On April 22, 2021 ASLAN Pharmaceuticals (Nasdaq:ASLN), a clinical-stage immunology focused biopharmaceutical company developing innovative treatments to transform the lives of patients, reported financial results for the fourth quarter and full year ended December 31, 2020 and provided an update on its clinical development activities (Press release, ASLAN Pharmaceuticals, APR 22, 2021, View Source [SID1234578351]).

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Dr Carl Firth, Chief Executive Officer, ASLAN Pharmaceuticals, said: "ASLAN made significant progress in the fourth quarter of 2020 and that momentum has carried over into 2021. After completing recruitment of the third cohort in our multiple ascending dose trial for ASLAN004 in the fourth quarter, we announced positive interim data supportive of its potential as a novel, first-in-class antibody targeting IL-13R with a differentiated efficacy and safety profile in atopic dermatitis. We are on track to complete enrolment of the expansion cohort with an additional 27 patients by mid-2021 and anticipate reporting topline data in the third quarter of 2021. At the same time, we are continuing to prepare for our Phase 2b study, which we expect to initiate in the second half of 2021. ASLAN is in a strong financial position with the necessary resources to fund its development activities to achieve important value creating milestones for shareholders."

Fourth quarter 2020 and recent business highlights

Clinical development

ASLAN004

In March 2021, ASLAN announced positive interim unblinded data from the three dose cohorts of the ongoing Phase 1 randomised, double-blind placebo controlled multiple ascending dose (MAD) study of ASLAN004 for the treatment of moderate to severe atopic dermatitis (AD). ASLAN004, a novel, first-in-class antibody, was well tolerated across all doses and showed improvements compared to placebo in all efficacy endpoints, supporting its potential as a differentiated treatment for AD.
ASLAN003

New data from a study conducted by the University of Liverpool, UK, published in the Toxicology in Vitro Journal, demonstrated that, out of a panel of six dihydroorotate dehydrogenase (DHODH) inhibitors tested, ASLAN003 has the lowest potential for hepatotoxicity despite being one of the most potent inhibitors of DHODH.
Corporate updates

In March 2021, raised gross proceeds of approximately US$69 million, including the full exercise of an over-allotment option, through an underwritten public offering of 17,250,000 American Depositary Shares (ADSs) representing 86,250,000 ordinary shares at a price to the public of US$4.00 per ADS.
In February 2021, raised gross proceeds of approximately US$18 million resulting from the sale of its ordinary shares through a private placement to new institutional investors, Vivo Capital and Surveyor Capital (a Citadel company).
Between October 2020 and February 2021, raised gross proceeds of approximately US$21.5 million through at-the-market offerings.
Appointed Neil Graham, MBBS, MD, MPH and Kathleen M. Metters, PhD as independent directors. Dr Graham is an expert in immunology and inflammation with more than 30 years’ experience in global drug development and commercialisation, including 10 years at Regeneron Pharmaceuticals, Inc., where he was instrumental in the development of dupilumab. Dr Metters has more than 30 years’ experience in the discovery and development of novel therapies for the treatment of chronic diseases, including autoimmune diseases. She held a number of senior positions at Merck & Co., previously leading work on External Discovery and Preclinical Sciences and was Senior Vice President and Head of Worldwide Basic Research.
Anticipated upcoming milestones

Completion of MAD clinical study of ASLAN004 in moderate-to-severe AD patients and topline clinical results expected in the third quarter of 2021.
Initiation of Phase 2b study of ASLAN004 for AD expected in the second half of 2021.
Fourth quarter 2020 financial highlights

Cash used in operations for the fourth quarter of 2020 was US$5.1 million compared to US$5.1 million in the same period in 2019.
Research and development expenses were US$2.9 million in the fourth quarter of 2020 compared to US$2.7 million in the fourth quarter of 2019.
General and administrative expenses were US$3.0 million in the fourth quarter of 2020 compared to US$3.3 million in the fourth quarter of 2019.
Net loss for the fourth quarter of 2020 was US$5.7 million compared to a net loss of US$29.6 million for the fourth quarter of 2019, which included a one-time impairment charge of US$23 million related to the write-down of varlitinib in the fourth quarter of 2019.
Full year 2020 financial highlights

