On March 18, 2021 Shanghai Fosun Pharmaceutical (Group) Co., Ltd. ("Fosun Pharma"; stock code: 600196.SH; 02196.HK) reported that its subsidiary YaoPharma Co., Ltd. ("YaoPharma") had recently received the approval from the NMPA regarding the clinical trial for the YP01001 capsules (the "Investigational New Drug") for the treatment of advanced solid tumors (Press release, Fosun Pharma, MAR 18, 2021, View Source [SID1234626451]).

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The Investigational New Drug is an innovative small molecule chemical drug intended for the treatment of advanced solid tumors (including, among others, liver cancer and gastric cancer). YaoPharma plans to conduct Phase I clinical trial for the Investigational New Drug in China (excluding Hong Kong, Macao and Taiwan) recently after the conditions are available.

As of February 2021, Fosun Pharma has invested about RMB 33.66 million (unaudited) in the R&D of the Investigational New Drug at this stage.

Driven by innovation and R&D, Fosun Pharma continuously improves its drug R&D system based on the combination of generic and innovator drugs and builds international R&D platforms for small molecule innovator drugs, high-cost generic drugs, biologics and new technology-based therapies, etc.

In the future, the Company will continue to focus on clinical demands, increase its investment in R&D and improve innovation efficiency, so as to bring high-quality and affordable innovator drugs to patients.

On March 18, 2021 Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer and autoimmune/inflammatory diseases, reported financial results for the fourth quarter and year ended December 31, 2020 (Press release, Alpine Immune Sciences, MAR 18, 2021, View Source [SID1234576861]).

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"This past year has been transformative for the company. We are building on our strong momentum to accelerate development of innovative therapies for patients living with cancer and autoimmune/inflammatory disease," said Mitchell H. Gold, M.D., Executive Chairman and Chief Executive Officer of Alpine. "Over the next year we will continue to execute on our clinical-stage programs and plan to initiate an international Phase 2 trial of ALPN-101 in adults with systemic lupus erythematosus (SLE), and further progress our ongoing NEON-1 Phase 1 study of ALPN-202 in advanced malignancies. We also plan to progress key pipeline programs, including the initiation of a Phase 1 healthy volunteer study for ALPN-303. In summary, I believe 2021 will be another year of significant opportunity for Alpine as we advance our pipeline of innovative therapeutic candidates."

Full Year 2020 and Recent Corporate and Clinical Updates

ALPN-101: Dual CD28/ICOS inhibitor
Announced Option and License Agreement with AbbVie: In June 2020, Alpine announced an exclusive worldwide option and license agreement for ALPN-101. Under the terms of the agreement, Alpine received an upfront payment of $60 million and is eligible to receive up to an aggregate of $805 million for exercise of the option and success-based development, regulatory, and commercial milestones, including up to $75 million in pre-option development milestones. In addition, Alpine is eligible to receive tiered royalties on net sales of ALPN-101.
Preparation for a global Phase 2 study in SLE is ongoing with an intent to initiate the study in mid-2021.
ALPN-202: Conditional CD28 costimulator and dual checkpoint inhibitor
Presentation of NEON-1 trial design at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting 2020: The design of NEON-1, our first-in-class conditional CD28 costimulator and dual checkpoint inhibitor, was presented at the AACR (Free AACR Whitepaper) Virtual Annual Meeting I, in the Phase I Trials in Progress Virtual Poster Session.
Enrollment in NEON-1, a first in human monotherapy Phase 1 clinical trial, continues to progress through its dose escalation cohorts. Alpine plans to share interim data at an appropriate scientific forum and to determine expansion cohorts later this year.
Alpine is also targeting the initiation of NEON-2, a Phase 1 combination study of ALPN-202 and a PD-1 inhibitor, later this year.
ALPN-303: Dual APRIL/BAFF inhibitor
Presentation of Preclinical Data on Novel B cell Modulatory Domains, Alone or in Combination with Novel Domains that Inhibit T Cell Costimulation, for Autoimmune Diseases: In June 2020, Alpine presented a poster at the European Congress of Rheumatology (EULAR) E-Congress detailing preclinical work with novel B cell modulatory variant TNF Receptor domains (vTDs), alone or in combination with variant Ig Domains (vIgDtm) that inhibit T cell costimulation. This work included ALPN-303, a novel dual BAFF/APRIL inhibitor, in multiple preclinical models, including the NZB/NZW F1 murine lupus model.
Alpine is targeting completion of activities to support initiation of a Phase 1 healthy volunteer study with ALPN-303 in the fourth quarter of this year.
General Corporate
Raised $60 Million in a Private Placement: In July 2020, Alpine raised $60 million in gross proceeds through a private placement led by Omega Funds with participation from Avidity Partners, EcoR1 Capital, LLC, Invus Public Equities, L.P., and Samsara BioCapital, among others.
Appointed industry veteran Natasha Hernday to the Board of Directors: Ms. Hernday brings more than 20 years of experience in corporate development and corporate strategy, and currently serves as Executive Vice President, Corporate Development and as a member of the Executive Committee for Seagen, Inc.
Promotion of Remy Durand, Ph.D. to Chief Business Officer: Dr. Durand joined Alpine Immune Sciences in 2018 as Vice President, Business Development, and has played a leading role in building the company’s partnerships with AbbVie and Adaptimmune, and has represented the company at investor meetings and conferences.
Appointed Pamela Holland, Ph.D. as Senior Vice President, Research: Dr. Holland is an experienced cancer biologist with a proven track record of successfully discovering and progressing multiple preclinical therapeutics into clinical development, most recently at Surface Oncology and Amgen.
Full Year 2020 Financial Results

