LIDDS Interim report January – December 2020

On February 25, 2021 LIDDS reported that Interim report January – December 2020 (Press release, Lidds, FEB 25, 2021, View Source [SID1234575741])

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JANUARY – DECEMBER 2020

Net sales amounted to MSEK 0.3 (0.0)
Operating expenses amounted to MSEK -33.0 (-31.4)
Profit/loss before and after tax amounted to MSEK -32.3 (-31.4)
Earnings per share amounted to SEK -1.20 (-1.34)
Cash flow from operating activities amounted to MSEK -27.4 (-31.2)
Equity amounted to MSEK 42.8 (15.5) and the debt/equity ratio was 79% (72%)
OCTOBER – DECEMBER 2020

Net sales amounted to MSEK 0.3 (0.0)
Operating expenses amounted to MSEK -12.9 (-8.5)
Profit/loss before and after tax amounted to MSEK -12.5 (-8.5)
Earnings per share amounted to SEK -0.42 (-0.36)
Cash flow from operating activities amounted to MSEK -10.1 (-9.8)
Equity amounted to MSEK 42.8 (15.5) and the debt/equity ratio was 79% (72%)
SIGNIFICANT EVENTS DURING THE FORTH QUARTER 2020

Intratumoral injection of NZ-TLR9, NanoZolid formulation of a Toll-Like Receptor 9 (TLR9), results in strong antitumoral efficacy combined with prominent antitumoral immune responses in mouse tumor models. NZ-TLR9 forms an intratumoral depot which releases the TLR9 agonist for least 6 weeks and thus minimizes the need for repeated injections.
The warrants programme was finalized in October 2020 and 0.8 percent of the warrants issued was exercised.
A newly revised guideline from the Chinese National Medical Products Administration (NMPA) has extended the requirements to a full registration dossier for the conditional market approval (CMA). LIDDS licensee Jiangxi Puheng Pharma is therefore aiming to submit the application for CMA for the prostate cancer drug candidate Liproca Depot in China during Q1, 2021.
A review article in OncoTargets and Therapy journal defines LIDDS as a key player in TLR9 agonist research field and being the only provider having a sustained release product in development.
LIDDS announced that the company will apply for a relisting of its shares from Nasdaq First North to Nasdaq Stockholm Main Market in 2021.
Nina Herne will assume the role of CEO for LIDDS as from April 19, 2021. Monica Wallter will continue in LIDDS as Senior Adviser.
SIGNIFICANT EVENTS FOLLOWING THE END OF THE PERIOD

The Phase I study where NanoZolid is combined with docetaxel continues with dose escalation. New patients are in recruitment to receive injection in solid tumors with new dose of docetaxel.
European Urology Focus has accepted a scientific article describing LIDDS Phase IIb study results with Liproca Depot.
LIDDS has filed a patent application for local intracranial treatment of brain tumors. The NanoZolid technology enables controlled and sustained release of oncology drugs. Preclinical study has showed that NanoZolid injected as a depot in brain is tolerable and safe.
LIDDS has signed a manufacturing agreement for NZ-TLR9 with Pharmidea in Latvia.

The interim report is available on the company’s website, go to View Source

argenx to Report Full Year 2020 Financial Results and Fourth Quarter Business Update on March 4, 2021

On February 25, 2021 argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases and cancer, reported that it will host a conference call and audio webcast on March 4, 2021 at 2:30 p.m. CET (8:30 a.m. ET) to discuss its full year 2020 financial results and provide a fourth quarter business update (Press release, argenx, FEB 25, 2021, View Source [SID1234575740]).

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A webcast of the live call may be accessed on the Investors section of the argenx website at argenx.com/investors. A replay of the webcast will be available on the argenx website for one year following the call.

Aurinia Reports Fourth Quarter and Full Year 2020 Financial Results and Recent Operational Highlights

On February 25, 2021 Aurinia Pharmaceuticals Inc. (NASDAQ: AUPH / TSX: AUP) ("Aurinia" or the "Company") reported its financial results for the fourth quarter and year ended December 31, 2020 (Press release, Aurinia Pharmaceuticals, FEB 25, 2021, View Source [SID1234575738]). Amounts, unless specified otherwise, are expressed in U.S. dollars.

