On May 10, 2021 Chugai Pharmaceutical Co., Ltd. (Head office: Tokyo; President & CEO: Osamu Okuda; "Chugai") and SBI Pharmaceuticals Co., Ltd. (Head office: Tokyo; Representative Director & President: Yoshitaka Kitao; "SBI Pharma") reported that the companies decided to terminate the license agreement for the photodynamic diagnostic agent "ALAGLIO Divided Granules 1.5g ("ALAGLIO")" and that Chugai will consequently discontinue marketing of the product (Press release, Chugai, MAY 10, 2021, View Source [SID1234579497]).

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Both companies entered into a license agreement for ALAGLIO on March 13, 2017 which granted Chugai exclusive marketing rights of ALAGLIO in Japan. SBI Pharma obtained regulatory approval for ALAGLIO on September 27, 2017 for the indication of diagnostic agent to visualize non-muscle invasive bladder cancer at the operation of its transurethral resection, and Chugai launched the product on December 19 after the product had been listed on the National Health Insurance reimbursement price list on November 22.

Chugai will discontinue marketing and information provision for ALAGLIO on May 31, 2021. SBI Pharma will announce how these activities will be conducted from June 1, 2021.

Chugai and SBI Pharma will corporate to accomplish a smooth transition and maintain promotion of proper use of the product during the transition period.

Trademarks used or mentioned in this release are protected by law.

On May 10, 2021 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, reported its first quarter 2021 financial results, and provided recent corporate and program highlights (Press release, Aeglea BioTherapeutics, MAY 10, 2021, View Source [SID1234579541]).

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"We’ve had a strong start to 2021 and I am excited to see the significant momentum we have built for our pegzilarginase program from a clinical perspective as well as strengthening our commercial foundation in preparation for a potential FDA approval and launch. In just the last two months, we completed patient randomization for PEACE, secured a commercialization partner for pegzilarginase in Europe and the Middle East and launched THINK ARGININE, an Arginase 1 Deficiency disease education and diagnostic testing initiative in the United States," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "This substantial progress sets us up well for the rest of 2021. We expect to provide topline data from the PEACE study in the fourth quarter, continue progressing our commercial strategy for pegzilarginase and advance our AGLE-177 clinical program in Homocystinuria. These accomplishments are critical milestones as we move closer to our mission of bringing impactful therapies to patients with rare metabolic disorders who currently have limited treatment options."

Recent Highlights and Updates

Corporate

In March, Aeglea announced it has entered into a license and supply agreement with Immedica Pharma AB granting Immedica exclusive commercialization rights in Europe and several Middle Eastern countries. Aeglea will receive an upfront payment of $21.5 million and is eligible for commercial and regulatory milestones of up to approximately $130 million and mid-twenties percentage royalties on net sales.
Pegzilarginase in Arginase 1 Deficiency

In April, the Company completed patient screening and randomization for PEACE, its pivotal Phase 3 clinical trial. Trial enrollment of 32 patients exceeded the target of 30 patients, underscoring the high levels of interest seen from patients, caregivers and investigators. Topline data are expected in the fourth quarter of 2021.
In May, we announced the launch of THINK ARGININE, a disease education initiative to improve the awareness and diagnosis of Arginase 1 Deficiency (ARG1-D). The initiative consists of a comprehensive healthcare provider education campaign and a no-charge diagnostic testing program for adults and children with suspected ARG1-D in the United States.
The no-charge diagnostic testing program includes amino acid testing working with Mayo Clinic Laboratories and genetic testing partnered with Invitae.
Upcoming Events

Aeglea will participate in the upcoming virtual conferences and events:

ISPOR 2021, May 17-20
Virtual Rare Disease Week on Capitol Hill 2021, July 14-22
First Quarter 2021 Financial Results

As of March 31, 2021, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $128.5 million, which excludes the upfront license receivable from Immedica for $21.5 million. Based on Aeglea’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations into 2023.

Research and development expenses totaled $11.9 million for the first quarter of 2021 and $14.6 million for the first quarter of 2020. The decrease was primarily associated with completing certain pre-commercial manufacturing activities for Aeglea’s lead product candidate, pegzilarginase.

General and administrative expenses totaled $6.4 million for the first quarter of 2021 and $4.5 million for the first quarter of 2020. This increase was primarily due to ramping-up the Company’s commercial capabilities and infrastructure.

