On January 8, 2024 Blueprint Medicines Corporation (Nasdaq: BPMC) reported its 2024 corporate strategy to deliver accelerated revenue growth, sustainable research and development, and a clear path to profitability (Press release, Blueprint Medicines, JAN 8, 2024, View Source [SID1234639060]).

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Kate Haviland, Chief Executive Officer of Blueprint Medicines, said:

"As we enter 2024, AYVAKIT’s early launch success in indolent systemic mastocytosis has grown our conviction that AYVAKIT has the potential to be a multi-billion-dollar therapy that will drive long-term growth into the next decade. We know that the first few quarters of a launch are critical in defining the trajectory of a new medicine, and we have built a strong foundation for success with AYVAKIT as we continue to drive growth in the U.S. and expand our launch in Europe this year.

Throughout 2023, we also made significant progress across our research and development pipeline enabling us to focus investments on our most promising programs. A core component of our growth strategy is to build on our leadership position in SM by expanding to other allergic-inflammatory diseases where mast cells play a core role in the biology, and we have integrated infrastructure that we can efficiently scale. Across our portfolio, we are investing in our most compelling opportunities to deliver innovative, life-changing medicines to patients, while maintaining a strong and sustainable financial profile."

Focused investment strategy in 2024 to drive long-term growth and maintain durable cash position

1. Prioritized programs for investment

Blueprint Medicines is building portfolio scale in therapeutic areas where there are significant medical needs in large patient populations and the company has a deep understanding of biological pathways, a potential to drive best-in-class efficacy and an ability to leverage expertise and infrastructure.

Mast cell diseases

· Extend the company’s leadership position in systemic mastocytosis with the ongoing launch of AYVAKIT in the U.S. and EU and continued development of the next-generation KIT D816V inhibitor elenestinib.
· Expand into larger patient populations with allergic-inflammatory diseases with BLU-808, an oral wild-type KIT inhibitor, including chronic urticaria and other diseases where mast cells are core to the biology.

Breast cancer and other solid tumors

· Advance combination development of BLU-222, a highly selective CDK2 inhibitor with best-in-class potential, in HR+/HER2- breast cancer based on positive previously reported monotherapy clinical data.
· Progress ongoing strategic partnership discussions to maximize the potential of BLU-222 as a backbone combination therapy in HR+/HER2- breast cancer and other CDK2-vulnerable cancers.
· Advance additional programs including BLU-956, a next-generation CDK2 inhibitor development candidate nominated in 2023, and targeted protein degrader research programs for CDK2 and an undisclosed target to support long-term lifecycle management.

2. De-prioritized programs

Blueprint Medicines is discontinuing investment in specific programs, based on the evolving external landscape, emerging clinical data and partnering considerations.

Lung cancer

· Discontinue further investment in the early clinical-stage therapies BLU-945 and BLU-451 for EGFR-mutant NSCLC and explore strategic options, including potential out-licensing, based on the evolving external landscape and emerging clinical data.
· In February 2023, Blueprint Medicines announced Roche’s decision to terminate the global collaboration agreement for GAVRETO (pralsetinib). Given Blueprint Medicines’ lack of global infrastructure in lung and thyroid cancer, the company has decided to discontinue global development and marketing of GAVRETO in territories excluding the U.S. and Greater China. The companies will continue working on transition and wind-down activities anticipated to begin in the first quarter of 2024; further information on product discontinuation timing to be provided in the near future.
· Blueprint Medicines has identified a potential alternate partner for GAVRETO in the U.S. and is continuing to work with the involved parties to define a scenario that enables continued availability of GAVRETO in the U.S.
· Blueprint Medicines expects the wind-down of the Roche collaboration for GAVRETO will result in significantly lower year-over-year operating expenses related to GAVRETO in 2024 and will not affect the $175 million upfront payment received under a 2022 financing agreement with Royalty Pharma.

As a result of continued strategic portfolio prioritization, Blueprint Medicines expects a year-over-year decline in operating expenses in 2024. The company plans to provide financial guidance for 2024, including anticipated AYVAKIT revenue, when it reports fourth quarter and full-year 2023 financial results in February 2024.

2024 Corporate Milestones

The company’s anticipated 2024 corporate milestones include:

Mast cell diseases

· Present long-term safety and efficacy data from the PIONEER trial of AYVAKIT in indolent SM (ISM) in the first half of 2024.
· Submit an investigational new drug (IND) application for BLU-808 in the second quarter of 2024.
· Initiate the registration-enabling Part 2 of the HARBOR trial of elenestinib in ISM in the second half of 2024.

Breast cancer and other solid tumors

· Continue ongoing strategic business development discussions.
· Present data for BLU-222 in combination with ribociclib and fulvestrant in patients with HR+/HER2- breast cancer in the first half of 2024.
· Provide update on BLU-222 registration plan in HR+/HER2- breast cancer in the second half of 2024.