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"Over the past year, Aurinia matured into a fully-integrated biopharmaceutical company with capabilities spanning R&D, clinical, regulatory, CMC, and commercial. The recent FDA approval and immediate launch of LUPKYNIS underscores the exemplary performance and expertise of the Aurinia team," commented Peter Greenleaf, President and Chief Executive Officer of Aurinia. "During 2020, we made calculated investments following the positive AURORA clinical trial results by building out a world-class commercial team, signing a major ex-US partnership with Otsuka, and ensuring we can meet future market demand for LUPKYNIS by securing our supply chain by expanding our manufacturing agreement with Lonza. After just 30 days, we are pleased by the uptake of LUPKYNIS by the healthcare community and believe we are on track to meet our internal expectations."

"Launching LUPKYNIS within hours of our approval allows us to focus on getting LN patients who need intervention onto therapy as soon as possible," said Max Colao, Chief Commercial Officer at Aurinia. "We look forward to translating years of innovation and development work, and our early preparation and planning for launch, into commercial success for LUPKYNIS."

Recent Highlights

FDA Approval and Commercial Launch of LUPKYNISTM

On January 22, 2021, the FDA approved LUPKYNIS in combination with a background immunosuppressive therapy regimen to treat adult patients with active LN. LUPKYNIS was approved by the FDA under Priority Review and was previously granted Fast Track designation from the Agency in 2016.

Collaboration and Licensing Agreement with Otsuka Pharmaceutical Co., Ltd.

On December 17, 2020, Aurinia announced it had entered into a collaboration and licensing agreement with Otsuka Pharmaceutical Co., Ltd., for the development and commercialization of oral LUPKYNIS for the treatment of LN in the European Union (EU), Japan, as well as the United Kingdom, Russia, Switzerland, Norway, Belarus, Iceland, Liechtenstein, and Ukraine.

As part of the agreement, Aurinia received an upfront cash payment of $50 million for the license agreement, and has the potential to receive up to an additional $50 million in regulatory milestones. Aurinia will receive tiered royalties on future sales ranging from 10 to 20 percent on net sales upon commercialization, along with additional milestone payments based on the attainment of certain annual sales by Otsuka. In addition, Aurinia will provide LUPKYNIS to Otsuka via a supply agreement under a cost plus arrangement.

Agreement for Dedicated LUPKYNIS Manufacturing Capacity

On December 15, 2020, Aurinia entered into a collaborative agreement with Lonza Ltd. (Lonza) to build a dedicated manufacturing capacity within Lonza’s existing small molecule facility in Visp, Switzerland. The dedicated facility (also referred to as "monoplant") will be equipped with state-of-the-art manufacturing equipment to provide cost and production efficiency for the manufacture of LUPKYNIS, while expanding existing capacity and providing supply security to meet future commercial demand. Upon completion of the monoplant, Aurinia will have the right to maintain exclusive use of the monoplant by paying a quarterly fixed facility fee. The first capital expenditure payment was made in February 2021.

Financial Liquidity at December 31, 2020

As of December 31, 2020, Aurinia had cash, cash equivalents and investments of $423 million compared to $306 million at December 31, 2019. Net cash used in operating activities was $69.9 million for the year ended December 31, 2020 compared to $63.6 million for the year ended December 31, 2019.

The Company believes that it has sufficient financial resources to fund its current plans, which include funding commercial launch activities, manufacturing and packaging of commercial drug supply, conducting our planned R&D programs, and operating activities into at least 2023.

Financial Results for the Year Ended December 31, 2020

For the year ended December 31, 2020, Aurinia recorded a consolidated net loss of $102.7 million or $0.87 per common share.

Revenues were $50.1 million and $0.3 million for the years ended December 31, 2020 and 2019, respectively. The increase of $49.8 million in 2020 was due to the upfront license payment received from Otsuka of $50 million, recorded as licensing revenue in the fourth quarter of 2020.

Research and development (R&D) expenses decreased to $50.3 million for the year ended December 31, 2020 compared to $52.9 million for the year ended December 31, 2019. The primary driver for the decrease of $2.5 million in R&D spend in 2020 was a decrease in drug manufacturing and supply costs, lower Contract Research Organization (CRO) expenses and other third party clinical trial expenses, partially offset by an increase in regulatory related costs as Aurinia prepared for FDA approval.

