a well-tolerated safety profile.
Presented preclinical data at the 2024 AACR (Free AACR Whitepaper) Annual Meeting demonstrating superior preclinical drug properties and synergy data in prostate cancer models, reinforcing the promise of ORIC-944 as a potential best-in-class treatment for combination with AR inhibitors.
Proceeding with combination of ORIC-944 with AR inhibitor(s) in metastatic prostate cancer and expect to provide a program update in mid-2024.
ORIC-533: a highly potent, orally bioavailable small molecule inhibitor of CD73

The company is completing a Phase 1b trial and plans to pursue strategic partnership for combination studies.
Discovery Pipeline:

Presented at the 2024 AACR (Free AACR Whitepaper) annual meeting the first preclinical data on ORIC-613, a potential first- and best-in-class development candidate selectively inhibiting PLK4.
Corporate Highlights:

Strengthened cash position and runway with a $125 million private placement financing from new and existing healthcare specialist funds in January 2024.
First Quarter 2024 Financial Results

Cash, Cash Equivalents and Investments: Cash, cash equivalents and investments totaled $331.5 million as of March 31, 2024, which the company expects will be sufficient to fund its operating plan into late 2026.

R&D Expenses: Research and development (R&D) expenses were $22.0 million for the three months ended March 31, 2024, compared to $19.5 million for the three months ended March 31, 2023, an increase of $2.4 million. The increase was due to a net increase in external expenses related to the advancement of product candidates and discovery programs, as well as higher personnel costs, including additional non-cash stock-based compensation of $0.7 million.

G&A Expenses: General and administrative (G&A) expenses were $7.0 million for the three months ended March 31, 2024, compared to $6.2 million for the three months ended March 31, 2023, an increase of $0.9 million. The increase was primarily due to higher personnel costs, including additional non-cash stock-based compensation of $0.7 million.