J.P. Morgan Healthcare Conference Presentation Information

Kate Haviland, Chief Executive Officer of Blueprint Medicines, will present a company overview and 2024 outlook at the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8 at 10:30 a.m. PT / 1:30 p.m. ET. A live webcast of the presentation and Q&A breakout session will be available by visiting the "Events and Presentations" 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 January 8, 2024 bluebird bio, Inc. (Nasdaq: BLUE) (the Company; bluebird) reported updates to be presented at the 42nd Annual J.P. Morgan Healthcare conference including commercial launch progress, 2024 program milestones and financial outlook (Press release, bluebird bio, JAN 8, 2024, View Source [SID1234639059]). Andrew Obenshain, chief executive officer, is scheduled to present Tuesday, January 9 at 10:30 a.m. PT/1:30 p.m. ET.

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"In 2023 bluebird cemented our status as a gene therapy leader, securing our third FDA approval in under two years and establishing a commercial footprint that will support growth in the coming year and beyond," said Andrew Obenshain, chief executive officer, bluebird bio. "In 2024 we are leveraging our validated commercial strategy to accelerate the launch of LYFGENIA and drive continued strong uptake for ZYNTEGLO. We are extremely pleased with the indicators of demand from both patients and providers in the weeks following FDA approval of LYFGENIA and are focused on using our real-world experience to support timely and equitable access and deliver a positive treatment experience."

Highlights from the Company’s update include:

Synergies with ZYNTEGLO commercial network are accelerating LYFGENIA commercial launch in 2024

Established Qualified Treatment Center (QTC) network in place, with 48 centers activated for ZYNTEGLO for beta-thalassemiai as of January 5, 2024.
35 of 48 centers ready to receive referrals for LYFGENIA for sickle cell disease as of January 5, 2024.
All centers are anticipated to be ready to treat with both ZYNTEGLO and LYFGENIA by end of Q1 2024.
Validated access and reimbursement strategy is driving favorable coverage landscape for LYFGENIA and ZYNTEGLO

bluebird has signed outcomes-based agreements for LYFGENIA with national payer organizations representing dozens of downstream plans and covering approximately 200 million U.S. lives.
Advanced discussions are ongoing with additional commercial payers and with more than 15 Medicaid agencies representing 80% of individuals with sickle cell disease in the U.S.
bluebird has designed outcomes-based agreements that are unique to LYFGENIA and offer payers meaningful risk sharing tied to VOE-related hospitalizations, with patients followed for three years. Informed by input from state Medicaid agencies, the Company has designed an offering specifically for Medicaid that addresses the need for predictability and operational ease that is essential for states grappling with resource constraints.
Outcomes-based agreements are in place for ZYNTEGLO with both commercial and Medicaid payers, and more than 200 million U.S. lives are covered through contracts or favorable coverage policies. Timely access to ZYNTEGLO for people living with beta-thalassemia continues, with zero ultimate denials across both Medicaid and commercial payers.
bluebird also continues to engage with Center for Medicare and Medicaid Innovation (CMMI) on its Cell and Gene Therapy Access Model, which is anticipated to be implemented in 2025.
Strong commercial momentum is poised to translate into sustained revenue recognition

26 patient starts were completed in 2023 across bluebird’s commercial portfolio, including 20 for ZYNTEGLO and 6 for SKYSONA. 2023 patient starts will drive revenue recognition in 2024 as patients complete the gene therapy treatment journey.
bluebird anticipates the first patient start for LYFGENIA in Q1 2024.
The Company anticipates 85 to 105 patient starts combined across all three of its FDA approved therapies (LYFGENIA, ZYNTEGLO, SKYSONA) in 2024.
Liquidity and Cash Runway Update

The Company’s preliminary unaudited cash and cash equivalents and marketable securities balance was approximately $275 million, including restricted cash of approximately $53 million, as of December 31, 2023. bluebird expects its cash, cash equivalents, and marketable securities, excluding restricted cash, will be sufficient to meet bluebird’s planned operating expenses and capital expenditure requirements into the first quarter of 2025 as bluebird progresses its launch of LYFGENIA gene therapy for sickle cell disease and continues to scale its launches of ZYNTEGLO and SKYSONA for beta-thalassemia and cerebral adrenoleukodystrophy, respectively.

The Company has taken additional steps to strengthen its financial position by entering into an accounts receivable factoring agreement which will accelerate cash collection related to patient starts across its portfolio of approved therapies.

Presentation at the 2024 J.P. Morgan Healthcare Conference

Andrew Obenshain, chief executive officer, bluebird bio, will present a corporate update on Tuesday, January 9 at 10:30 a.m. PT/1:30 p.m. ET. A live webcast of the presentation will be available on the "Events & Presentations" page within the Investors & Media section of the bluebird bio website at View Source A replay of the webcast will be available on the bluebird bio website for 30 days following the event.

On January 8, 2024 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) reported preliminary, unaudited ORLADEYO (berotralstat) net revenue for the fourth quarter and full year 2023 (Press release, BioCryst Pharmaceuticals, JAN 8, 2024, View Source [SID1234639058]). The company also provided guidance for full year 2024 ORLADEYO net revenue, full year 2024 operating expenses, expected peak ORLADEYO sales and an accelerated path to profitability.