Cash used in operations for the year ended 31 December 2020 was US$15.1 million compared to US$25.8 million in 2019.
Research and development expenses were US$9.3 million for the full year 2020 compared to US$16.6 million in 2019. The decrease was driven by the completion of clinical studies related to varlitinib.
General and administrative expenses were US$7.2 million for the full year 2020 compared to US$8.5 million in 2019.
Net loss attributable to stockholders for the full year 2020 was US$16.2 million compared to a loss of US$47.0 million in 2019, which included a one-time impairment charge of US$23 million related to the write-down of varlitinib in the fourth quarter of 2019. Excluding the non-cash impairment charge, net loss for the full year 2019 was US$23.9 million.
Cash and cash equivalents totaled US$14.3 million as of 31 December 2020, including US$7.4 million raised from at-the-market offerings in 2020, compared to US$22.2 million as of 31 December 2019. The cash balance as of year-end 2020 excludes proceeds from the additional financing activities completed in the first quarter of 2021 which raised combined gross proceeds of approximately US$101 million. With the additional financing activities in the first quarter of 2021, management believes that its cash and cash equivalents will be sufficient to fund operations into 2023.The weighted average number of ADSs outstanding for the year 2020 was 38.4 million compared to 32.5 million for 2019. One ADS is the equivalent of five ordinary shares.

On April 22, 2021 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in cell therapy to treat cancer, reported that it will report financial results and provide a business update for the first quarter ended March 31, 2021, before the US markets open on Thursday, May 6, 2021 (Press release, Adaptimmune, APR 22, 2021, View Source [SID1234578367]). Following the announcement, the Company will host a live teleconference and webcast at 9:00 a.m. EDT (2:00 p.m. BST) that same day (details below).

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The press release will be available in the investor section of Adaptimmune’s corporate website at www.adaptimmune.com. A live webcast of the conference call and replay can be accessed at https://bit.ly/2Ry9DdR.

To participate in the live conference call, please dial (833) 652-5917 (U.S. 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 (9271335).

On April 22, 2021 Shanghai Henlius Biotech, Inc. (2696.HK) reported that the New Drug Application (NDA) of serplulimab injection (HLX10), a novel anti-PD-1 monoclonal antibody (mAb), for the treatment of unresectable or metastatic microsatellite instability-high (MSI-H) solid tumors that fail to respond to the standard therapy, has been accepted by the National Medical Products Administration (NMPA) and proposed to be granted priority review (Press release, Shanghai Henlius Biotech, APR 22, 2021, View Source [SID1234578383]). Serplulimab is potentially to be the first anti-PD-1 mAb in MSI-H solid tumors in China. For this indication, patients are screened for specific MSI-H tumor markers, rather than classifying the tumor types, covering a wide range of cancer types. Henlius adopts the "Combo+Global" development strategy for serplulimab, focusing on indication differentiation and combination therapy, with a total of 10 clinical trials conducted worldwide.

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The co-leading principal investigator of serplulimab Phase 2 clinical trial in unresectable or metastatic microsatellite instability-high or mismatch repair-deficient (MSI-H/dMMR) solid tumors, Professor Shukui Qin of Cancer Center of Nanjing Jinling Hospital, said, "Serplulimab showed excellent anti-tumor activity in preclinical and early-stage clinical studies, and the results of the Phase 2 clinical study in MSI-H solid tumors also states the favorable safety and efficacy. We hope that this China-developed novel PD-1 inhibitor will be launched as soon as possible, so that more patients with solid tumors could be accessible to high quality immunotherapy new options."

Executive Director, Chief Executive Officer and President of Henlius, Mr. Wenjie Zhang, said, "Serplulimab is the core innovative mAb of Henlius. Focusing on combination immunotherapy and international clinical trials, we have built up a differentiation development strategy for the product. Steady progress has been made in all clinical studies. We are very grateful to all the subjects, physicians and colleagues that have contributed to the NDA acceptance of serplulimab. We will push forward the clinical process in more indications as soon as possible and look forward to benefiting more patients around the world."

Several pivotal Phase 3 clinical trials covering comprehensive cancer types with high incidence

With the "Combo+Global" strategy, steady progress has been made in the clinical studies of 2 monotherapies and 8 combination therapies of serplulimab with chemotherapy, in-house VEGF and EGFR targets mAbs. Henlius actively carries out combination therapies that cover a wide range of tumor types with high incidence such as lung cancer, esophageal cancer, hepatocellular cancer, gastric cancer, head and neck cancer, and implements differentiation clinical development based on the Chinese cancer characteristics. Among them, while there are few explorations in the gastric cancer neoadjuvant/adjuvant area of PD-1 inhibitors, Henlius has conducted Phase 3 clinical studies in this field, leading the clinical trial progress around the globe, with the aim of enabling gastric cancer patients to benefit from the early line of immunotherapy. What’s more, the company has achieved all-around layout in the first-line of lung cancer, and has entered pivotal Phase 3 clinical trials in first-line squamous non-small cell lung cancer (sqNSCLC), first-line non-squamous non-small cell lung cancer (nsqNSCLC) and first-line small cell lung cancer (SCLC). The NDA filing of HLX10 in combination with chemotherapy for the first-line treatment of sqNSCLC in China will also be expected in the second half of 2021.

