On October 26, 2023 Blueprint Medicines Corporation (NASDAQ: BPMC) reported financial results and provided a business update for the third quarter ended September 30, 2023 (Press release, Blueprint Medicines, OCT 26, 2023, View Source [SID1234636364]).

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"In the first full quarter following AYVAKIT’s launch in indolent systemic mastocytosis, we saw strong and steady growth in both patients treated and revenue, reflecting a highly favorable reception to AYVAKIT’s unique and compelling clinical profile and the effectiveness of our ongoing efforts to bring AYVAKIT to all patients who can benefit from treatment," said Kate Haviland, Chief Executive Officer at Blueprint Medicines. "Through the end of this year and into 2024, we anticipate continued steady growth in AYVAKIT revenue driven by both existing and new prescribers as we execute a disciplined approach to managing operating expenses and allocating capital to our most important investments."

Third Quarter 2023 Highlights and Recent Progress

Systemic mastocytosis (SM) and other mast cell disorders

· Third quarter AYVAKIT net revenue grew 90 percent year-over-year to $54.2 million, with $49.1 million coming from the U.S, in the first full quarter of ISM launch.

Upcoming 2023 Milestones

Blueprint Medicines plans to achieve the following milestones by the end of 2023:

· Present data from Part 1 of the HARBOR trial of elenestinib in indolent SM at the American Society of Hematology (ASH) (Free ASH Whitepaper) conference in December 2023.

Third Quarter 2023 Results

· Revenues: Revenues were $56.6 million for the third quarter of 2023, including $54.2 million of net product revenues from sales of AYVAKIT/AYVAKYT and $2.4 million in collaboration revenues. Blueprint Medicines recorded revenues of $65.9 million in the third quarter of 2022, including $28.6 million of net product revenues from sales of AYVAKIT/AYVAKYT and $37.3 million in collaboration and license revenues.
· Cost of Sales: Cost of sales was $2.8 million for the third quarter of 2023, as compared to $3.0 million for the third quarter of 2022. The decrease was primarily due to a decrease in the cost of collaboration-related sales.
· R&D Expenses: Research and development expenses were $110.3 million for the third quarter of 2023, as compared to $128.0 million for the third quarter of 2022. This decrease was primarily due to a focused approach towards optimizing operational efficiency across our portfolio as we execute across our top priority programs and the timing of manufacturing of clinical trial materials. Research and development expenses included $11.2 million in stock-based compensation expenses for the third quarter of 2023.
· SG&A Expenses: Selling, general and administrative expenses were $70.7 million for the third quarter of 2023, as compared to $57.6 million for the third quarter of 2022. This increase was primarily due to an increase in compensation and personnel related costs driven by our first quarter field force expansion to support the AYVAKIT launch in ISM. Selling, general, and administrative expenses included $11.9 million in stock-based compensation expenses for the third quarter of 2023.

· Net Loss: Net loss was $133.7 million for the third quarter of 2023, or a net loss per share of $2.20, as compared to a net loss of $133.2 million for the third quarter of 2022, or a net loss per share of $2.23.
· Cash Position: As of September 30, 2023, cash, cash equivalents and investments were $827.2 million, as compared to $1,078.5 million as of December 31, 2022. Our cash and investments provide a durable capital position which enables us to reach a self-sustainable profile.

Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:00 a.m. ET today to discuss third quarter 2023 financial results and recent business activities. The conference call may be accessed by dialing 833-470-1428 (domestic) and referring to conference ID 368229. 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 the following upcoming investor conferences:

· Stifel Healthcare Conference on Wednesday, November 15, 2023 at 12:40 pm ET.

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

On October 26, 2023 SEED Therapeutics Inc. ("SEED"), a biotech company focused on harnessing and engineering Molecular Glues for targeted protein degradation (TPD) of disease-causing proteins, reported at Targeted Protein Degradation (TPD) Think Tank symposium that it had successfully discovered a ST-00937, a new chemical entity (NCE) molecular glue that has already achieved IND Candidate status and will be advanced for the treatment of cancers in SEED’s first IND filing, as early as 2024 (Press release, BeyondSpring Pharmaceuticals, OCT 26, 2023, View Source;utm_medium=rss&utm_campaign=seed-therapeutics-disclosesnovel-molecular-glue-ind-candidate-for-cancerat-targeted-protein-degradation-think-tank-symposium [SID1234636363]).