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"After three years on the market, ORLADEYO continues on a steady growth trajectory to achieve $1 billion at peak. This commercial success, alongside our proven discovery platform that is producing additional first-in-class or best-in-class molecules, uniquely positions BioCryst to achieve financial independence from the capital markets and accelerate our path to profitability," said Jon Stonehouse, president and chief executive officer of BioCryst.

The company also announced that, if its ongoing proof-of-concept trial produces best-in-class data, it plans to out-license late-stage development and commercialization of BCX10013, its potential once-daily, oral Factor D inhibitor, to a partner that can drive the speed and breadth of investment required to accelerate BCX10013 for patients across multiple complement-mediated diseases and maximize the commercial potential of the program. As a result, the company has reduced the size of its R&D organization and accelerated its timeline to profitability.

Preliminary Fourth Quarter and Full Year 2023 ORLADEYO Revenue and 2024 ORLADEYO Outlook
Preliminary, unaudited ORLADEYO net revenue in the fourth quarter of 2023 was $89.9 million (+27 percent y-o-y). Preliminary, unaudited ORLADEYO net revenue for full year 2023 was $325 million (+29 percent y-o-y).

The company expects full year 2024 global net ORLADEYO revenue to be between $380 million and $400 million. The general pattern of revenue throughout 2024 is expected to be similar to past years, with the seasonal impact of prescription reauthorizations and the potential impact of the Inflation Reduction Act in the first quarter driving a quarter-over-quarter revenue decline in the first quarter, followed by a strong return to growth in the second quarter.

"ORLADEYO growth remained strong in the fourth quarter of 2023 as hereditary angioedema patients gain the excellent attack control they expect. Comparing U.S. patient trends year over year (y-o-y), we had more new patient prescriptions and a lower average rate of monthly discontinuations in 2023 compared to 2022. U.S. performance combined with continued global expansion keep ORLADEYO on track for $1 billion in peak sales," said Charlie Gayer, chief commercial officer of BioCryst.

Operating Expense and Profitability Outlook
The company expects full year 2024 operating expenses to be between $365 million and $375 million, flat to expected full year 2023 operating expenses. The company now expects that R&D expenses in 2024 will be reduced by $20 million versus 2023. This represents a $45 million to $55 million reduction from the 2024 R&D expense guidance it provided at its R&D Day in November 2023, and reflects both the R&D restructuring and the postponement of previously planned capital expenditures at its Discovery Center in Alabama. SG&A expenses are expected to increase by $20 million in 2024, primarily to support the continued U.S. and global growth of ORLADEYO to $1 billion in peak sales.

This operating expense outlook does not reflect non-cash stock compensation expense, or one-time expenses related to the reduction of 59 jobs (10 percent of total organization) in the first quarter of 2024.

Based on the company’s disciplined approach to capital allocation, and the revenue expected from ORLADEYO, the company expects to achieve a full-year operating profit in 2024 (not including non-cash stock compensation), be approaching quarterly positive earnings per share (EPS) and positive cash flow in the second half of 2025 (not including non-cash stock compensation), and be profitable on an EPS basis, with positive cash flow, for full year 2026. The company expects it can achieve these financial milestones without raising additional funds and does not intend to draw the additional $150 million of debt available to it from Pharmakon.

Presentation Tuesday at 42nd Annual J.P. Morgan Healthcare Conference
On Tuesday, January 9, 2024 at 6:00 p.m. ET, the company will present at the 42nd Annual J.P. Morgan Healthcare Conference in San Francisco. Links to a live audio webcast and replay of the presentation may be accessed in the Investors section of BioCryst’s website at https://www.biocryst.com/.

On January 8, 2023 Beam Therapeutics presented its corporate presentation (Presentation, Beam Therapeutics, JAN 8, 2024, View Source [SID1234639057]).

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On January 8, 2024 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, reported that it has entered into a definitive agreement for the issuance and sale of pre-funded warrants to purchase 27,272,727 shares of its common stock at a purchase price of $0.55 per pre-funded warrant share in a registered direct offering to entities affiliated with an existing institutional investor (Press release, Atara Biotherapeutics, JAN 8, 2024, View Source [SID1234639056]). The pre-funded warrants will have an exercise price of $0.0001 per share, and will be immediately exercisable upon issuance. The offering is expected to close on or about January 10, 2024, subject to the satisfaction of customary closing conditions.

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The gross proceeds to Atara from the offering are expected to be $15 million, before deducting estimated offering expenses payable by Atara. Atara currently intends to use the net proceeds from the offering for working capital and general corporate purposes.

The securities described above are being offered by Atara pursuant to a shelf registration statement on Form S-3 (No. 333-275256), including a base prospectus, that was previously filed by Atara with the U.S. Securities and Exchange Commission (the "SEC") and was declared effective on November 13, 2023. A prospectus supplement containing additional information relating to the offering will be filed with the SEC and will be available on the SEC’s website located at View Source

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.