On March 18, 2021 UroGen Pharma Ltd. (Nasdaq: URGN), a biopharmaceutical company dedicated to building and commercializing novel solutions that treat specialty cancers and urologic diseases, reported that the Company has entered into a strategic funding agreement with RTW Investments, LP and its affiliated entities ("RTW") (Press release, UroGen Pharma, MAR 18, 2021, View Source [SID1234576881]).

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!

Under the terms of the agreement, subject to customary closing conditions, UroGen will receive $75 million in upfront cash from RTW in return for tiered, future cash payments based on aggregate worldwide annual net product sales of Jelmyto (mitomycin) for pyelocalyceal solution as well as UGN-102, if approved. In exchange for the $75 million upfront payment, RTW will receive tiered, future cash payments on worldwide annual net product sales of Jelmyto ranging from high to low single digits based on certain annual sales thresholds, subject to upward adjustment if certain annual sales and regulatory milestones are not met. RTW will also receive tiered, future cash payments on worldwide annual net product sales of UGN-102, if approved, in the low single digits based on certain annual sales thresholds.

The future payments on both Jelmyto and UGN-102 will terminate following the date that RTW has received an aggregate amount pursuant to such payments of $300 million.

"UroGen’s RTGel platform has demonstrated the ability to overcome historical treatment barriers, work with anatomical complexity and thus unlock new therapeutic potential, including those conditions targeted by Jelmyto and UGN-102," said Brad Sitko, Managing Director of Strategic Finance, RTW Investments, LP. "We are proud to partner with the UroGen management team and look forward to supporting the Company’s goal of building a leading uro-oncology company."

"This important non-dilutive transaction provides a clear path to fund the ongoing launch of Jelmyto and to advance our UGN-102 program, including the ongoing Phase 3 ATLAS study," said Molly Henderson, Chief Financial Officer of UroGen Pharma. "We are proud to partner with RTW, a leading, research-driven healthcare investor, allowing us to access capital that is aligned with our vision and invest further in our mission of developing treatments for patients with urologic and specialty cancers."

Cowen acted as financial advisor to UroGen on the transaction. Cooley acted as legal advisor to UroGen. Gibson Dunn acted as legal advisor to RTW.

On March 18, 2021 Boston Pharmaceuticals reported a pioneering three-year out-license and option agreement with GlaxoSmithKline PLC (LSE/NYSE: GSK). Boston Pharmaceuticals will become a preferred GSK partner for select pre-phase 2 programs (Press release, Boston Pharmaceuticals, MAR 18, 2021, View Source [SID1234576880]). This new agreement builds on the relationship established in 2018 with Boston Pharmaceuticals’ acquisition of five GSK programs.

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!

"This new agreement validates Boston Pharmaceuticals as a trusted development partner with whom pharmaceutical companies can collaborate," said Robert Armstrong, Ph.D., CEO of Boston Pharmaceuticals. "GSK has been an excellent partner and we look forward to advancing these pre-phase 2 assets into and through the clinic to evaluate their potential to improve patient lives."

Initially, GSK will out-license and option two programs to Boston Pharmaceuticals:

GSK3903371 – a monoclonal antibody targeting the Interleukin-1 Receptor Accessory Protein (IL1RAP), a tumor-associated antigen driving tumor growth and immunosuppression.
GSK3502421 – an orally available, small molecule inhibitor for potential neurological disorders that targets Receptor Interacting Serine/Threonine Kinase 1 (RIPK1), a key component of the TNF-driven inflammation and necroptosis pathway.
"We are pleased to further strengthen our relationship with Boston Pharmaceuticals as they continue to help us translate great science into medicines," said John Lepore, SVP, Research of GSK. "This agreement makes strong strategic sense as it helps us assess the potential of multiple early-stage programs and focus on progressing our own internal assets, while maintaining pipeline optionality for the future."

Under the agreement, Boston Pharmaceuticals will be responsible for further development of select programs through proof-of-concept (PoC). Following the completion of PoC studies, GSK will have the option to reacquire each program under pre-agreed terms for subsequent development and worldwide commercialization.

If GSK exercises its repurchase option, Boston Pharmaceuticals will receive a one-time payment, as well as be eligible for approval and sales milestones and royalties. In the event GSK chooses not to reacquire a program, Boston Pharmaceuticals may continue development and potential commercialization of the program. GSK will then be eligible to receive milestones and royalty payments.