Net loss totaled $18.2 million and $18.7 million for the first quarter of 2021 and 2020, respectively, with non-cash stock compensation expense of $1.8 million and $1.3 million for the first quarter of 2021 and 2020, respectively.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is a novel recombinant human enzyme, which has been shown to rapidly and sustainably lower levels of the amino acid arginine in plasma. Aeglea is developing pegzilarginase for the treatment of patients with ARG1-D, a rare debilitating and progressive disease characterized by the accumulation of arginine. ARG1-D presents in early childhood and patients experience spasticity, seizures, developmental delay, intellectual disability and early mortality. Aeglea’s Phase 1/2 and Phase 2 open-label extension data for pegzilarginase in patients with ARG1-D demonstrated clinical improvements and sustained lowering of plasma arginine. The Company’s ongoing single, global pivotal Phase 3 PEACE trial is designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction. Pegzilarginase has received multiple regulatory designations, including Rare Pediatric Disease, Breakthrough, Fast Track and Orphan Drug Designations from the FDA as well as Orphan Drug Designation from the European Medicines Agency.

About AGLE-177 in Homocystinuria

AGLE-177 is a novel recombinant human enzyme, which degrades the amino acid homocysteine and its related homocystine dimer. Aeglea is developing AGLE-177 for the treatment of patients with cystathionine beta synthase (CBS) deficiency, also known as Classical Homocystinuria, a rare inherited disorder of methionine metabolism that results in elevated levels of homocysteine and homocystine. Homocysteine accumulation plays a key role in multiple progressive and serious disease-related complications, including thromboembolic vascular events, skeletal abnormalities (including severe osteoporosis), developmental delay, intellectual disability, lens dislocation and severe near sightedness. Preclinical data demonstrated that AGLE-177 improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. AGLE-177 has received U.S. Rare Pediatric Disease and Orphan Drug Designations as well as EU Orphan Drug Designation. Aeglea initiated a Phase 1/2 trial in 2020 and continues patient identification and administrative activities.

On May 10, 2021 Pliant Therapeutics, Inc. (Nasdaq: PLRX) ("the Company"), a clinical stage biotechnology company focused on discovering and developing novel therapeutics for the treatment of fibrosis, reported a corporate update and reported first quarter 2021 financial results (Press release, Pliant Therapeutics, MAY 10, 2021, View Source [SID1234579585]).

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"During the first quarter, we continued the strong momentum of advancing our broad and novel clinical and development-stage portfolio," said Bernard Coulie, M.D., Ph.D., President and Chief Executive Officer of Pliant Therapeutics. "With this progress and a financial runway taking us into 2023, we are well positioned to advance toward our objective of bringing potentially transformative treatments to patients in need."

First Quarter and Recent Highlights

PLN-74809 Phase 2a positron emission tomography (PET) imaging trial preliminary data expected in the first half 2021. This open-label, dose ranging trial is evaluating target receptor occupancy levels of PLN-74809 in the lungs of IPF patients across multiple single-dose cohorts utilizing a PET tracer of the integrin αvβ6. The goal of the trial is to confirm the ability of PLN-74809, a dual selective inhibitor of αvβ6/αvβ1, to penetrate highly fibrotic areas of the lung where αvβ6 expression is highest and bind to its target receptor. Additionally, the trial will establish a pharmacokinetic/ pharmacodynamic (PK/PD) relationship between PLN-74809 plasma exposure and αvβ6 receptor occupancy, allowing us to build a PK/PD model that will inform dose selection in later stage trials.

PLN-74809 Phase 2a INTEGRIS trial in idiopathic pulmonary fibrosis (IPF) continued to build momentum, currently on track to complete enrollment by the end of 2021. The primary endpoints of this 12-week randomized, dose-ranging, double-blind, placebo-controlled trial are the evaluation of PLN-74809’s safety, tolerability, and pharmacokinetics in IPF patients. The Company will also evaluate exploratory efficacy endpoints including Quantitative Lung Fibrosis, or QLF, imaging as well as pulmonary function tests.

PLN-74809 Phase 2a INTEGRIS trial in primary sclerosing cholangitis (PSC) continued to advance, currently on track to complete enrollment in the first half 2022. The primary endpoints of this 12-week randomized, dose-ranging, double-blind, placebo-controlled trial are the evaluation of PLN-74809’s safety, tolerability, and pharmacokinetics in PSC patients. The Company will also evaluate exploratory efficacy endpoints including fibrosis biomarkers such as Pro-C3 and ELF, changes in ALP, and liver imaging.

Successful completion of PLN-1474 Phase 1 trial and transfer of PLN-1474 to Novartis. The Phase 1 trial of PLN-1474 was a safety, tolerability, and pharmacokinetics dose-escalating first-in-human trial that enrolled 84 healthy volunteers. PLN-1474 was rapidly absorbed and well tolerated with no dose-or treatment-limiting toxicities or severe/ serious adverse events observed. In preclinical studies, PLN-1474 was observed to selectively block the αvβ1 integrin-mediated activation of TGF-β, reducing liver fibrosis in animal models. Following the successful completion of this study, PLN-1474 has been transferred to Novartis.
COVID-19 Preparedness

The Company continues to develop and maintain policies and procedures to enable us to operate safely and productively during the COVID-19 pandemic. The Company has experienced delays in clinical trial operations which have impacted and may further impact the expected timing of data readouts. The Company continues to work closely with clinical sites to continue site initiation and operation activities in compliance with study protocols while observing government and institutional guidelines.