As of December 31, 2020, we had cash, cash equivalents, and investments totaling $131.4 million. Net cash provided by operating activities for the year ended December 31, 2020 was $30.1 million compared to net cash used in operating activities of $35.3 million for the year ended December 31, 2019. We recorded net losses of $27.9 million and $41.9 million for the years ended December 31, 2020 and 2019, respectively.

Collaboration revenue for the year ended December 31, 2020 was $9.3 million compared to $1.7 million for the year ended December 31, 2019. The increase was primarily attributable to the revenue recognized under our AbbVie Agreement.

Research and development expenses for the year ended December 31, 2020 were $27.2 million compared to $35.8 million for the year ended December 31, 2019. The decrease was primarily attributable to decreases in contract manufacturing and process development of our product candidates and other direct research activities. The decreases were partially offset by increases in clinical trial activity for NEON-1.

General and administrative expenses for the year ended December 31, 2020 were $10.9 million compared to $9.5 million for the year ended December 31, 2019. The increase was primarily attributable to increases in professional and legal services, stock-based compensation and insurance and facility costs to support the growth and expansion of our business.

Alpine expects that its current cash resources, combined with the potential $75 million in pre-option exercise milestones payable under its option and license agreement with AbbVie, for the development and commercialization of ALPN-101, are sufficient to fund Alpine’s planned operations through 2023.

Fourth Quarter and Full Year 2020 Conference Call and Webcast Details

Alpine will host a conference call and live webcast to discuss the fourth quarter and full year performance today, March 18, 2021 at 4:30 p.m. ET/1:30 p.m. PT.

To access the live call by phone, dial (800) 816-3005 (domestic) or (857) 770-0069 (international) and reference conference ID: 2539338. A live webcast of the presentation will be available online in the investor relations section of the company’s website at View Source A replay of the presentation will be available on the company website for 90 days following the webcast.

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]).

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"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 AstraZeneca reported that The UK National Institute for Health and Care Excellence (NICE) has recommended Calquence for use on the NHS to treat a common form of leukaemia (Press release, AstraZeneca, MAR 18, 2021, View Source [SID1234576924]).

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NICE has recommended the twice-daily Calquence (Acalabrutinib) for the treatment of certain adult patients with chronic lymphocytic leukaemia (CLL).

It has been approved as monotherapy for CLL if there is a 17p deletion or TP53 mutation or if fludarabine plus cyclophosphamide and rituximab (FCR), or bendamustine plus rituximab (BR) is unsuitable.

The approval of Calquence will offer a new chemotherapy-free treatment option for these patients, which could be particularly useful during the COVID-19 pandemic as patients would less require hospital visits.

Patients experts involved in NICE’s consultation for the drug also highlighted that Calquence is well tolerated and causes fewer side effects compare to current treatments for CLL.

For a 30-day pack of Calquence 100mg tablets, the list price is £5,059 – although AZ has made the drug available to the NHS with a discount.

This appraisal only considered Calquence as monotherapy, as the company did not submit any data for the combination of this drug with another cancer treatment commonly used to treat CLL – Roche’s Gazyvaro (Obinutuzumab).