On March 18, 2021 Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications, reported its unaudited financial results for the three months and year ended December 31, 2020 (Press release, Legend Biotech, MAR 18, 2021, View Source [SID1234576879]).

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!

"This was a defining year for Legend Biotech, as we achieved important pipeline advancements and completed our initial public offering and Nasdaq listing," said Ying Huang, PhD, CEO and CFO of Legend Biotech. "Despite the challenges presented by the COVID-19 pandemic, we have achieved a strong 2020 and are continuing this momentum with significant advancements planned for our oncology pipeline during 2021. Through our collaboration with Janssen Biotech, Inc. (Janssen)*, in 2020, we initiated a global Phase 3 study and expanded the multi-cohort Phase 2 study as part of a comprehensive clinical development program for ciltacabtagene autoleucel (cilta-cel), including as an earlier lines of multiple myeloma treatment. In 2021, we expect to achieve important milestones in advancing the regulatory approval process for cilta-cel."

*In December 2017, Legend Biotech entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. to develop and commercialize cilta-cel.

Fourth Quarter 2020 & Recent Highlights

In February 2021, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) accepted a request from Janssen for the accelerated assessment of the cilta-cel Marketing Authorisation Application (MAA) for the treatment of adults with RRMM.
In December 2020, Legend Biotech announced that Janssen had initiated a rolling submission to the U.S. Food and Drug Administration (FDA) of a biologics license application (BLA) for cilta-cel for the treatment of adults with RRMM. Pursuant to the terms of Legend Biotech’s agreement with Janssen, Legend received a $75.0 million milestone payment relating to the clinical development of cilta-cel in connection with the initiation of the BLA submission.
In December 2020, the FDA cleared an investigational new drug (IND) application for Legend Biotech to evaluate LB1901 in a Phase 1 clinical study for the treatment of adults with RR TCL.
Data from the combined Phase 1b/2 CARTITUDE-1 study of cilta-cel was presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual meeting in December 2020, showing a high overall response rate that deepened over time, with 97% of patients achieving an overall response and 67% of patients achieving a stringent complete response (sCR) at a median follow-up of 12.4 months.
Recent Appointments

During 2020, Legend Biotech bolstered its leadership team with the appointment of Dr. Ying Huang as Chief Executive Officer, the appointment of Ye (Sally) Wang as Chairwoman of the Board of Directors, and the appointment of two new directors, Dr. Li Zhu and Dr. Patrick Casey.
In March 2021, Lori Macomber was appointed as Legend Biotech’s Vice President, Finance, in which capacity Ms. Macomber will serve as Legend Biotech’s principal financial officer and principal accounting officer. Ms. Macomber has served as Legend Biotech’s Vice President of Supply Chain Finance and Controller since September 2019. Prior to joining Legend Biotech, Ms. Macomber served as Business Unit Controller at Ametek PDS, a leading supplier of components and systems for the aerospace and defense industries, from April 2018 and as U.S. CFO and Controller of Cello Health from March 2017 until February 2018. Before this Ms. Macomber held various positions, most recently AVP Finance Site Leader, at Eli Lilly & Company where she was employed from May 2010 until March 2017. Ms. Macomber holds a Bachelor of Science in Accounting from Pennsylvania State University and is a Certified Public Accountant.
In January 2021, Lida Pacaud, M.D., joined Legend Biotech as its Vice President of Clinical Development. Dr. Pacaud, joined Legend Biotech from Novartis International AG, where she held various positions from September 2013 through January 2021, most recently serving as the Global Clinical Program Head and Executive Medical Director in its Cell & Gene unit. Prior to this, Dr. Pacaud worked at Roche and Wyeth. Dr. Pacaud has been the Medical Lead on several global Phase I, II & III trials and led the clinical development and filling for the first approved CAR T therapy worldwide. Dr. Pacaud holds a Doctor of Medicine degree and certification in Pediatrics from Tbilisi State Medical University and has trained in Pediatric Oncology and hematology in France.
Key Upcoming Milestones