Corporate, administration and business development expenses increased to $96 million for the year ended December 31, 2020 compared to $22.3 million for the year ended December 31, 2019. The primary driver for the increase of $73.6 million was the build out of commercial infrastructure in advance of approval, which included an increase in salaries and employee benefits, share based compensation expense and professional fees incurred during the year.

Financial Results for the Fourth Quarter Ended December 31, 2020

For the three months ended December 31, 2020, Aurinia recorded a consolidated net loss of $8.1 million or $0.05 per common share.

Revenues were $50 million and $0.03 million for the three months ended December 31, 2020 and 2019, respectively. The increase of $50 million in 2020 was due to the upfront payment from Otsuka of $50 million recorded as licensing revenue.

R&D expenses decreased to $13.2 million for the three months ended December 31, 2020 compared to $13.3 million for the three months ended December 31, 2019. The primary drivers for the slight decrease in R&D spend in 2020 was a decrease in drug manufacturing and supply costs, lower CRO expenses and other third party clinical trial expenses, partially offset by an increase in regulatory related costs as Aurinia prepared for FDA approval.

Corporate, administration and business development expenses increased to $38.8 million for the three months ended December 31, 2020 compared to $7.3 million for the three months ended December 31, 2019. The primary driver for the increase of $31.5 million in 2020 was the build out of commercial infrastructure in advance of approval, which included an increase in salaries and employee benefits, share based compensation expense and professional fees incurred during the quarter.

This press release is intended to be read in conjunction with the Company’s audited financial statements and the Management’s Discussion and Analysis for the year ended December 31, 2020 in the Company’s Annual Report on Form 10-K, which is accessible on Aurinia’s website at www.auriniapharma.com, on SEDAR at www.sedar.com or on EDGAR at www.sec.gov/edgar.

Aurinia will host a conference call and webcast to discuss the fourth quarter and year ended December 31, 2020 financial results today, Wednesday, February 24, 2020 at 4:30 p.m. ET. This event can be accessed on the investor section of the Aurinia website at www.auriniapharma.com.

About Lupus Nephritis

LN is a serious progression of systemic lupus erythematosus (SLE), a chronic and complex autoimmune disease. About 200,000-300,000 people live with SLE in the U.S. and approximately one out of three of these individuals have already developed LN at the time of SLE diagnosis. If poorly controlled, LN can lead to permanent and irreversible tissue damage within the kidney, resulting in kidney failure. Black and Asian individuals with SLE are four times more likely to develop LN and individuals with Hispanic ancestry are approximately twice as likely to develop the disease when compared with Caucasian individuals. Black and Hispanic individuals with SLE also tend to develop LN earlier and have poorer outcomes when compared to Caucasian individuals.

Adaptimmune Reports Fourth Quarter / Full Year 2020 Financial Results and Business Update

On February 25, 2021 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in cell therapy to treat cancer, reported financial results for the fourth quarter and year ended December 31, 2020, and provided a business update (Press release, Adaptimmune, FEB 25, 2021, View Source [SID1234575726]).

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"We are building the cell therapy company of the future for people with cancer. With our ‘2-2-5-2’ by 2025 strategic plan, we will deliver value with marketed SPEAR T-cell products starting with ADP-A2M4 for people with synovial sarcoma," said Adrian Rawcliffe, Adaptimmune’s Chief Executive Officer. "We completed enrollment in our SPEARHEAD-1 trial in approximately 12 months to support our first BLA, which is strong evidence of our ability to execute rapidly. We are focusing the SURPASS trial, using our next-generation ADP-A2M4CD8 product, on lung, gastroesophageal, head and neck, and bladder cancers to identify new indications to take forward to late-stage development."

Planned 2021 data updates1

SPEARHEAD-1 trial with ADP-A2M4 for people with synovial sarcoma
June: preliminary data at American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)
November: full update at Connective Tissue Oncology Society (CTOS)
SURPASS Phase 1 trial with ADP-A2M4CD8 (next-generation product targeting MAGE-A4)
September: update at European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper)
Additional clinical updates
September: update at International Liver Cancer Association (ILCA) conference for ADP-A2AFP Phase 1 trial for people with liver cancer
October: update at American Society for Radiation Oncology (ASTRO) for radiation sub-study of the ADP-A2M4 Phase 1 trial2
November: ADP-A2M4 translational data update at Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)
"2-2-5-2" by 2025 strategic plan
At an Investor Day held in November 2020, the Company outlined its "2-2-5-2" by 2025 strategic plan encompassing:

"2" – Two marketed products targeting MAGE-A4
Estimated potential addressable population in tumor types with significant MAGE-A4 expression, factored for HLA-A23, is ~39,000 patients per year in the US and EU

Durable responses in synovial sarcoma – on track to file a Biologics License Application (BLA) for ADP-A2M4 in 2022

The first commercial opportunity for SPEAR T-cells targeting MAGE-A4 will be in synovial sarcoma with plans to file a BLA in the US in 2022
In 2020, the Company received positive endorsements from regulatory authorities with Regenerative Medicine Advanced Therapy (RMAT) designation and Orphan Drug Designation (ODD) in the US, and access to PRIority MEdicines (PRIME) regulatory support and ODD in the EU
Data from the Phase 1 trial with ADP-A2M4 (presented at CTOS 2020)
In 16 patients with synovial sarcoma, there was an Overall Response Rate of 44% and a Disease Control Rate of 94%
Responses were durable with a median duration of response of 28 weeks with ongoing responses beyond 72 weeks in two patients (median overall survival had not been reached)
These data are considerably superior4 to response rates observed with available second line therapies in synovial sarcoma
SURPASS-2 in esophageal and esophagogastric junction (EGJ) cancers in 1H 2021

The Company will initiate a Phase 2 trial, SURPASS-2, with ADP-A2M4CD8 (next-generation SPEAR T-cells targeting MAGE-A4 that co-express CD8α intended to increase potency) for patients with esophageal or EGJ cancers in 1H 2021
SURPASS-2 is supported by encouraging data from the Phase 1 SURPASS trial (presented at SITC (Free SITC Whitepaper) 2020) with one confirmed partial response (PR) in a patient with EGJ cancer and tumor reductions in two additional patients (1 with esophageal and 1 with EGJ cancer)
The trial will be conducted at multiple centers in North America and the EU, and is intended to enroll 45 people with esophageal or EGJ cancers to be treated with doses up to 10 billion SPEAR T-cells
The Company also presented preclinical data at SITC (Free SITC Whitepaper) 2020 indicating that AKT inhibition during the manufacture of SPEAR T-cells results in a more consistent expansion and phenotype of the final product. This process is currently being used for the Phase 1 SURPASS trial.
"2" – Two additional BLAs for SPEAR T-cell products

Adaptimmune’s Phase 1 SURPASS trial with ADP-A2M4CD8 continues to enroll patients, focusing on lung, gastroesophageal, head and neck, and bladder cancers – indications for which the Company has reported responses or signs of efficacy with its MAGE-A4 targeted products
In 2020, Adaptimmune initiated SPEARHEAD-2 with its first-generation SPEAR T-cells targeting MAGE-A4 in combination with pembrolizumab for people with head and neck cancers
The Phase 1 trial with ADP-A2AFP for people with liver cancer is ongoing. As presented at ILC 2020, nine patients were treated as of the data cut-off and best responses were:
One patient with a complete response, one with stable disease (SD), and two with progressive disease (PD) among the four patients who received ~5 billion or more SPEAR T-cells
Five patients with SD who received doses of 100 million and 1 billion SPEAR T-cells in the first two dose cohorts
"5" – Five new autologous products in the clinic

Adaptimmune has a deep preclinical pipeline from which it expects to bring five new products into the clinic.
These include multiple possibilities for next-generation autologous SPEAR T-cells such as:
ADP-A2AFP SPEAR T-cells co-expressing CD8α
ADP-A2M4 SPEAR T-cells co-expressing IL-7, IL-15, dnTGFβ, and/ or PDE7
Enhancing SPEAR T-cells with IL-7 for proliferation and survival and CCL19 for migration into tumor in collaboration with Noile-Immune Biotech, Inc.
Enhancing SPEAR T-cells using transmembrane and surface immunoregulatory mechanisms with Alpine Immune Sciences, Inc.
The Company is also developing new products, including:
Expanding into HLAs beyond HLA-A2 to increase the addressable patient population
HLA-independent TCR (HiT) candidates for multiple targets including GPC3
A new program for next-generation TILs co-expressing IL-7 in melanoma in collaboration with leading TIL therapy center (CCIT, Denmark)
"2" – Two allogeneic products in the clinic