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SEED is a subsidiary of BeyondSpring, Inc. (NASDAQ: BYSI), a clinical-stage biopharmaceutical company focusing on developing innovative therapies.

Dr. James Tonra, SEED President, and Chief Scientific Officer, said, "Preclinical data over the last two to three years has uncovered new and important cancer indications for agents that can inhibit or degrade the RNA splicing factor RBM39, but clinical activity in these new indications is minimal or absent."

According to Dr. Tonra, SEED’s research and development scientists recognized this unmet need, as well as the opportunity to discover yet unrecognized additional indications for an RBM39 targeted molecular glue. "Our seasoned R&D team proceeded to discover and file a patent application for an oral RBM39 degrader that causes complete tumor regressions in select tumor models without detectable toxicities. SEED’s rapid progress on this program towards clinical testing within a year of project initiation reflects the power and potential of TPD science, as well as SEED’s expertise and determination to lead the way to patient benefits and the first regulatory approval with an RBM39 degrader."

The SEED TPD Think Tank was led by SEED Co-Founders who are at the forefront of TPD thought leadership: Nobel Laureate Dr. Avram Hershko, who uncovered the Ubiquitin Proteasome System almost 50 years ago; SEED Chairman and CEO Dr. Lan Huang, who pioneered the understanding of E3 ligase structure by solving the first E3 ligase structure over 20 years ago; Professor Michele Pagano, Howard Hughes Medical Institute Investigator, TPD biology and cancer expert, and chair of New York University biochemistry and molecular pharmacology departments; and Professor Ning Zheng, Howard Hughes Medical Institute Investigator, TPD structural biologist, and Professor of Pharmacology at the University of Washington.

According to Dr. Huang, "The SEED TPD Think Tank shared key TPD developments across academia and industry. But importantly, our SEED Co-Founders, our invited TPD field thought leaders who also presented, and our domestic and foreign attendees engaged in an interactive session that encouraged and challenged scientists, researchers, and developers to extend the boundaries and prospects for TPD through new thinking on TPD molecular mechanism, structural basis, protein proximity in live cells, rational degrader design, and the use of AI-based modeling and computation to speed up discovery efforts."

Invited presenters shared their latest research and insights on translating TPD science to overcoming the hurdles to target "undruggable" disease-causing proteins:

Prof. Chittaranjan Das, Purdue University, E3 Ligase Structure
Prof. Gaurav Bhardwaj, University of Washington, Computational Design
Prof. Lan Huang, University of California Irvine, Proteomics/Proximity
Prof. Luca Busino, University of Pennsylvania, TPD Cancer Biology
Dr. Jesus Izaguirre, AtomMap, AI Drug Design
According to Dr. Huang, "The full impact of TPD on human disease can only be realized with continued scientific advancements on the selection of right E3 ligase for most, if not all disease-causing protein."

Dr. Huang concluded: "At SEED, we thrive on our ability to leverage the scientific leadership of our distinguished Co-Founders. Their research, insight, and conviction allow SEED to focus and accelerate basic research into a fast-expanding TPD drug pipeline, enabled by our unique ability to identify the right E3. This symposium provided a valuable exchange on new directions, opportunities, and challenges on TPD drug development. Together, we are building an exciting ecosystem of best minds in TPD field that will continue to benefit from open collaborations and synergies between academic and industry, as we continue to translate high science into medicine for patients with significant unmet medical needs."

The first "TPD Think Tank" symposium was held at SEED Therapeutics’ laboratory in King of Prussia, PA.

On October 26, 2023 Ascendis Pharma A/S (Nasdaq: ASND) reported updated and new data from its ongoing Phase 1/2 IL-Believe Trial of TransCon IL-2 β⁄γ in a poster presentation at ESMO (Free ESMO Whitepaper) 2023, the annual meeting of the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) held in Madrid, Spain (Press release, Ascendis Pharma, OCT 26, 2023, View Source [SID1234636362]). The data included longer-term follow up of previously presented TransCon IL-2 β⁄γ monotherapy data from the IL-Believe Trial, and was the first presentation of dose escalation data informing recommended Phase 2 dose (RP2D) for TransCon IL-2 β⁄γ in combination with a checkpoint inhibitor.