Legend Biotech’s collaborator, Janssen, anticipates submitting a MAA for cilta-cel for the treatment of adults with RRMM to the EMA in the first half of 2021.
Legend Biotech intends to use the data from the CARTIFAN-1 study in support of a regulatory submission to the China Center for Drug Evaluation (CDE) seeking approval of cilta-cel for the treatment of adults with RRMM. Legend Biotech expects the submission of the application to occur in the second half of 2021.
Legend Biotech’s collaborator, Janssen, anticipates submitting a New Drug Application (NDA) to the Japan Ministry of Health, Labor and Welfare (JMHLW) in the second half of 2021 seeking approval of cilta-cel for the treatment of adults with RRMM.
Legend Biotech expects to initiate its Phase 1 clinical trial of LB1901 in RR TCL in the United States in 2021.
Legend Biotech, in collaboration with Janssen, intends to present updated data from the CARTITUDE-1 and data from CARTITUDE-2 studies at major medical conferences in 2021.
Legend Biotech anticipates supporting investigators with publishing a clinical data update from LEGEND-2 study in 2021.
As the global COVID-19 pandemic continues to evolve, Legend Biotech has continuously monitored the situation in regards to its operations and has put significant measures in place to protect supply chain, operations, employees and the execution of clinical trials. Given the dynamic global situation, Legend Biotech notes that certain clinical trial timelines may be impacted.

Financial Results for the Quarter and Year Ended December 31, 2020

Cash and Cash Equivalents and time deposits:

As of December 31, 2020, Legend Biotech had approximately $455.7 million of cash and cash equivalents and approximately $50.0 million in time deposits.

Revenue

Revenue for the three months ended December 31, 2020 was $40.8 million compared to $19.5 million for the three months ended December 31, 2019. The increase of $21.3 million was primarily due to revenue recognition of additional milestone payment achieved of higher amount pursuant to Legend Biotech’s agreement with Janssen and the associated constrained variable consideration is relieved. Revenue for the year ended December 31, 2020 was $75.7 million compared to $57.3 million for the year ended December 31, 2019. Similarly, the increase of $18.4 million for the year ended December 31, 2020 was primarily driven by revenue recognized from an additional milestone achieved of higher amount. Legend Biotech has not generated any revenue from product sales to date.

Research and Development Expenses

Research and development expenses for the three months ended December 31, 2020 were $66.9 million compared to $66.1 million for the three months ended December 31, 2019. This increase of $0.8 million was primarily due to an increase in employee benefit expense and research and development expense, net-off by a decrease in collaborative research and development expenses. Research and development expenses for the year ended December 31, 2020 was $232.2 million compared to $161.9 million for the year ended December 31, 2019 with a $70.3 million increase. The year-over-year increase was primarily due to a higher number of clinical trials, a higher number of patients enrolled in those trials and a higher number of research and development product candidates in the year ended December 31, 2020.

Administrative Expenses

Administrative expenses for the three months ended December 31, 2020 were $9.2 million compared to $2.0 million for the three months ended December 31, 2019. The increase of $7.2 million was primarily due to Legend Biotech’s expansion of supporting administrative functions to aid continued research and development activities. Due to the consistent business expansion, administrative expenses for the year ended December 31, 2020 increased by $16.3 million, which was $23.1 million compared to $6.8 million for the year ended December 31, 2019.

Selling and Distribution Expenses

Selling and distribution expenses for the three months ended December 31, 2020 were $24.2 million compared to $13.4 million for the three months ended December 31, 2019. This increase of $10.8 million was primarily due to increased costs associated with commercial preparation activities for cilta-cel. Driven by the same commercial preparation activities, selling and distribution expenses for the year ended December 31, 2020 was $49.6 million compared to $25.6 million for the year ended December 31, 2019.

Other Income and Gains

Other income and gains for the three months ended December 31, 2020 was $2.1 million compared to $0.5 million for the three months ended December 31, 2019. The increase was primarily driven by increase in foreign exchange gain. Other income and gains for the year ended December 31, 2020 was $6.1 million compared to $7.1 million for the year ended December 31, 2019. The decrease of $1.0 million was primarily driven by reduced average interest rate for holding of time deposits that generate interest income.

Finance Costs

Finance costs the year ended December 31, 2020 were $4.2 million compared to $0.2 million for the year ended December 31, 2019. The increase was primarily due to finance costs related to the issuance of convertible redeemable preferred shares, which have been fully converted into ordinary shares upon the completion of Legend Biotech’s initial public offering in June 2020.

Loss for the Period

For the three months ended December 31, 2020, net loss was $57.8 million, or $0.22 per share, compared to a net loss of $63.9 million, or $0.32 per share, for the three months ended December 31, 2019. Net loss was $303.5 million, or $1.28 per share, for the year ended December 31, 2020 compared to $133.0 million, or $0.66 per share, for the year ended December 31, 2019. (Press release, Legend Biotech, MAR 18, 2021, View Source [SID1234576879])

On March 18, 2021 SQZ Biotechnologies (NYSE: SQZ), a cell therapy company developing novel treatments for multiple therapeutic areas, reported full year 2020 financial results and recent business highlights (Press release, SQZ Biotech, MAR 18, 2021, View Source [SID1234576878]).

