On May 4, 2023 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported that Brian Mueller, Executive Vice President, Chief Financial Officer of BioMarin will present at the Bank of America 2023 Health Care Conference on Wednesday, May 10, 2023 at 3:40pm PT, in Las Vegas, NV (Press release, BioMarin, MAY 4, 2023, View Source [SID1234631015]).

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An audio webcast of the presentation will be available live. You can access the webcast at: View Source An archived version of the remarks will also be available through the Company’s website for a limited time following the conference.

On May 4, 2023 Bicycle Therapeutics plc (NASDAQ: BCYC), a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, reported financial results for the first quarter ended March 31, 2023 and provided recent corporate updates (Press release, Bicycle Therapeutics, MAY 4, 2023, View Source [SID1234631014]).

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"The beginning of 2023 has been impactful for Bicycle and our platform," said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. "We reported encouraging Phase I dose escalation results from BT8009 that showed anti-tumor activity in heavily pre-treated urothelial, lung and breast cancer patients and a median duration of response estimated to be 14 months in the 5 mg/m2 cohort of patients with urothelial cancer. We look forward to providing further updates on BT8009 as well as on our other clinical-stage programs later this year."

"In addition, we entered into multiple research and development collaborations with organizations at the forefront of the development of radiopharmaceuticals to develop Bicycle radio-conjugates (BRCs)," added Dr. Lee. "These collaborations provide further validation of our platform and enable us to advance a broad pipeline of partnered and wholly owned BRCs. This represents the third pillar of our oncology strategy beyond our Bicycle toxin conjugate (BTC) and Bicycle tumor-targeted immune cell agonist (Bicycle TICA) programs. We are pleased with the progress we have made advancing our entire pipeline and expanding the potential applications of the Bicycle platform."

First Quarter 2023 and Recent Highlights

Announced Multiple Strategic Collaborations for Developing BRCs for Potential Oncology Targets
Strategic Collaboration with Novartis. In March 2023, Bicycle announced a strategic collaboration with Novartis to collaborate on the discovery, development and commercialization of BRCs for multiple undisclosed oncology targets. Bicycle will utilize its proprietary phage platform to discover Bicycles to be developed into BRCs. Novartis will be responsible for and fund further development, manufacture and commercialization of the BRCs. Bicycle received a $50 million upfront payment in April 2023 and is eligible for development and commercial-based milestone payments totaling up to $1.7 billion. Bicycle will also be eligible to receive tiered royalties on Bicycle-based medicines commercialized by Novartis.
Collaboration with the German Cancer Research Center. In May 2023, Bicycle announced a collaboration with the German Cancer Research Center (DKFZ) to develop and discover BRCs for potential oncology targets. Bicycle and DKFZ have been collaborating on preclinical development of tumor targeting BRCs which have shown encouraging tumor uptake in preclinical studies. With this new alliance, the relationship has expanded to include a more continuous and purpose-driven commitment towards advancing wholly owned BRCs. Bicycle intends to commence initial testing of its wholly owned BRCs in patients by the end of 2024.
Presented Completed BT8009 Phase I Dose Escalation Results from Ongoing Phase I/II Study of BT8009 at the 2023 American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary (GU) Cancers Symposium. In February 2023, Bicycle presented monotherapy Phase I dose escalation results of the ongoing Phase I/II trial of BT8009, a novel BTC targeting Nectin-4, at the ASCO (Free ASCO Whitepaper) GU Cancers Symposium. BT8009 demonstrated anti-tumor activity in heavily pre-treated urothelial, lung and breast cancer patients with signs of differentiation compared to antibody-based approaches. Bicycle announced that it dosed its first patient in the Phase II expansion portion of the trial and established recommended Phase II doses of 5 mg/m2 weekly and 7.5 mg/m2 administered two-weeks on, one-week off over a 21-day cycle in November 2022. Enrollment in the clinical trial as well as discussions with the U.S. Food and Drug Administration (FDA) remain ongoing. The company expects to provide clinical and regulatory updates on BT8009 in 2023.

