On May 4, 2023 Blueprint Medicines Corporation (NASDAQ: BPMC) reported financial results and provided a business update for the first quarter ended March 31, 2023 (Press release, Blueprint Medicines, MAY 4, 2023, View Source [SID1234631016]).

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"Our first quarter was marked by executional progress across the multiple growth opportunities that Blueprint Medicines has to expand our impact and transform outcomes for thousands of patients worldwide. We drove strong AYVAKIT revenue growth, presented seminal data for AYVAKIT in indolent systemic mastocytosis, and worked to progress our broad pipeline by rapidly resolving the partial clinical hold for BLU-222 and generating early clinical data across our portfolio," said Kate Haviland, Chief Executive Officer at Blueprint Medicines. "Blueprint Medicines is ready to further solidify our leadership in systemic mastocytosis and scale the impact of AYVAKIT by addressing the medical needs of a substantially larger group of patients with our anticipated FDA label expansion for indolent systemic mastocytosis in a few weeks."

First Quarter 2023 Highlights and Recent Progress

Systemic mastocytosis (SM)

· Presented full registrational PIONEER trial data for AYVAKIT in patients with indolent systemic mastocytosis (ISM) at the 2023 American Academy of Asthma, Allergy, and Immunology (AAAAI) Annual Conference. These data included a statistically significant and clinically meaningful improvement in total symptom score that deepened over time, with improvements shown across all individual symptoms and in quality-of-life measures. AYVAKIT showed a favorable safety profile compared to placebo, supporting potential for chronic treatment. Read the press release here.
· U.S. Food and Drug Administration (FDA) target action date for AYVAKIT supplemental new drug application for ISM is May 22, 2023.

EGFR-driven non-small cell lung cancer (NSCLC)

· Announced plans to present results from the ongoing dose escalation of the Phase 1/2 CONCERTO trial of BLU-451 in EGFR exon 20 insertion-positive NSCLC, showing early safety and clinical activity, at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
· Announced plans to present updated results from the dose escalation of the Phase 1/2 SYMPHONY trial showing safety and tolerability of BLU-945 both as a monotherapy and in combination with osimertinib in late-line EGFR-driven NSCLC at the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting.
· Presented real-world data showing that NSCLC patients with an EGFR L858R mutation have worse outcomes compared to patients with an EGFR exon 19 deletion mutation when treated with first-line osimertinib, and new preclinical data showing the combination of BLU-945 and osimertinib enhanced tumor regression and survival compared to osimertinib alone in a treatment-naïve EGFR L858R-mutant cell-derived model at the 2023 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. Read the poster presentation here.
· Today announced acceptance of an investigational new drug (IND) application to the FDA for BLU-525.

CDK2-vulnerable breast and other cancers

· Announced the FDA lifted the partial clinical hold on patient enrollment in the VELA trial of BLU-222. Patients already enrolled in the trial have continued on the study and trial sites are working to resume patient enrollment.
· Announced plans to present results from the ongoing dose escalation of the Phase 1/2 VELA trial of BLU-222 in breast cancer and other cancers vulnerable to CDK2 inhibition, showing evidence of monotherapy safety and pathway modulation, at the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting.

Key Upcoming Milestones

The company plans to achieve the following milestones by mid-2023:

· Obtain FDA approval and initiate launch of AYVAKIT in ISM in mid-2023.
· Present initial CONCERTO trial dose escalation data in EGFR exon 20 NSCLC at ASCO (Free ASCO Whitepaper) 2023.
· Present initial VELA trial dose escalation data at ASCO (Free ASCO Whitepaper) 2023.
· Nominate a development candidate targeting wild-type KIT for chronic urticaria by mid-2023.

First Quarter 2023 Results

· Revenues: Revenues were $63.3 million for the first quarter of 2023, including $39.1 million of net product revenues from sales of AYVAKIT/AYVAKYT and $24.2 million in collaboration revenues. Blueprint Medicines recorded revenues of $62.7 million in the first quarter of 2022, including $23.8 million of net product revenues from sales of AYVAKIT/AYVAKIT and $38.9 million in collaboration revenues.
· Cost of Sales: Cost of sales was $3.2 million for the first quarter of 2023, as compared to $5.1 million for the first quarter of 2022.
· R&D Expenses: Research and development expenses were $112.1 million for the first quarter of 2023, as compared to $103.1 million for the first quarter of 2022. This increase was primarily due to increased compensation related costs driven by increased headcount, along with increased costs related to early discovery efforts. Research and development expenses included $10.1 million in stock-based compensation expenses for the first quarter of 2023.
· SG&A Expenses: Selling, general and administrative expenses were $71.0 million for the first quarter of 2023, as compared to $57.1 million for the first quarter of 2022. This increase was primarily due to increased compensation related costs driven by increased headcount, along with increased costs associated with expanding our commercial infrastructure for commercialization of AYVAKIT/AYVAKYT. Selling, general, and administrative expenses included $13.1 million in stock-based compensation expenses for the first quarter of 2023.
· Net Loss: Net loss was $129.6 million for the first quarter of 2023, or a net loss per share of $2.15, as compared to a net loss of $106.0 million for the first quarter of 2022, or a net loss per share of $1.79.
· Cash Position: As of March 31, 2023, cash, cash equivalents and investments were $961.3 million, as compared to $1,078.5 million as of December 31, 2022.

2023 Financial Guidance

Blueprint Medicines has updated its financial guidance and now anticipates approximately $135 million to $145 million in AYVAKIT net product revenues for advanced SM and GIST in 2023, and $40 million to $50 million in collaboration revenues from existing collaborations in 2023. This guidance excludes revenue from the anticipated AYVAKIT indication expansion in ISM in mid-2023. The company continues to expect that its existing cash, cash equivalents and investments, together with anticipated future product revenues, will provide sufficient capital to enable the company to achieve a self-sustainable financial profile.

Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:00 a.m. ET today to discuss first quarter 2023 financial results and recent business activities. The conference call may be accessed by dialing 833-470-1428 (domestic) or 929-526-1599 (international), and referring to conference ID 668091. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

Upcoming Investor Conferences

Blueprint Medicines will participate in one upcoming investor conference:

· Goldman Sachs 44th Annual Global Healthcare Conference on Wednesday, June 14, 2023 at 12:20 pm ET.

A live webcast of each presentation will be available by visiting the Investors & Media section of Blueprint Medicines’ website at View Source A replay of the webcasts will be archived on Blueprint Medicines’ website for 30 days following each presentation.

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.