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 2020, we experienced the most important moment in SQZ’s journey so far: dosing our first patient. This accomplishment was coupled with exciting progress across our pipeline and the transition to being a public company. Despite the many challenges of COVID-19, our team continued to maintain focus on our mission to create transformative cell therapies for patients, for which I am proud and thankful," commented Armon Sharei, chief executive officer. "We have many exciting milestones ahead and are committed to delivering on SQZ’s potential for patients across diseases."

2020 and Recent Pipeline Developments

SQZ Antigen Presenting Cell ("APC") Platform

Oncology

Announced initial safety data from the first 12 patients in SQZ-PBMC-HPV-101, the first clinical trial of the SQZ APC platform, demonstrated no dose limiting toxicities or grade 3 or higher treatment-related adverse events through year end 2020
Presenting biomarker data from SQZ-PBMC-HPV-101 monotherapy cohorts in mid-2021
Infectious Diseases

Generated preclinical data across multiple infectious disease antigens showing specific and robust CD8 T cell responses and selected HBV as initial disease target
Next-Gen SQZ Enhanced Antigen Presenting Cell ("eAPC") Platform

Advanced eAPC platform leveraging multiplexed delivery of mRNA cargos to incorporate additional functionality and potentially broaden addressable patient population
Presenting preclinical data at AACR (Free AACR Whitepaper) 2021 and anticipate filing an investigational new drug application ("IND") in 2021
SQZ Activating Antigen Carriers ("AAC") Platform in Oncology

Announced SQZ AAC IND in HPV+ tumors was cleared by the FDA in January 2021 for a Phase 1 study to evaluate SQZ-AAC-HPV as a monotherapy and in combination with immune-oncology agents
Continuing preclinical work for SQZ-AAC-KRAS for potential application across KRAS tumors
SQZ Tolerizing Antigen Carriers ("TAC") Platform in Immune Tolerance

Presented preclinical data at ASIT showing how SQZ TACs drive multiple antigen specific tolerance mechanisms, including deletion, anergy, and Treg expansion
Manufacturing and Patient Experience

Produced doses for each patient in SQZ-PBMC-HPV-101 trial in under 24 hours
Developing a point-of-care manufacturing prototype that could potentially enable further efficiencies in our process and improve patient accessibility to novel cell therapies
2020 and Recent Corporate Highlights

Raised over $200 million in gross proceeds from equity financings, including a private round closed in the first half of 2020, an initial public offering in October 2020, and a follow-on public offering in February 2021
The Company believes that the cash, cash equivalents and marketable securities as of December 31, 2020 along with the subsequent follow-on offering in February 2021 will be sufficient to fund operating expenses and capital expenditure requirements through the first half of 2023
Added key board members and advisors, including Sapna Srivastava, PhD, Paul Bolno, MD, and Marc Schegerin, MD to the Board of Directors and Kai Wucherpfenning, MD, PhD to our Scientific Advisory Board
Added key members of senior management, including Micah Zajic as chief business officer and David First as chief people officer
2020 Full Year Financial Highlights

Closed 2020 with $170.4 million in cash, cash equivalents and marketable securities compared to $98.3 million as of December 31, 2019
Revenue was $21.0 million for the year ended December 31, 2020, compared to $20.1 million for the year ended December 31, 2019
Research and development expenses were $51.5 million for the year ended December 31, 2020, as compared to $36.1 million for the year ended December 31, 2019. The increase was primarily attributable to higher development and manufacturing costs associated with translating our lead product candidate, SQZ-PBMC-HPV, into the clinic, as well as increased personnel-related costs to support continued progress with the company’s pipeline
General and administrative ("G&A") expenses were $20.5 million for the year ended December 31, 2020, compared to $18.3 million for the year ended December 31, 2019. The increase in G&A expenses was primarily due to an increase in personnel and other corporate-related costs, including costs to operate as a public company
Net loss for the year ended December 31, 2020 was $50.6 million compared to $32.2 million for the year ended December 31, 2019

On March 18, 2021 The Italfarmaco Group reported a publication in the Blood Cancer Journal outlining the first interim analysis results from follow-up of a mean of four years in 51 patients with polycythemia vera (PV), a type of hematological malignancy belonging to BCR-ABL1-negative chronic myeloproliferative neoplasms (MPNs) (Press release, Italfarmaco, MAR 18, 2021, View Source [SID1234576877]). The results show a long-term benefit in PV patients treated with givinostat, the company’s proprietary histone deacetylase (HDAC) inhibitor. The publication covers the overall long-term data from patients that were a part of three Phase 1/2 studies, as well as from a compassionate use program. The data preliminarily support the long-term use of givinostat. The open access article is available via this link.