BT8009 Granted Fast Track Designation (FTD) by the FDA. In January 2023, Bicycle announced that the FDA granted BT8009 FTD as a monotherapy for the treatment of adult patients with previously treated locally advanced or metastatic urothelial cancer.

Enhanced Leadership Team with Key Appointments

Bicycle appointed Santiago Arroyo M.D., Ph.D., as Chief Development Officer in March 2023. Dr. Arroyo brings to Bicycle over 20 years of biopharmaceutical industry experience, leading the clinical development of therapeutics across multiple disease areas. Dr. Arroyo will be responsible for overseeing all aspects of the pipeline as it continues to expand and advance and as Bicycle prepares to transition into late-stage clinical development.
Bicycle promoted Jennifer Perry, Pharm.D., to Senior Vice President, Commercial. In this role, she will be responsible for establishing and growing the commercial organization.
Financial Results

Cash and cash equivalents were $293.8 million as of March 31, 2023, compared to $339.2 million as of December 31, 2022. The decrease in cash is primarily due to cash used in operating activities. Cash at March 31, 2023 does not include the upfront payment from Novartis.

Research and development expenses were $32.2 million for the three months ended March 31, 2023, compared to $14.3 million for the three months ended March 31, 2022. The increase in expense of $17.9 million was primarily due to increased clinical program expenses for BT5528 and BT8009, Bicycle TICA program development expenses, and other discovery and platform related expenses, as well as increased personnel-related expenses, including incremental non-cash share-based compensation expense of $2.2 million associated with equity grants issued in the three months ended March 31, 2023, offset by incremental UK research and development tax credit reimbursements.
General and administrative expenses were $14.5 million for the three months ended March 31, 2023, compared to $17.0 million for the three months ended March 31, 2022. The decrease of $2.5 million for the three months ended March 31, 2023 as compared to the same period in the prior year was primarily due to a decrease in non-cash share-based compensation expense compared to the previous year.

Net loss was $39.1 million, or $(1.30) basic and diluted net loss per share, for the three months ended March 31, 2023, compared to net loss of $27.6 million, or $(0.93) basic and diluted net loss per share, for three months ended March 31, 2022.

On May 4, 2023 Bicycle Therapeutics plc (NASDAQ: BCYC), a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, reported that it has entered into a collaboration with the German Cancer Research Center (DKFZ), to develop and discover Bicycle radio conjugates (BRCs) for a range of oncology targets.

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Founded in 1964, DKFZ is the largest biomedical research institution in Germany. It is a premier radioligand therapy organization that has a proven track record of supporting the development of such therapies and, together with the University of Heidelberg, was vital to the early development of Pluvicto.

Bicycle and DKFZ have been collaborating on tumor targeting BRCs which in preclinical studies, have shown superior tumor uptake compared to antibody-based approaches. Results from the preclinical study were published in Cancer Research in February 2019 and can be accessed here. With this new alliance, the relationship has expanded to include a more continuous and purpose-driven commitment towards advancing candidates towards the clinic.

Professor Matthias Eder commented that he and his colleagues at DKFZ were excited to work with Bicycle to develop novel radiopharmaceutical therapies. "We have experience working with Bicycle scientists on radiochemistry projects and believe that the physicochemical properties of Bicycle’s constrained bi-cyclic peptides make them ideal ligands for targeted radioisotope delivery."

"We believe the advancement of BRCs is a promising application of the Bicycle platform and represents the third leg within our core oncology focus. Building on the recent announcement of our collaboration with Novartis, we are excited to deepen our relationship with DKFZ, a leading radioligand research organization, to seek to accelerate the development of our wholly owned BRCs," said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. "The goal of the collaboration is to develop a pipeline of wholly owned BRC compounds with the potential to become new treatment options for patients living with cancer."

