Alector to Present at the Bank of America Securities Virtual Health Care Conference

On May 7, 2020 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported that Shehnaaz Suliman, M.D., MBA, M.Phil., president and chief operating officer of Alector, will present at the Bank of America Securities Virtual Health Care Conference on Wednesday, May 13, 2020, at 3:40 p.m. ET (Press release, Alector, MAY 7, 2020, View Source [SID1234557342]).

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A live webcast of the presentation will be available on the "Events & Presentations" page within the Investors section of the Alector website at View Source A replay will be available on the Alector website for 30 days following the event.

Aeglea BioTherapeutics Reports First Quarter 2020 Financial Results and Corporate Highlights

On May 7, 2020 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare and other high-burden diseases, reported its first quarter 2020 financial results, and provided recent corporate and program highlights (Press release, Aeglea BioTherapeutics, MAY 7, 2020, View Source [SID1234557331]).

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"These past few months have brought unique challenges as we navigate the impact of COVID-19, and reminds us all of the critical need for healthcare innovation and new medicines. These needs are all too familiar for people with rare diseases, like Arginase 1 Deficiency, where adequate treatment options are often not available," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "With our recently completed financing, we have the resources to advance our pegzilarginase program for Arginase 1 Deficiency through regulatory submission and potential FDA approval. With the recent approval of our Clinical Trial Application for ACN00177 for the treatment of Homocystinuria, the company is positioned to bring forward its second clinical-stage human enzyme – both with the potential to be transformative solutions for rare genetic disorders."

Recent Highlights & COVID-19 Update

Pegzilarginase in Arginase 1 Deficiency

Aeglea is working with treating physicians to implement individual treatment plans and potentially developing a home healthcare option for patients enrolled in the Phase 3 PEACE trial.
To date, most enrolled patients have continued to receive treatment. The Company is developing a plan to analyze results for patients that are missing data points. The Phase 3 PEACE trial protocol is designed in such a way that a patient may miss a few doses without being disqualified from the trial.
The supply chain has not experienced any significant impact at this time and the Company currently has sufficient supply available for completion of its ongoing clinical trials.
The Company expects to complete enrollment of its Phase 3 PEACE trial in the third quarter of 2020 and to provide topline data in the first quarter of 2021.
ACN00177 in Homocystinuria

In April, Aeglea announced the approval of its Clinical Trial Application (CTA) by the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) for ACN00177, a novel engineered human enzyme therapy designed to treat Homocystinuria, a serious metabolic disorder characterized by elevated plasma homocysteine which leads to a wide range of life-altering complications and reduced life expectancy.
The Company continues its patient identification and administrative activities to quickly move forward with dosing patients once trial sites are able to initiate clinical trials.
While Aeglea continues to prepare to initiate a Phase 1/2 trial for ACN00177 in the second quarter of 2020, the timing will depend on determination by individual sites that each is ready to open for recruitment in light of COVID-19; the Company’s priorities at this time are to avoid further overburdening hospital staff and to minimize the risk of trial participants exposure to COVID-19.
Corporate Highlights

In April, the Company strengthened its financial position with a public offering resulting in gross proceeds of $138 million, which extended its cash runway through 2022.
The Company implemented policies and practices to protect the health and wellbeing of the Company’s employees and communities, including asking employees to work from home and implementing a work rotation for essential lab employees.
Aeglea suspended all business travel and transitioned all meetings, including conference attendance and industry events, to virtual meetings.
First Quarter 2020 Financial Results

As of March 31, 2020, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $50.5 million. In addition, in April 2020 the Company raised approximately $129.6 million in net proceeds from a public offering. Based on Aeglea’s current operating plan, and taking into account the net offering proceeds, management believes it has sufficient capital resources to fund anticipated operations through 2022.

Research and development expenses totaled $14.6 million for the first quarter of 2020 and $14.4 million for the first quarter of 2019. The increase was primarily associated with investing in manufacturing and pre-commercial activities for Aeglea’s lead product candidate, pegzilarginase; ramp-up in toxicology, nonclinical studies, and manufacturing activities for ACN00177 in Homocystinuria; and personnel-related expenses. The increase was offset by a decrease in clinical development expenses as a result of completing a Phase 1/2 clinical trial in patients with Arginase 1 Deficiency, a Phase 1 clinical trial in patients with advanced solid tumors, and concluding enrollment of a Phase 1/2 combination trial in patients with small cell lung cancer.