First Quarter 2021 Financial Results

Research and development expenses were $18.5 million, as compared to $13.9 million for the prior-year quarter. The increase was due primarily to employee related expenses and higher costs related to the advancement of several programs and ongoing Phase 1/2 clinical trials.
General and administrative expenses were $6.6 million, as compared to $4.0 million for the prior-year quarter. The increase was due to higher personnel-related and professional services expenses.
Net loss of $22.9 million as compared to a net income of $11.0 million for the prior-year quarter due to a decrease in related party revenue driven by the achievement of a first-patient-first-dose milestone in the first quarter of 2020 for the PLN-1474 Phase 1 trial.
As of March 31, 2021, the Company had cash, cash equivalents and short-term investments of $264.1 million. The Company believes it has sufficient funds to meet its operating and capital requirements into 2023.

On May 10, 2021 Sesen Bio (Nasdaq: SESN), a late-stage clinical company developing targeted fusion protein therapeutics for the treatment of patients with cancer, reported operating results for the first quarter ended March 31, 2021 (Press release, Sesen Bio, MAY 10, 2021, View Source [SID1234579604]). The Company’s Biologics License Application (BLA) for the Company’s lead program, Vicineum, is currently under Priority Review with the Food and Drug Administration (FDA) for the treatment of BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with a target Prescription Drug User Fee Act (PDUFA) date of August 18, 2021.

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"We are very pleased with the tremendous progress we continue to make in our four biggest global markets: the US, Europe, the Middle East and North Africa region and China," said Dr. Thomas Cannell, president and chief executive officer of Sesen Bio. "In the US, we continue to work with the FDA as we approach our target PDUFA date, and we are making substantial progress toward launch readiness. We are laser focused on bringing a product to market that we believe will improve patient outcomes while reducing overall healthcare costs to patients globally, and we expect to continue to make progress around the world in the coming months."

US and OUS Regulatory Update

US:

In February 2021, Sesen Bio received notice from the FDA that the BLA for Vicineum was accepted for filing. Along with the acceptance, the Company was granted Priority Review with a target PDUFA date of August 18, 2021 for a decision on the BLA. The FDA also stated that an advisory committee meeting was not currently planned to discuss the BLA.
Europe:

On March 25, 2021, the Company was notified by the European Medicines Agency (EMA) that the Company’s Marketing Authorization Application (MAA) for Vysyneum was found to be valid and that the review procedure had officially started. Sesen Bio submitted the MAA on March 5, 2021, and the Company remains on-track for potential approval of Vysyneum in the EU in early 2022.
On March 31, 2021, the Company received notice from the Committee for Medicinal Products for Human Use (CHMP) of the EMA that the CHMP has conditionally accepted the proprietary brand name Vysyneum for the Company’s product candidate, oportuzumab monatox, in the EU. The Company believes Vysyneum is a brand name with strong marketing potential given its identical pronunciation to the US proprietary brand name Vicineum. Final approval of the Vysyneum brand name is conditional on EMA product approval.
China:

The Investigational New Drug (IND) application for Vicineum for the treatment of BCG-unresponsive NMIBC submitted to the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) was approved on March 19, 2021 ahead of the original timeline of April 2021. This approval triggers a $3M milestone payment to the Company, and authorizes Qilu Pharmaceutical, the Company’s partner in China, to conduct the proposed clinical trial to assess the efficacy and safety of Vicineum in China, at the sole cost of Qilu Pharmaceutical. Assuming a successful trial, Qilu Pharmaceutical anticipates submission of the product market application for Vicineum in 2022, with potential approval in China expected in 2023.
Middle East and North Africa (MENA):

The Company continues to work closely with its partner, Hikma Pharmaceuticals, to submit marketing authorization applications for Vicineum in 2021 in four key markets in the region: the Kingdom of Saudi Arabia, Jordan, Morocco and Egypt. These four markets represent a significant opportunity in the MENA region, as Saudi Arabia, Jordan and Morocco have some of the most advanced healthcare systems in the region while Egypt is the second largest economy in Africa. The Company anticipates the first wave of potential country approvals for Vicineum in the MENA region as early as 2022.
Commercial Update

The Company continues to build its commercial organization with key leadership appointments and a partnership with a leading contract sales organization (CSO), Syneos Health, as it prepares for the anticipated commercial launch of Vicineum in the US in 3Q 2021. Sesen Bio has begun to hire key commercial roles and has entered into a partnership with Syneos Health who will provide speed and logistical support in the hiring and deployment of the sales force. The sales force will include 35 sales representatives across four regions to target approximately 2,000 high prescribers of BCG.
First Quarter 2021 Financial Results