Dr. Lee continued, "We are thrilled to have now announced multiple collaborations in the radiopharmaceutical space this year, which we believe provides further validation of the Bicycle platform. Likewise, we remain encouraged by data generated to date from our wholly owned pipeline of Bicycle Toxin Conjugates (BTCs) and Bicycle tumor-targeted immune cell agonists (Bicycle TICAs) which we believe demonstrates differentiation compared to antibody-based approaches and other modalities. We look forward to providing multiple updates this year as we advance BT8009, BT5528 and BT7480 in the clinic. In addition, we intend to commence initial testing of our own BRCs in patients by the end of 2024."

On May 4, 2023 Aligos Therapeutics, Inc. (Nasdaq: ALGS), a clinical stage biopharmaceutical company focused on developing novel therapeutics to address unmet medical needs in liver and viral diseases, reported recent business progress and financial results for the first quarter 2023 (Press release, Aligos Therapeutics, MAY 4, 2023, View Source [SID1234631012]).

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"Our team is off to a great start in 2023, having already achieved multiple important milestones," said Lawrence Blatt, PhD, MBA, Chairman & CEO of Aligos. "For our key NASH program, we recently collected and presented data demonstrating the excellent PK properties for our Phase 2 gelcap formulation, which has enabled the initiation of Ph2 drug supply manufacturing activities. We remain on track to file an IND for Ph2 in Q4 of this year. For our COVID-19 protease inhibitor, ALG-097558, we recently submitted the CTA to enable initiation of a first in human safety and PK study with dosing anticipated to start mid-year. For our chronic hepatitis B portfolio, our capsid assembly modulator continues to show best-in-class HBsAg lowering activity with data recently presented at the Global Hepatitis Summit meeting showing subjects dosed with 300 mg ALG-000184 for up to 28 weeks achieving HBsAg reductions of up to 1.65 log10 IU/mL. Finally, our CHB siRNA drug, ALG-125755, recently completed enrollment in the 3rd cohort of Part 2, which is evaluating single ascending doses in CHB patients. We plan to continue to share emerging data from both of these CHB clinical programs next month at EASL 2023 and at future scientific meetings."

Recent Business Progress

Aligos Portfolio of Drug Candidates

NASH Program

In Part 3 of Study ALG-055009-301, the bioavailability of the Phase 2 gel cap formulation was found to compare favorably relative to the liquid formulation used in the SAD (Part 1) and MAD (Part 2) portions of the study. Exposures for the gel cap formulation were similar to the liquid formulation with low variability and no evidence of a meaningful food effect.
After conducting a comprehensive data review, the proposed doses for Ph2 were identified and include a planned top dose (gel cap) approximately equivalent to 0.75 mg (liquid formulation). Because this planned top dose was not specifically studied in earlier MAD cohorts, Aligos plans to conduct in Q2 2023 a MAD cohort evaluating the safety, PK and PD of 0.75 mg ALG-055009 (liquid formulation) x 14 days.
Phase 2 enabling activities, including 13 week GLP toxicology studies and gel cap manufacturing, were initiated in Q1 and both activities remain on track for a Q4 2023 Ph2 IND filing.
COVID-19

The CTA for the FIH Study ALG-097558-701 was successfully submitted in the UK in April. Additional Ph1 startup activities are ongoing to support initiation of ALG-097558 dosing, which is anticipated to start mid-year.
HBV Programs

Additional HBsAg lowering data from ongoing cohorts in Study ALG-000184-201 were presented at the Global Hepatitis Summit in April. These data showed that subjects dosed with 300 mg ALG-000184 + entecavir for up to 28 weeks are achieving HBsAg reductions of up to 1.65 log10 IU/mL and a majority (4 of 5 subjects) that have been dosed for ~24 weeks demonstrated at least a 1.00 log10 IU/mL reduction in HBsAg levels.
Financial Results for the First Quarter 2023

Cash, cash equivalents and investments totaled $103.5 million as of March 31, 2023, compared with $125.8 million as of December 31, 2022. We continue to believe our cash balance provides sufficient cash to fund planned operations through the end of 2024.