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As of the August 15, 2023 data cutoff, 46 patients were enrolled into dose escalation: 25 to monotherapy and 21 to combination therapy. Patients were heavily pretreated including some who previously progressed on checkpoint inhibitors. Anti-tumor clinical responses were observed with TransCon IL-2 β⁄γ both as monotherapy (colorectal cancer with confirmed partial response (PR)) and in combination with pembrolizumab (small cell lung cancer (SCLC), 1 with confirmed PR and, subsequent to the August 15, 2023 data cutoff, 1 ongoing with an unconfirmed complete response).

"These updated data reinforce the promising Phase 1 monotherapy data for TransCon IL-2 β⁄γ reported earlier this year, further strengthening our confidence in its best-in-class potential," said Stina Singel, M.D., Ph.D., Executive Vice President, Head of Clinical Development, Oncology at Ascendis Pharma. "In addition to previously reported monotherapy clinical activity, we are particularly encouraged to see anti-tumor responses in two of the three patients with SCLC in the combination portion of the trial who had previously progressed on checkpoint inhibitors. In the Phase 2 portion of IL-Believe, we continue enrolling into indication-specific cohorts and look forward to sharing preliminary data from these cohorts in the second half of 2024."

TransCon IL-2 β⁄γ is an investigational long-acting prodrug with sustained release of an IL-2Rβ⁄γ-selective analog (IL-2 β⁄γ) designed to address the known limitations of interleukin-2 (IL-2) cancer immunotherapy through prolonged activation of IL-2Rβ⁄γ with low Cmax. IL-Believe is investigating the safety and tolerability of TransCon IL-2 β⁄γ alone or in combination with the check-point inhibitor pembrolizumab and/or chemotherapy or TransCon TLR7/8 Agonist in participants with locally advanced or metastatic solid tumors. RP2D for IL-Believe is 120 µg/kg of TransCon IL-2 β⁄γ administered intravenously every three weeks in both the monotherapy and combination therapy arms.

Additional details and highlights from the ESMO (Free ESMO Whitepaper) poster are available on the Investor & News section of the Ascendis Pharma website at View Source

On October 26, 2023 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported tis financial results for the third quarter ending September 30, 2023 (Press release, Alligator Bioscience, OCT 26, 2023, View Source [SID1234636361]).

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"During another busy and productive quarter for Alligator we achieved one more important milestone in the clinical and commercial development of our lead asset with the addition of orphan designation for mitazalimab in pancreatic cancer by the European regulator. This follows our receipt of orphan drug designation in the US from the FDA and means we have now secured greater commercial protection for mitazalimab upon approval in our two key markets, the US and the EU. Over the summer, we showcased mitazalimab’s scientific achievements at several prestigious industry conferences, through which we continue to raise mitazalimab’s profile among the world’s top oncologists, investors and potential partners. On the partnership front, we reported the achievement of Technical Feasibility in one of our collaboration programs with Orion, which triggered a milestone payment to us. Our work with Orion is a great example of the benefits these strategic collaborations can deliver for our company and our shareholders. We have much to look forward to in the coming months, in particular the next major inflection point in our OPTIMIZE-1 study, which is due to report its top-line Phase 2 data at the start of next year and which will mark the start of the next stage of mitazalimab’s route to market."
Søren Bregenholt, CEO of Alligator Bioscience