General and administrative expenses totaled $4.5 million for the first quarter of 2020 and $3.3 million for the first quarter of 2019. This increase was primarily due to additional employee headcount, ramping up commercial capabilities, and additional facilities to support company growth.

Net loss totaled $18.7 million and $17.2 million for the first quarter of 2020 and 2019, respectively, with non-cash stock compensation expense of $1.3 million and $1.1 million for the first quarter of 2020 and 2019, respectively.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is an enhanced human arginase that enzymatically lowers levels of the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency (ARG1-D), a rare debilitating disease presenting in childhood with persistent hyperargininemia, severe progressive neurological abnormalities and early mortality. Pegzilarginase is intended for use as an enzyme therapy to reduce elevated blood arginine levels in patients with ARG1-D. 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 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.

About ACN00177 in Homocystinuria

Aeglea is developing ACN00177 for the treatment of patients with cystathionine beta synthase (CBS) deficiency, also known as Classical Homocystinuria. 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. ACN00177 has been designed as a novel recombinant human enzyme, which degrades the amino acid homocysteine and its related homocystine dimer. With this mechanism, ACN00177 is intended to lower the abnormally high blood levels of homocysteine in patients with Homocystinuria. Preclinical data demonstrated that ACN00177 improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. The Company received approval from the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) for its Clinical Trial Application (CTA) and continues to prepare to initiate a Phase 1/2 trial in the second quarter of 2020.

Cellectar Reports First Quarter 2020 Financial Results and Provides a Corporate Update

On May 7, 2020 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported financial results for the three months ended March 31, 2020 and provided a corporate update (Press release, Cellectar Biosciences, MAY 7, 2020, View Source [SID1234557330]).

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First Quarter and Recent Corporate Highlights

·Announced CLR 131 achieved primary efficacy endpoints from its Phase 2 CLOVER-1 study in median third line relapsed/refractory (r/r) and Non-Hodgkin’s lymphomas (NHL) and median sixth line r/r multiple myeloma (MM) and in the Phase 1 r/r MM dose escalation study at the 50mCi and 75mCi total body doses. The 75mCi total body dose demonstrated:

§Overall response rate (ORR) 42.8% in r/r MM

§33% ORR in triple class refractory r/r MM

§50% ORR in high risk r/r MM

§43.0% ORR and 14.3% complete responses (CR) in r/r NHL

§100% ORR and 25% CR in r/r Lymphoplasmacytic lymphoma/Waldenstrom’s macroglobulinemia (LPL/WM) across all therapeutic doses tested

·Completed the pre-planned highest dose cohort of the Phase 1 r/r MM dose escalation study

·Received Orphan Drug Designation for CLR 131 in LPL/WM from the U.S. Food and Drug Administration (FDA)

·Appointed Dr. Igor Grachev, Chief Medical Officer

·Extended the Phase 2 CLOVER-1 study in relapsed/refractory B-cell lymphomas to enroll additional patients at a dose of CLR 131 delivering 100mCi in a two-cycle dosing regimen

"CLR 131 has consistently demonstrated favorable safety and tolerability across dosing levels and encouraging response rates even in a very difficult to treat sixth line patient population that included high risk, triple class refractory and daratumamab refractory patients," said James Caruso, president and CEO of Cellectar. "We continue to enroll patients at the 100mCi level and believe the two-cycle dosing regimen we now use will further increase response rates, the durability of responses and maintain or improve upon CLR 131’s predictable safety and tolerability product profile."

The health and safety of our employees has always been paramount and that does not change during this pandemic. Within our employee base, we are not aware of any confirmed cases of COVID-19 to date. We will continue taking appropriate measures to prevent the outbreak from affecting our employees, while also managing the financial well-being of the company. Furthermore, at this time, the pandemic has not caused any disruption in our supply chain or in enrollment within our ongoing clinical studies.