Net losses for the three months ended March 31, 2023, were $23.0 million or basic and diluted net loss per common share of $(0.53), compared to net losses of $35.6 million or basic and diluted net loss per common share of $(0.84) for the three months ended March 31, 2022.

Research and development (R&D) expenses for the three months ended March 31, 2023, were $18.1 million compared with $31.7 million for the same period of 2022. The decrease in R&D expenses for this comparative period is primarily attributable to a decrease in third-party expenses due to our continued wind down related to the discontinuation of our STOPS and ASO programs, and the manufacturing of drug supply in advance of our clinical and nonclinical activities. Total R&D stock-based compensation expense incurred for the three months ended March 31, 2023, was $2.2 million compared with $2.0 million for the same period of 2022.

General and administrative (G&A) expenses for the three months ended March 31, 2023, were $8.5 million compared with $6.5 million for the same period of 2022. The increase in G&A expenses for this comparative period is primarily attributable to an increase in legal and related costs offset by a decrease in facility expenses. Total G&A stock-based compensation expense incurred for the three months ended March 31, 2023, was $1.5 million compared with $1.8 million for the same period of 2022.

On May 4, 2023 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported first quarter 2023 financial results and recent portfolio and business updates. As of March 31, 2023, Alector’s cash, cash equivalents and investments totaled $669.3 million (Press release, Alector, MAY 4, 2023, View Source [SID1234631011]).

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"We continue the momentum built in 2022, making steady progress in the first quarter advancing our late-stage immuno-neurology programs," said Arnon Rosenthal, Ph.D., Chief Executive Officer of Alector. "We remain on track to engage with regulatory authorities this summer regarding the INFRONT-3 trial of latozinemab, and we are targeting early 2025 for a data readout from the pivotal trial. As we look ahead to the remainder of 2023, we anticipate important milestones across our pipeline, including completing enrollment in the Phase 2 clinical trial of our TREM2 candidate, AL002, in patients with early Alzheimer’s disease and new data from the INFRONT-2 clinical trial of latozinemab in FTD-C9orf72. I look forward to providing updates on our late-stage clinical and early research programs in the coming months."

Gary Romano, M.D., Ph.D., Chief Medical Officer of Alector added, "We have an exciting year ahead of us as we continue to progress latozinemab, AL101 and AL002 through the clinic. We are very encouraged by recent drug development programs in neurological disorders that have successfully leveraged treatment effects on biomarkers to support clinical efficacy. All our clinical studies are designed to deliver robust biomarker and clinical data that will evaluate the therapeutic effects of our antibody candidates on disease pathophysiology and clinical disease progression. In our Phase 2 INFRONT-2 trial of latozinemab, we have observed normalization of CSF and plasma levels of GFAP. Increased levels of GFAP are associated with faster rates of brain atrophy in FTD. We look forward to sharing additional data from the FTD-C9orf72 cohort later this year, and we continue to advance our pivotal Phase 3 INFRONT-3 study, which evaluates those biomarkers and clinical endpoints in a double-blinded, randomized, placebo-controlled trial of latozinemab for the treatment of FTD-GRN."

Recent Clinical Updates

Immuno-Neurology Portfolio
Progranulin Programs (latozinemab (AL001) and AL101) Being Developed in Collaboration with GSK

Alector is preparing to engage with regulatory authorities in mid-2023 to discuss statistical analysis plans for the pivotal Phase 3 INFRONT-3 clinical trial evaluating the efficacy and safety of latozinemab (AL001) in patients with frontotemporal dementia due to a progranulin gene mutation (FTD-GRN). Emerging knowledge in the field may enable completion of the study with fewer patients and/or a shorter treatment duration. The company is targeting an INFRONT-3 data readout in early 2025 with the potential for a Biologics License Application (BLA) filing in late 2025 subject to regulatory discussion outcomes.