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!

"These long-term results support the potential of givinostat as a disease-altering treatment in patients with PV who are currently confined to symptomatic care," said Alessandro Rambaldi, M.D., Professor of Hematology at the University of Milan, Head, Hematology and Bone Marrow Transplant Unit at ASST Papa Giovanni XXIII in Bergamo, lead author and investigator of the trial. "The overall response rate, which continues to be persistent over the four-year mean follow-up, combined with the good safety profile, justifies the global pivotal Phase 3 study, which will start this year, to further evaluate givinostat and generate clinical evidence necessary to bring the drug to patients."

"With over 100,000 polycythemia vera cases in the U.S. and considering that these patients have an increased risk of developing myelofibrosis or acute myeloid leukemia, in addition to the day-to-day complications caused by the disease, the data announced today provide PV patients with hope for a new treatment option," added Srdan Verstovsek, M.D., Ph.D., Professor of Medicine, Chief, Section for MPNs at the Department of Leukemia and Director at the Clinical Research Center for MPNs at MD Anderson Cancer Center in Houston, Texas. "I value the opportunity to participate in the upcoming pivotal and global Phase 3 trial, which will be the first of its kind in PV."

The results published in Blood Cancer Journal summarize the four-year mean follow-up of an open-label, long-term study (Clinicaltrials.gov: NCT01761968) that enrolled 51 patients with PV, who received clinical benefit from givinostat in the company’s previous three Phase 1/2 clinical trials and/or on compassionate use. These patients continued to receive givinostat at the last effective and tolerated dose. The follow-up analyses showed that givinostat had a good overall safety and tolerability profile. During the study, only five patients (10.0%) experienced Grade 3 Adverse Events (AE) and no treatment-related Grade 4 or Grade 5 AEs occurred, supporting the strong safety profile reported in previous analyses. Over 80% of the patients responded to treatment according to the ELN Response Criteria, i.e., the internationally recognized response criteria that evaluates the effect of treatment on haematology, spleen volume and symptoms in PV (Barosi G et al., Blood 2009). Such a response was maintained for the duration of the follow-up period.

A reduction in the mean Janus Kinase 2 (JAK2) allele burden, a gene believed to be key in the pathogenesis of PV, was also observed regularly at annual patient visits. Importantly, the majority of the PV patients continued treatment with givinostat after the conclusion of the study period as they were receiving relevant clinical benefit, supporting its long-term use.

Italfarmaco expects to initiate the global Phase 3 trial with givinostat in PV patients by mid-2021 with more than 100 participating sites in Europe, US, Canada and Asia.

About Polycythemia Vera
Polycythemia Vera is a rare blood disease characterized by an overproduction of red blood cells, white blood cells and platelets, which thickens the blood and increases the risk of blood clots, a major underlying cause of life-threatening conditions such as thrombosis, embolisms, heart attacks or strokes. In up to 95% of patients, the disease is associated with a mutation in the Janus Kinase 2 (JAK2) gene and disease-related symptoms include headaches, itching and microvascular symptoms. The current standard of care ranges from phlebotomy alone or in combination with low-dose aspirin, to drugs such as the cytoreductive hydroxycarbamide, interferon (ropeginterferon) or the JAK inhibitor (ruxolitinib). These reduce symptoms, but no treatments targeting the underlying disease mechanism are available.

About Givinostat
Givinostat is an investigational drug discovered through Italfarmaco’s internal research. It is being evaluated for safety and efficacy for the treatment of Duchenne- and Becker- Muscular Dystrophy and Polycythemia Vera. Givinostat inhibits histone deacetylases (HDACs), which are enzymes that prevent gene translation by changing the three-dimensional folding of DNA in the cell. Givinostat selectively targets the specific JAK2 mutation responsible for MPNs, thereby inhibiting cell growth and proliferation of hematopoietic cells.