First Quarter 2020 Financial Highlights

·Cash and Cash Equivalents: As of March 31, 2020, the company had cash and cash equivalents of $7.1 million compared to $10.6 million at December 31, 2019. Cash used in operating activities was approximately $3.5 million during the three months ended March 31, 2020 as compared to $2.8 million during the three months ended March 31, 2019. Consistent with prior guidance, the company believes its cash on hand is adequate to fund operations into the first quarter of 2021.

·Research and Development Expense: R&D expense for the three months ended March 31, 2020 was $2.6 million, compared to $2.3 million for the three months ended March 31, 2019. The increase in R&D expense of approximately 13% was primarily a result of general R&D cost from personnel related expenses and clinical project costs. Manufacturing and related costs and pre-clinical studies were relatively consistent.

·General and Administrative Expense: G&A expense for both the three months ended March 31, 2020 and March 31, 2019 was $1.3 million and remained relatively consistent in both periods.

·Net Loss: The net loss attributable to common stockholders for the three months ended March 31, 2020 was ($4.0) million, or ($0.42) per share, compared to ($3.6) million, or ($0.76) per share, in 2019.

Center in Singapore Expanding Access to Radiation Therapy Technology in the Fight Against Cancer

On May 7, 2020 Varian (NYSE: VAR) and the National University Cancer Institute, Singapore (NCIS), reported that are partnering to expand access to cancer solutions in Singapore in the fight against cancer (Press release, Varian Medical Systems, MAY 7, 2020, View Source [SID1234557329]). In 2018, 26,164 cases of cancer were diagnosed in Singapore, and it is expected to more than double to 57,319 cases over the next 20 years .1

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Two Varian VitalBeam medical linear accelerators, together with an Edge radiosurgery system with HyperArc technology, will be installed at NCIS, thereby expanding radiation therapy treatment options for cancer patients in Singapore.

Dr Francis Ho, head of the Department of Radiation Oncology at NCIS said: "We are proud to continue bringing in innovative therapy treatments, such as HyperArc, to help treat patients with cancer. We are committed to providing quality patient care to the people in Singapore. With this technology, we hope to reduce inefficiencies in plan creation and treatment delivery, leading to reduced treatment times for patients."

Kenneth Tan, president, Varian Asia Pacific said: "With an ever-rising number of patients being diagnosed with cancer and an increasingly aging population, it is now more important than ever to help patients and clinicians in Singapore in the fight against cancer. Through this partnership with the National University Cancer Institute, Singapore, and by bringing in high-quality radiation therapy combined with connected cancer care solutions, we are working towards our vision of living in a world without fear of cancer."

HyperArc technology is designed to automate and simplify sophisticated treatments such as stereotactic radiosurgery (SRS) and make them available to more cancer patients around the world. VitalBeam offers high-quality, high-throughput radiation therapy, which enables healthcare professionals to expand clinical capabilities and serve more patients with advanced treatment technology over time.

Momenta Pharmaceuticals Reports First Quarter 2020 Financial and Operating Results

On May 7, 2020 Momenta Pharmaceuticals, Inc. (Nasdaq: MNTA), a biotechnology company focused on discovering and developing novel biologic therapeutics to treat rare immune-mediated diseases, reported its financial results for the first quarter ended March 31, 2020 (Press release, Momenta Pharmaceuticals, MAY 7, 2020, View Source [SID1234557328]).

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"Despite the turbulence imposed by the COVID-19 pandemic, Momenta has remained focused on advancing our development pipeline for rare, immune-mediated diseases," said Craig A. Wheeler, President and Chief Executive Officer of Momenta Pharmaceuticals. "We are targeting key readouts from our lead programs by the end of the third quarter. These readouts will include interim proof-of-concept data from nipocalimab in MG and data from Part B of our Phase 1/2 study of M254 in ITP. We expect data from both programs will further demonstrate the potential of these programs, each with franchise opportunities across a range of large market auto- and alloimmune diseases. Finally, we remain in a strong corporate position, with cash to fund operations through at least the third quarter 2021, as we continue to leverage our platform for growth."