As previously announced, Alector plans to present additional data from the INFRONT-2 Phase 2 clinical trial of latozinemab in FTD-C9orf72 during the second half of 2023. The data is expected to include additional results from the entire FTD-C9orf72 cohort. To date, the company has presented 12-month results from six symptomatic FTD-C9orf72 trial participants treated with latozinemab. The FTD-C9orf72 cohort represents the first clinical dataset in an indication where latozinemab elevated progranulin above physiological levels, supporting the company’s efforts to expand the progranulin franchise into additional neurodegenerative disease indications, including Alzheimer’s disease (AD), Parkinson’s disease (PD) and amyotrophic lateral sclerosis (ALS).

The company submitted a poster, PK/PD modeling of progranulin elevation in blood and CSF to support AL101 dose selection and Phase 2 study design, for the 2023 Annual Meeting of the American College of Clinical Pharmacology (ACCP) being held in Bellevue, Washington from September 10-12, 2023. The pharmacokinetic and pharmacodynamic (PK/PD) modeling supports dose selection for the Phase 2 study of AL101 in AD. AL101 is intended to elevate progranulin levels in a manner similar to latozinemab but with different PK/PD properties, and the company plans to investigate AL101 for the treatment of AD and PD. As previously reported, Alector and GSK plan to initiate a global Phase 2 clinical trial with AL101 in early AD.
TREM2 Program (AL002) Being Developed in Collaboration with AbbVie

Alector received a $17.8 million milestone payment from AbbVie in March 2023 after enrolling and dosing the first patient in a long-term extension (LTE) of the INVOKE-2 Phase 2 clinical trial in patients with early AD. Alector may also receive up to an additional $12.5 million from AbbVie to support enrollment in the INVOKE-2 trial. Alector is on track to complete enrollment in the trial in the third quarter of 2023, with top-line data expected by the fourth quarter of 2024. The INVOKE-2 trial is designed to evaluate the efficacy and safety of AL002 in slowing disease progression in individuals with early AD.

AbbVie has an exclusive option to globally develop and commercialize AL002. AbbVie’s exercise of that option would prompt a $250 million payment to Alector.
Early Research Pipeline

Alector continues to strategically invest in and advance its innovative research portfolio to fuel its development pipeline. The company’s target discovery engine robustly integrates functional genomics, external and internal data, and machine learning. Additionally, the company is applying its proprietary blood brain barrier technologies to support its next-generation product candidates.
Recent Corporate Updates

Kristina Cutter, M.P.H., was promoted to Chief Regulatory, Pharmacovigilance, and Quality Assurance Officer. Ms. Cutter has over 20 years of biotechnology industry experience and oversees the global regulatory and safety strategy as well as Alector’s quality systems, which ensure the right to operate across Alector’s immuno-neurology portfolios.
First Quarter 2023 Financial Results

Revenue. Collaboration revenue for the quarter ended March 31, 2023, was $16.5 million, compared to $24.5 million for the same period in 2022. This decrease was primarily due to less revenue recognized from the GSK agreement.

R&D Expenses. Total research and development expenses for the quarter ended March 31, 2023, were $51.9 million, compared to $53.0 million for the same period in 2022. The decrease in R&D expenses was mainly driven by the decrease in our latozinemab programs due to the timing of manufacturing activities offset by an increase in the AL002 program due to higher enrollment activities and the addition of the LTE trial.

G&A Expenses. Total general and administrative expenses for the quarter ended March 31, 2023, were $14.8 million, compared to $15.6 million for the same period in 2022 mainly driven by a decrease in consulting expenses related to accounting, recruiting, IT, and other general expenses.

Net Loss. For the quarter ended March 31, 2023, Alector reported a net loss of $45.9 million, or $0.55 per share, compared to a net loss of $44.6 million, or $0.54 per share, for the same period in 2022.

Cash Position. Cash, cash equivalents, and investments were $669.3 million as of March 31, 2023. Management expects that this will be sufficient to fund current operations through 2025.

2023 Guidance. Management anticipates, for the year ending 2023, collaboration revenue to be between $15 million and $25 million, total research and development expenses to be between $225 million and $245 million and total general and administrative expenses to be between $60 million and $70 million.