In January 2020, Adaptimmune announced it had entered into an agreement to co-develop and co-commercialize stem-cell derived allogeneic cell therapies with Astellas
The first target nominated is a HiT targeting mesothelin
At ASGCT (Free ASGCT Whitepaper) 2020, Adaptimmune presented data with evidence of its allogeneic platform demonstrating differentiation of functional T-cells from human-induced pluripotent stem cells (hiPSCs) that can kill MAGE-A4 expressing target cells in vitro –targeted to become the Company’s first allogeneic product in the clinic
Corporate updates from 2020

Despite the impact of the COVID-19 pandemic on the biotechnology industry, Adaptimmune continued to see improved enrollment in its clinical trials
In Q1, the Company received an upfront payment of $50 million from Astellas. The Company is also entitled to receive research funding of up to $7.5 million per collaboration target per year
Underwritten public offering in Q1 generated net proceeds of approximately $90 million
Underwritten public offering in Q2 generated net proceeds of approximately $244 million
Financial Results for the fourth quarter and year ended December 31, 2020

Cash / liquidity position: As of December 31, 2020, Adaptimmune had cash and cash equivalents of $56.9 million and Total Liquidity5 of $368.2 million.

Revenue: Revenue for the fourth quarter and year ended December 31, 2020 was $1.5 million and $4.0 million, respectively, compared to $0.7 million and $1.1 million for the same periods in 2019. The increase was due to revenue arising under the collaboration agreement with Astellas, which was entered into in January 2020.

Research and development (R&D) expenses: R&D expenses for the fourth quarter and year ended December 31, 2020 were $25.8 million and $91.6 million, respectively, compared to $20.4 million and $97.5 million for the same periods in 2019. R&D expenses in the year ended December 31, 2019 included the accrual of a purchase commitment and higher costs for in-process research and development; excluding the impact of these, research and development expenses have increased as the Company progresses development of its cell therapies.

General and administrative (G&A) expenses: G&A expenses for the fourth quarter and year ended December 31, 2020 were $13.2 million and $45.8 million, respectively, compared to $10.7 million and $43.4 million for the same periods in 2019. The increase in G&A expenses was due to an increase in general corporate costs, including professional fees and insurance.

Net loss: Net loss attributable to holders of the Company’s ordinary shares for the fourth quarter and year ended December 31, 2020 was $36.6 million and $130.1 million ($(0.15 per ordinary share), compared to $29.4 million and $137.2 million ($(0.22) per ordinary share) for the same periods in 2019.
Financial Guidance
The Company believes that its existing cash, cash equivalents and marketable securities will fund the Company’s current operations into early 2023, as further detailed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, to be filed with the Securities and Exchange Commission following this earnings release.

Conference Call Information
The Company will host a live teleconference and webcast to provide additional details at 8:00 a.m. EST (1:00 p.m. GMT) today, February 25, 2021. The live webcast of the conference call will be available via the Events page of Adaptimmune’s corporate website at www.adaptimmune.com. An archive will be available after the call at the same address. To participate in the live conference call, if preferred, please dial (833) 652-5917 (US or Canada) or +1 (430) 775-1624 (International). After placing the call, please ask to be joined into the Adaptimmune conference call and provide the confirmation code (2099860).

Milestone Pharmaceuticals to Present at Upcoming Investor Conferences

On February 25, 2021 Milestone Pharmaceuticals Inc. (Nasdaq: MIST), a biopharmaceutical company focused on the development and commercialization of innovative cardiovascular medicines, reported that Joseph Oliveto, President and Chief Executive Officer, will present at the following investor conferences (Press release, Milestone Pharmaceuticals, FEB 25, 2021, View Source [SID1234575722]):

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The Cowen 41st Annual Health Care Conference’s panel on Cardiovascular Disease and Hospital Products on Thursday, March 4, 2021 at 1:30 p.m. Eastern Time.
The H.C. Wainwright Global Life Sciences Conference on Tuesday, March 9, 2021 on demand beginning at 7:00 a.m. Eastern Time.
The Oppenheimer 31st Annual Healthcare Conference on Tuesday, March 16, 2021 at 10:00 a.m. Eastern Time.
A replay of the pre-recorded H.C. Wainwright presentation and live webcast Oppenheimer presentation can be accessed in the News & Events section of Milestone’s website at www.milestonepharma.com. An archived replay of the webcasts will be available on the same website for approximately 90 days following the presentations.