First Quarter 2020 Highlights, Recent Events and Anticipated Upcoming Milestones

Novel Therapeutics Pipeline:

Nipocalimab (M281): a fully human anti-neonatal Fc receptor (FcRn) aglycosylated immunoglobulin G (IgG1) monoclonal antibody (mAb)

•We announced in February that Vivacity-MG, the Company’s Phase 2 clinical study of nipocalimab in generalized myasthenia gravis (gMG), achieved its target enrollment. Momenta remains on track to report top-line interim data from this study by the third quarter of 2020.

•Unity, the Company’s global multi-center Phase 2 clinical study of nipocalimab in hemolytic disease of the fetus and newborn (HDFN), continues to enroll patients at sites where they can be safely accommodated.

•Momenta continues to activate sites globally for its Energy Study, the Company’s adaptive Phase 2/3 clinical study of nipocalimab in warm autoimmune hemolytic anemia (wAIHA), however it has temporarily suspended patient enrollment due to the COVID-19 pandemic.

M254 (hsIgG): a hypersialylated immunoglobulin designed as a high potency alternative for intravenous immunoglobulin (IVIg)

•The Company’s multi-part Phase 1/2 clinical trial in idiopathic thrombocytopenic purpura (ITP) is progressing through Part B, which is evaluating M254 in a single ascending dose (SAD) cohort of ITP patients, followed by 1,000mg/kg of IVIg. Enrollment is ongoing, however it has slowed due to the COVID-19 pandemic. The Company plans to complete and report data from Part B in the third quarter of 2020 and expects to initiate Part C of the study in the fourth quarter of 2020.

•The Company’s planned Phase 2 study of M254 in chronic inflammatory demyelinating polyneuropathy (CIDP) is expected to initiate in 2021, following completion of Parts C and D from its ongoing Phase 1/2 trial of M254 in ITP.

M230 (CSL730): a recombinant Fc multimer being developed in collaboration with CSL

•A Phase 1 clinical trial to evaluate the safety and tolerability of M230 in healthy volunteers is ongoing. Momenta’s partner, CSL, plans to introduce a subcutaneous formulation into the Phase 1 program in 2020.

Momenta’s SIFbody platform combines multiple Fc’s with antibody Fabs to optimally activate Fc effector function, and to effectively deplete target cells.

•The Company has initiated IND-enabling studies for M267, a SIFbody candidate targeting CD38. Pre-clinical data suggest this candidate has the potential to be a best-in-class therapeutic to target CD38-expressing cells, which are prevalent in plasmacyte-mediated diseases such as multiple myeloma, AL amyloidosis and other rare, autoantibody-mediated diseases.

Legacy Products:

GLATOPA 20 mg and 40 mg: U.S. Food and Drug Administration (FDA) approved generic versions of COPAXONE 20 mg and 40 mg, developed and commercialized in collaboration with Sandoz

•In the first quarter of 2020, Momenta recorded $8.7 million in product revenue from Sandoz’s sales of GLATOPA products.

M710: a proposed biosimilar to EYLEA (aflibercept) candidate being developed in collaboration with Mylan

•Mylan continues its pivotal clinical trial in patients with diabetic macular edema to compare safety, efficacy and immunogenicity of M710 with EYLEA. Mylan expects to target U.S. submission in 2021, while monitoring and navigating potential COVID-19 issues.

Corporate:

•In April 2020, Momenta provided an update on clinical trial activities and business operations amid the COVID-19 pandemic, including measures implemented to protect the health and safety of its personnel, as well as patients and healthcare professionals involved with its clinical studies; to ensure its clinical studies were able to continue unimpeded for patients already enrolled; and to preserve the integrity of its clinical data, in line with updated guidance from the FDA. These business continuity measures currently remain in place, as the Company continues to closely monitor the situation and guidance from public health authorities. Momenta expects to resume activities for affected studies when conditions permit.

First Quarter 2020 Financial Results

Revenue:

In the first quarter of 2020, the Company recorded $8.7 million in product revenue from Sandoz’s sales of GLATOPA, compared to $2.4 million for the same period in 2019. The increase in product revenue from the prior year period was primarily due to higher net sales of GLATOPA, driven by volume increases.