The all-around international layout to benefit emerging markets

The manufacturing and development of serplulimab is strictly in accordance with international standards and its manufacturing facility based in Shanghai has passed the European Union (EU) and China GMP certification. Henlius developed the international layout for serplulimab, which has been approved for clinical trials in China, the United States, the EU and other countries and regions. About 2000 patients have been enrolled in China, Turkey, Poland, Ukraine, Russia, etc., which shows confidence and recognition on the quality of the product in the international market. Apart from conducting international trials of HLX10, Henlius also actively seeks for international cooperation opportunities with the aim of benefiting more patients in the world, especially patients in emerging markets. Henlius has reached a collaboration agreement with PT Kalbe Genexine Biologics (KG Bio), upon which KG Bio is granted exclusive rights to develop and commercialize serplulimab in relation to its first monotherapy and two combination therapies in 10 Southeast Asian countries.

With the continuous development of the "Combo+Global" strategy, Henlius continues to build a diversified pipeline of innovative drug candidates with serplulimab as the lead, actively accelerating innovation and improving innovation efficiency, and committing to bringing affordable and high-quality innovative biologics to patients around the world.

About MSI-H solid tumors

The defect of mismatch repair (MMR) that can lead to base mismatch or insert in microsatellites during DNA replication, and the accumulation of incorrect bases usually causes microsatellite instability (MSI)[1]. MSI-H often occurs in several cancer types, such as endometrial cancer, colorectal cancer, gastric cancer, renal cell carcinoma, ovarian cancer, etc[2]. Studies have revealed that the prevalence of MSI-H across all tumor types is 14%[3]. Patients who suffer from this disease usually have higher response rates for immune checkpoint inhibitors[4-5]. Thus, MSI-H is becoming a more and more important biomarker for the immunotherapy predictions of patients with solid tumors. If the patient is MSI-H positive and meets the treatment criteria, the corresponding immunotherapy can be carried out without screening tumor sites and pathological classification, which aligns with the advanced concept of precision medicine and is applicable to a wide range of cancer types. Currently, the U.S. Food and Drug Administration (US FDA) has approved PD-1 target mAb for the treatment of second-line MSI-H/dMMR advanced solid tumors and first/second-line MSI-H/dMMR colorectal cancers. While there are still no anti-PD-1 mAb approved for MSI-H/dMMR advanced solid tumors in China, the treatment needs are far from being met.

On April 22, 2021 Bristol Myers Squibb reported that upcoming cell therapy plant in the Netherlands should help slash turnaround times for patients receiving its CAR-T therapies there (Press release, Bristol-Myers Squibb, APR 22, 2021, View Source [SID1234578425]).

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Right on the heels of not one, but two CAR-T approvals in the U.S., Bristol Myers Squibb is bolstering its commercial cell therapy ambitions on the other side of the Atlantic.

The company on Thursday pegged Leiden, Netherlands, as the home of its first cell therapy factory in Europe and its fifth worldwide. With planning for site design and development underway, BMS expects construction to kick off later this year.

BMS’ swift cell therapy scale-up, which also includes a commercial plant-to-be in Massachusetts, follows FDA green lights for the company’s closely-watched CAR-T therapies Breyanzi and Abecma in February and March, respectively. Neither med has won an approval yet in Europe.

The new Leiden plant will be kitted out for commercial manufacturing of multiple cell therapy products using cutting-edge tech. For one, the company will tap virtual reality to train incoming cell processing workers, a BMS spokeswoman said via email. The company will make use of electronic batch records, as well as automated bioreactors and automated cartridge-based flow cytometers at the plant, she added.

The site, which will stand at around 19,000 square meters (204,514 square feet), will also be equipped to handle future capacity upgrades.

BMS offered a glimpse at some of the other tech it’s working on during a virtual cell therapy manufacturing tour with reporters last month. At the time, the company said it was working on a platform that could cut CAR-T manufacturing timelines from weeks to less than 10 days, plus new tech to speed up testing, which takes eight days now.

As part of BMS’ cell therapy push in Europe, the company will hire "several hundred" new staffers over the coming years, Ann Lee, Ph.D., senior vice president of cell therapy development and operations at BMS, said in a release.

CAR-T meds are created using a patient’s own T cells, which are extracted, genetically modified and then infused back into patients to help the body kill disease.

The company’s lymphoma CAR-T Breyanzi scored approval in early February with a 24-day target turnaround time from T-cell collection to delivery back to the patient. In the U.S., BMS handles the bulk of the manufacturing work itself.

The Leiden plant will help BMS collect and manufacture cell therapies in Europe without the barrier of intercontinental shipping. Reducing manufacturing lag time is critical for all CAR-T players.