On March 18, 2021 Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, reported financial results for the fourth quarter and full year ended December 31, 2020 (Press release, Applied Therapeutics, MAR 18, 2021, View Source [SID1234576862]).

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"The fourth quarter was a productive period of internal planning and execution," said Shoshana Shendelman, PhD, Founder, CEO and Chair of the Board of Applied Therapeutics. "We are excited for what lies ahead in 2021, where our focus will be on advancing our late stage assets toward commercialization while initiating clinical development of our two new rare disease programs."

Recent Highlights

Announced Restart of Pediatric Galactosemia Study. In February 2021, the Company announced that the FDA lifted the clinical hold on the AT-007 ACTION-Galactosemia Kids pediatric clinical study, and the study resumed immediately. Applied Therapeutics worked closely with FDA to modify the trial, with the shared goal of ensuring that all patients have the opportunity to receive clinical benefit, and remains on target to submit an NDA no later than Q3 2021.

Announced the Launch of a Galactosemia Awareness and Education Initiative. Galactosemia Together is the first and only industry-led Galactosemia awareness and education campaign. Developed in partnership with the Galactosemia community, this initiative aims to address gaps in education by providing updated, reliable and credible resources to help connect, educate and support those families impacted by this disease.

Announced Magnetic Resonance Spectroscopy (MRS) Data from ACTION-Galactosemia Study. In December 2020, the Company shared MRS data on reduction of galactitol levels in the brain of Galactosemia patients treated with AT-007 in the ACTION-Galactosemia adult study. Overall, plasma reduction in galactitol correlated with brain reduction in galactitol. At the two doses which demonstrated statistically significant reduction in plasma galactitol, 20 and 40mg/kg, 3 out of 4 patients displayed substantial galactitol reduction ranging from 61.94% to 69.80% reduction from baseline.

Closed Public Offering of 3,000,000 Shares of Common Stock at a Price to the Public of $23.00 Per Share. In February 2021, the Company completed an underwritten public offering of 3,450,000 shares of common stock, including the exercise in full of the underwriters’ option to purchase 450,000 additional shares of common stock at a price of $23.00 per share, resulting in aggregate net proceeds of approximately $74.3 million.
Financial Results

Cash and cash equivalents and short-term investments totaled $96.8 million as of December 31, 2020, compared with $38.9 million at December 31, 2019. This does not include approximately $74.3 million in aggregate net proceeds the Company received from the underwritten public offering of common stock in February 2021.

Research and development expenses for the year ended December 31, 2020 were $61.8 million, compared to $32.4 million for the year ended December 31, 2019. The increase of $29.4 million related to an increase in clinical and pre-clinical expense of $11.3 million, primarily related to the progression of the AT-007 ACTION-Galactosemia adult extension and the AT-001 Phase 3 ARISE-HF clinical studies, as well as the commencement and progression of the AT-007 ACTION-Galactosemia Kids pediatric registrational study; an increase in drug manufacturing and formulation expenses of $15.4 million primarily related to the commencement of the 2020 manufacturing campaigns and the associated raw material deliveries and AT-007 and AT-001 drug product batch releases; an increase in personnel expenses of $0.6 million due to the increase in headcount in support of our clinical program pipeline; a decrease in stock-based compensation of $0.2 million due to the stock option modification expense recognized during the year ended December 31, 2019 for the acceleration of certain options vesting following the IPO; offset by an increase in expense recognized during the year ended December 31, 2020 due to new stock option and restricted stock grants; and an increase of regulatory and other expenses of $2.3 million primarily related to University of Miami license fees and increased clinical consulting fees recognized during the year ended December 31, 2020.

General and administrative expenses were $32.7 million for the year ended December 31, 2020, compared to $13.2 million for the year ended December 31, 2019. The increase of $19.4 million was an increase in professional and legal fees of $4.1 million due to increased operations and costs associated with being a public company for a full year; an increase of $5.7 million related to the establishment of a commercial department; an increase in personnel expenses of $4.2 million and an increase in stock-based compensation of $2.0 million due to an increase in headcount; an increase of insurance expenses of $1.8 million related to increased D&O insurance costs; and an increase in other expenses of $1.6 million, primarily relating to increased costs of rent and other office expenses.

Net loss for the year ended December 31, 2020 was $94.0 million, or $4.28 per basic and diluted common share, compared to a net loss of $45.5 million, or $3.55 per basic and diluted common share, for the year ended December 31, 2019.