BUSINESS UPDATE
Mitazalimab

On September 28-29, Alligator presented twice at the 2023 AACR (Free AACR Whitepaper) (American Association for Cancer Research) Special Conference on Pancreatic Cancer held in Boston. The first presentation was an oral presentation entitled "CD40 agonist mitazalimab in combination with mFOLFIRINOX in patients with metastatic pancreatic ductal adenocarcinoma (mPDAC): Interim efficacy results of the OPTIMIZE phase 1b/2 study". The second was a poster presentation entitled "Interim pharmacodynamic analyses of mitazalimab in combination with FOLFIRINOX in first-line metastatic pancreatic ductal adenocarcinoma (mPDAC) identify CD4 effector T cells as a correlate of treatment outcomes".
On September 21, Alligator presented a poster at the 2023 International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper) (CICON) held in Milan, entitled "Efficacy and pharmacodynamic biomarkers of mitazalimab in combination with chemotherapy in preclinical mouse models".
On August 21, Alligator announced that the European Medicines Agency had granted Orphan Designation to mitazalimab for the treatment of pancreatic cancer. The designation confers significant financial and regulatory benefits, including 10 years of marketing approval in the EU after product approval.
On July 19, Alligator presented an oral presentation at the 3rd Annual Tumor Myeloid-Directed Therapies Summit held in Boston entitled "Targeting CD40 on Myeloid Cells to Reverse the Suppressive Tumor Microenvironment & Enhance T Cell Priming".
Exercise of Warrants

On September 1, Alligator announced it would receive approximately SEK 13.8 million through the exercise of warrants of series TO 6, which represents an utilization rate of approximately 68 percent of the warrants attached to shares acquired through the preferential rights issue that took place in April 2023.
Milestone achieved in collaboration with Orion

On July 31, Alligator announced that Technical Feasibility had been achieved in the second program of the Company’s research collaboration and license agreement with Orion, triggering a new milestone payment to Alligator.
Significant events after the end of the period:

Mitazalimab

On October 11, the European Patent Office granted Alligator a new patent covering mitazalimab’s composition of matter until 2038, including a potential supplementary term. The patent expands protection for mitazalimab in Europe and brings the total number of patents in the mitazalimab intellectual property portfolio to 48 granted patents and 25 pending patents covering multiple territories, including Europe, North America, Asia and more.
ATOR-1017

On October 5, Alligator announced the publication of a peer-reviewed article entitled "ATOR-1017, an Fc-gamma receptor conditional 4-1BB agonist designed for optimal safety and efficacy, activates exhausted T cells in combination with anti-PD-1" in the journal Cancer Immunology, Immunotherapy.
FINANCIAL SUMMARY FOR Q3 2023 and YEAR-TO-DATE 2023
The financial summaries for the quarterly periods ending September 30th, 2023 and September 30th, 2022 are presented below.

All amounts in MSEK,
unless specified July – September 2023 July – September 2022
Net Sales 19.4 5.1
Operating profit/loss -52.7 -51.2
Profit/loss for the period -52.5 -51.4
Earnings per share (SEK)
before and after dilution -0.08 -0.23
Cash Flow -86.5 -45.6
Cash & Cash Equivalents including short-term financial assets 123.9 147.4

The financial summaries for the year-to-date periods ending September 30th, 2023 and September 30th, 2022 are presented below.

All amounts in MSEK,
unless specified January – September 2023 January – September 2022
Net Sales 46.4 15.6
Operating profit/loss -178.6 -140.1
Profit/loss for the period -178.8 -140.2
Earnings per share (SEK)
before and after dilution -0.45 -0.64
Cash Flow -23.1 -131.0
Cash & Cash Equivalents 123.9 147.4
The full report is attached as a PDF, and is also available on the company’s website via this link.

Alligator will host a conference call today at 12:30 p.m. CEST/ 6:30 a.m. ET for investors, analysts and media, where CEO Søren Bregenholt and CFO Marie Svensson will present and comment on the Q3 interim report, which will be followed by a Q&A session. The call will be held in English.

On October 26, 2023, 2seventy bio, Inc. (the "Company") reported that ABECMA (idecabtagene vicleucel, or ide-cel) generated approximately $69 million in U.S. revenue during the third quarter of 2023 (Press release, 2seventy bio, OCT 26, 2023, View Source [SID1234636360]). The Company and Bristol Myers Squibb ("BMS") share equally in all profits and losses related to development, manufacture and commercialization of ABECMA in the U.S. This announcement is based on information provided to the Company by BMS and is unaudited and does not present all information necessary for an understanding of the Company’s financial condition as of September 30, 2023 and its results of operations for the quarter then ended. The Company expects to announce its full results for the quarter ended September 30, 2023 on or before November 14, 2023. The Company also believes that the competitive dynamics impacting ABECMA will continue into the fourth quarter of 2023.

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