CAR-T manufacturing is a complicated beast—a lesson BMS knows only too well. In December, the FDA hit a Lonza facility producing viral vectors for Breyanzi with a Form 483, a development that might have played a role in the company’s inability to score an FDA approval for the drug in 2020. Breyanzi’s approval by year-end 2020 was a key requirement for a multibillion-dollar payout for investors.

The FDA had also flagged concerns at BMS’ Bothell, Washington, plant in October. Now, however, the Bothell facility is equipped for commercial production, and the company’s Summit, New Jersey, plant is making the switch to turn out therapies for the market.

In late February, BMS unveiled designs on a 244,000-square-foot cell therapy factory in Devens, Massachusetts. The company hopes to start engineering runs at the plant in late 2021 or early 2022, Snehal Patel, global head of cell therapy manufacturing at BMS, said during last month’s tour. Once the plant is up and running, BMS says it will use the site for a good chunk of its commercial cell therapy manufacturing.

On April 22, 2021 Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-driven biopharmaceutical group, reported its sales performance for the first quarter of 2021 (Press release, Ipsen, APR 22, 2021, View Source [SID1234578316]):

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Total Sales growth of 5.5% at CER, or 0.6% as reported, to €658.5m, despite impact of the pandemic
An increase in Specialty Care sales of 6.4%1 to €611.5m, driven by the growth of Cabometyx (cabozantinib), Decapeptyl (triptorelin), Somatuline (lanreotide) and Dysport (botulinum toxin type A)
A Consumer Healthcare sales decline of 5.4%1 to €47.0m, reflecting the ongoing effects of COVID-19, partly offset by recovery in China
European Commission approval of Cabometyx, in combination with nivolumab, as a first-line treatment for patients living with advanced renal cell carcinoma
Guidance for FY 2021 confirmed
David Loew, Chief Executive Officer, commented:
"We delivered a strong performance in the first quarter, despite the persistently difficult COVID-19 environment, driven by our unrelenting focus on patients. Our execution was in line with our plans and our financial guidance for the year. Ipsen’s Oncology medicines stood out in the period, while the regulatory approval in Europe of the Cabometyx combination with nivolumab in first-line renal cell carcinoma marked the start of an important launch. The anticipated registration of a lanreotide-generic medicine in Europe is fully aligned with our expectations.

In the near term, we look forward to trial results in first-line liver cancer for the Cabometyx combination with atezolizumab, as well as regulatory progress with palovarotene in FOP. We continue to support our ambitions of strengthening the pipeline and driving sustainable growth, based on our patient-focused strategy. I am very proud of the great execution by colleagues around the world, especially as many teams continue to experience challenging conditions during the pandemic."

FY 2021 guidance
The Company today confirms its financial guidance for FY 2021, which incorporates an assumed progressive global recovery from COVID-19 by H2 2021. A phased launch of generic lanreotide in Europe by mid-2021 is also assumed, as is a limited impact of the potential launch of octreotide or lanreotide generics in the U.S.

Total Sales

Growth of more than 4.0% at CER

Core Operating Margin

Greater than 30.0%, excluding any potential impact of incremental investments from external innovation

Currency impact
Ipsen anticipates an adverse impact of 2% from currencies on Total Sales in FY 2021, based on the level of exchange rates at the end of March 2021.

Somatuline-generic update
During the quarter, a positive outcome was received for a generic formulation of lanreotide in 60mg, 90mg and 120mg dose presentations by the Reference Member State, Denmark; the closure of the Decentralized Procedure was also achieved. National marketing authorizations have recently been granted for a lanreotide generic medicine in France, Denmark, Hungary and Latvia. These developments were consistent with Ipsen’s expectations.

Business development
In the quarter, Ipsen and Fusion Pharmaceuticals Inc. completed an asset purchase agreement to acquire Ipsen’s intellectual property and assets related to IPN-1087, a small molecule in Phase I development targeting neurotensin receptor 1, a protein expressed on multiple solid-tumor types.

Conference call
A conference call and webcast for investors and analysts will begin at 2:30pm Paris time today. Participants should dial in to the call early and can register here; a recording will be available on ipsen.com, while the webcast can be accessed here. The event ID is 2495337.

Calendar
The Company intends to publish its H1 2021 results on 29 July 2021 and its nine-months’ sales update on 21 October 2021. The Annual Shareholders’ Meeting will be held behind closed doors on 27 May 2021.

Notes
All financial figures are in € millions (€m). The performance shown in this announcement covers the three-month period to 31 March 2021 (the quarter, the first quarter or Q1 2021), compared to three-month period to 31 March 2020 (Q1 2020) respectively, unless stated otherwise. Growth rates are at CER, unless stated otherwise. Commentary on performance is based on CER, unless stated otherwise.