Cash Position: Cash, cash equivalents and restricted cash were $110 million as of March 31, 2021, compared to $55.4 million as of December 31, 2020.
R&D Expenses: Research and development expenses for the first quarter of 2021 were $6.1 million compared to $8.9 million for the same period in 2020. The decrease of $2.8 million was due to decreased costs associated with technology transfer and manufacturing ($4.4 million) and lower clinical trial expense as a result of the Company’s Phase 3 VISTA trial winding down ($0.1 million). This was partially offset by increases in professional services in support of the MAA submission to the EMA ($0.9 million), license milestone fees ($0.6 million) and employee-related compensation ($0.2 million).
G&A Expenses: General and administrative expenses for the first quarter of 2021 were $5.3 million compared to $3.4 million for the same period in 2020. The increase of $1.9 million was due primarily to increases in legal fees ($0.7 million), employee-related compensation driven by increased headcount as part of the commercial build ($0.7 million), professional services ($0.3 million), and other increases ($0.2 million).
Net Loss: Net loss was $55.5 million, or $0.35 per share, for the first quarter of 2021, compared to net income of $41.6 million, or $0.31 per basic share and $0.31 per diluted share, for the first quarter of 2020. The change from net income to net loss was attributable primarily to differences in the non-cash change in the fair value of contingent consideration that is recognized in earnings (or loss) for each respective period.
Conference Call and Webcast Information
Members of the Sesen Bio management team will host a conference call and webcast today at 8:00 AM ET to provide a corporate update and review the Company’s financial results. To participate in the conference call, please dial (844) 831-3025 (domestic) or (315) 625-6887 (international) and refer to conference ID 6229838. The webcast can be accessed in the Investor Relations section of the Company’s website at www.sesenbio.com. The replay of the webcast will be available in the investor section of the Company’s website at www.sesenbio.com for 60 days following the call.

About Vicineum
Vicineum, a locally administered fusion protein, is Sesen Bio’s lead product candidate being developed for the treatment of BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). Vicineum is comprised of a recombinant fusion protein that targets epithelial cell adhesion molecule (EpCAM) antigens on the surface of tumor cells to deliver a potent protein payload, Pseudomonas Exotoxin A. Vicineum is constructed with a stable, genetically engineered peptide tether to ensure the payload remains attached until it is internalized by the cancer cell, which is believed to decrease the risk of toxicity to healthy tissues, thereby improving its safety. In prior clinical trials conducted by Sesen Bio, EpCAM has been shown to be overexpressed in NMIBC cells with minimal to no EpCAM expression observed on normal bladder cells. Sesen Bio is currently in the follow-up stage of a Phase 3 registration trial in the US for the treatment of BCG-unresponsive NMIBC. In February 2021, the FDA accepted for filing the Company’s BLA for Vicineum for the treatment of BCG-unresponsive NMIBC and granted the application Priority Review with a target PDUFA date of August 18, 2021. Additionally, Sesen Bio believes that cancer cell-killing properties of Vicineum promote an anti-tumor immune response that may potentially combine well with immuno-oncology drugs, such as checkpoint inhibitors. For this reason, the activity of Vicineum in BCG-unresponsive NMIBC is also being explored at the US National Cancer Institute in combination with AstraZeneca’s immune checkpoint inhibitor durvalumab.

On May 10, 2021 BWXT Medical Ltd. (BWXT Medical) reported that it has entered into a new long-term, mutually exclusive agreement to manufacture TheraSphere Y-90 Glass Microspheres for Boston Scientific, a leading global medical device company (Press release, BWXT Medical, MAY 10, 2021, View Source [SID1234579625]).

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Developed for the treatment of patients with hepatocellular carcinoma (HCC), TheraSphere treatment is comprised of millions of glass microspheres containing radioactive Yttrium-90, which are delivered directly to liver tumors via catheter and result in minimal exposure to surrounding healthy tissue.

BWXT Medical is a subsidiary of BWX Technologies, Inc. (NYSE: BWXT) and a global supplier of medical isotopes and radiopharmaceuticals.

Under the terms of the agreement, BWXT Medical will invest to automate the production process at its Ottawa, Canada facility, and thereby significantly increase capacity and dependability to support a growing global demand for TheraSphere.

"We congratulate Boston Scientific on its recent receipt of FDA PMA approval of TheraSphere, which expands patient access to this unique technology," said Martyn Coombs, president of BWXT Medical. "We share the aspiration of making a significant difference in the lives of people suffering with cancer, and we look forward to continuing to build a strong supply chain foundation for future growth."