Research and development revenue for the first quarter of 2020 was $0.2 million, compared to $1.8 million for the same period in 2019. The decrease in research and development revenue of $1.6 million, or 89%, was primarily due to lower reimbursement revenue for GLATOPA expenses and lower revenue recognized from Mylan’s upfront payment associated with the biosimilar collaboration.

Total revenue for the first quarter of 2020 was $8.9 million compared to $4.1 million for the same period in 2019.

Operating Expenses:

Research and development expenses for the first quarter of 2020 were $34.2 million, compared to $28.0 million for the same period in 2019. The increase of $6.2 million, or 22%, was primarily due to an increase in manufacturing and clinical trial costs for nipocalimab and M254 and increased spending on M710, offset in part by lower lease costs.

General and administrative expenses for the first quarter of 2020 were $14.6 million, compared with $24.2 million for the same period in 2019. The decrease of $9.6 million, or 40%, was primarily due to lower depreciation and rent costs due to the modification of the Bent St. lease and lower legal costs.

Total GAAP operating expenses were $49.6 million in the first quarter of 2020. First quarter 2020 non-GAAP operating expense was $44.7 million. Non-GAAP operating expense is total operating expenses, less stock-based compensation expense, restructuring expense and collaborative reimbursement revenue. See "Non-GAAP Financial Information and Other Disclosures" and the table below entitled "Reconciliation of GAAP Results to Non-GAAP Financial Measures" for a reconciliation of GAAP operating expense to non-GAAP operating expense.

Net Loss:

The Company reported a net loss of $39.6 million, or $0.34 per share for the first quarter of 2020 compared to a net loss of $44.8 million, or $0.46 per share for the same period in 2019.

Liquidity:

At March 31, 2020, Momenta had $487.9 million in cash, cash equivalents, and marketable securities. This compares to $545.1 million at December 31, 2019 in cash, cash equivalents, and marketable securities.

2020 Financial Guidance

Momenta provides non-GAAP operating expense guidance, which it believes can enhance an overall understanding of its financial performance when considered together with GAAP financial measures. Refer to the section of this press release below entitled "Non-GAAP Financial Information and Other Disclosures" for further discussion of this subject.

Non-GAAP operating expense is total operating expenses, less stock-based compensation expense, restructuring expense and collaborative reimbursement revenues. Due to lower clinical trial enrollment trends as a result of the COVID-19 pandemic, Momenta anticipates its full-year non-GAAP operating expenses will be lower than $220 – $240 million, as previously guided for 2020. The Company anticipates providing an update on expected non-GAAP operating expense for 2020 as part of its second quarter 2020 financial results.

Non-GAAP Financial Information and Other Disclosures

Momenta uses a non-GAAP financial measure, non-GAAP operating expense, to provide operating expense guidance. Momenta believes this non-GAAP financial measure is useful to investors because it provides greater transparency regarding Momenta’s operating performance as it excludes non-cash stock compensation expense, restructuring expense and collaborative reimbursement revenue. This non-GAAP financial measure should not be considered a substitute or an alternative to GAAP total operating expense and should not be considered a measure of Momenta’s liquidity. Instead, non-GAAP operating expense should only be used to supplement an understanding of Momenta’s operating results as reported under GAAP. Momenta has not provided GAAP reconciliation for its forward-looking non-GAAP annual or quarterly operating expense because Momenta cannot reliably predict without unreasonable efforts the timing or amount of the factors that substantially contribute to the projection of stock compensation expense, which is excluded from the forward-looking non-GAAP financial measure. The Company does not expect restructuring expense and collaboration reimbursement revenue to be material.

Conference Call Information

Management will host a conference call and webcast today at 8:30 am ET to discuss these results and provide an update on the Company. A live webcast of the conference call may be accessed on the "Investors" section of the Company’s website, www.momentapharma.com. Please go to the site at least 15 minutes prior to the call in order to register, download, and install any necessary software. An archived version of the webcast will be posted on the Momenta website approximately two hours after the call.

To access the call, you may also dial (866) 209-9686 (domestic) or (825) 312-2288 (international) prior to the scheduled conference call time and provide the access code 9178486.