On January 09, 2023 bluebird bio, Inc. (Nasdaq: BLUE) (the Company) reported updates to be presented at the 41st Annual J.P. Morgan Healthcare conference including commercial launch progress, 2023 program milestones and financial outlook. CEO Andrew Obenshain is scheduled to speak on Thursday, January 12 at 7:30 a.m. PT (Press release, bluebird bio, JAN 9, 2023, View Source [SID1234626037]).

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"In 2022, bluebird bio emerged as a commercial stage gene therapy company, with a validated lentiviral vector platform built on more than a decade of scientific leadership," said Andrew Obenshain, chief executive officer, bluebird bio. "In a dynamic and challenging environment, bluebird has hit all its operational milestones, including nearly simultaneous launches of two first-in-class gene therapies, ZYNTEGLO and SKYSONA. We have strengthened our financial position and generated considerable momentum as we prepare for the opportunity ahead: bringing lovo-cel gene therapy to individuals living with sickle cell disease and their families."

Highlights from the Company’s Update Include:

Significant Momentum and Early Signs of Uptake for ZYNTEGLO (betibeglogene autotemcel) Commercial Launch

Apheresis for first commercial ZYNTEGLO patient completed; Company entered 2023 with multiple patient cell collections scheduled. First revenue anticipated for Q1 2023 as previously guided.
Early indicators continue to demonstrate significant patient demand for ZYNTEGLO. Approximately 40 patients have initiated benefits verification, with a significant proportion progressing to prior authorization approval across both Medicaid and commercial payer segments and zero ultimate denials to date.
bluebird’s qualified treatment center (QTC) network is scaling as planned, with 10 activated centers to date. More than 15 additional QTCs are in the on-boarding or master service agreement negotiation stage, aligned with the Company’s expectation to scale to 40-50 centers by the end of 2023.
Outcomes-based agreements are in place with 3 of the largest pharmacy benefit managers in the US, representing dozens of downstream national and regional plans. To date, approximately 190 million US lives are covered by a favorable coverage policy for ZYNTEGLO.
During ZYNTEGLO’s 2023 launch year, the Company expects to assess key metrics over time as launch progresses such as number of patient starts and percent of lives covered and does not expect to provide ZYNTEGLO revenue projections this year.
SKYSONA (elivaldogene autotemcel) Commercial Launch on Track

Cell collection for first patient to be treated commercially with SKYSONA scheduled for January 2023.
Clear path to lovotibeglogene autotemcel (lovo-cel) BLA submission in Q1 2023

bluebird completed vector and drug product analytical comparability studies in the 4th quarter of 2022, as previously guided. With this progress, bluebird remains on track to submit its biologics license application (BLA) to the U.S. Food and Drug Administration this quarter.
2023 Financial Outlook

The Company’s preliminary unaudited cash and cash equivalents and marketable securities balance was approximately $182 million, excluding restricted cash of approximately $45 million, as of December 31, 2022. As bluebird bio launches two first-in-class gene therapies and readies its third investigational gene therapy for SCD for the commercial setting, full-year 2023 cash burn is expected to be in the range of $270-$300 million. bluebird expects its cash, cash equivalents, restricted cash and marketable securities, including the proceeds from the sale of its second priority review voucher (PRV) for $95 million, will be sufficient to meet bluebird’s planned operating expenses and capital expenditure requirements into the first quarter of 2024.

Presentation at the 2023 J.P. Morgan Healthcare Conference

Andrew Obenshain, chief executive officer, bluebird bio, will present a corporate update on Thursday, January 12, at 7:30 a.m. PT. 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 9, 2023 Century therapeutics presented its corporate overview (Presentation, Century Therapeutics, JAN 9, 2023, View Source [SID1234626044]).

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On January 09, 2023 Biomea Fusion, Inc. (Nasdaq: BMEA), a clinical-stage biopharmaceutical company dedicated to discovering and developing novel covalent small molecules to treat and improve the lives of patients with genetically defined cancers and metabolic diseases, reported that Thomas Butler, Biomea Fusion’s Chief Executive Officer and Chairman of the Board, will present recent progress and 2023 corporate milestones at the 41st Annual J.P. Morgan Healthcare Conference on Wednesday, January 11, 2023 from 11:15 – 11:55 am ET, and that Biomea management will hold 1×1 meetings during the conference January 9 – 11 (Press release, Biomea Fusion, JAN 9, 2023, View Source [SID1234626036]).

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A live webcast of the presentation will be available on the Investors & Media page of Biomea’s website at: View Source

"2022 was a year of strong execution and fundamental infrastructure build as we transitioned to a clinical-stage company and expanded our pipeline. We enter 2023 with three clinical trials studying BMF-219 across 8 cancer indications covering both blood cancers and solid tumors as well as in Type 2 diabetes, the 7th leading cause of death in the United States," stated Thomas Butler, Biomea Fusion’s Chief Executive Officer and Chairman of the Board. "We anticipate advancing BMF-500 into the clinic during the first half of 2023, subsequent to FDA clearance of an IND, which will increase our clinical pipeline to 4 clinical trials covering 10 indications. COVALENT-111, our Phase I/II study in Type 2 diabetes is now due to report initial safety and efficacy from the first two cohorts of the Phase II portion by the end of Q1."

Mr. Butler further commented, "we continue to activate sites and enroll patients in our Phase I/Ib (COVALENT-101) study of BMF-219 in patients with several liquid tumor types, and plan to report initial clinical data from this study in the first half of 2023. In addition, we anticipate initiating dosing imminently in our Phase I/Ib (COVALENT-102) study of BMF-219 in patients with KRAS-mutated solid tumors. In 2023, we will continue the patient-centric urgency and disciplined execution that are now well-established hallmarks of Team Fusion."

RECENT & ANTICIPATED MILESTONES

ONCOLOGY

COVALENT-101 (BMF-219)
Presented robust anti-tumor activity of covalent menin small molecule inhibitor, BMF-219, as a single agent and mechanistic evidence for novel inhibition of the menin protein in preclinical models of diffuse large B-cell lymphoma (DLBCL), multiple myeloma (MM), and chronic lymphocytic leukemia (CLL). BMF-219 displayed single agent potency, surpassing greater than 90% cell killing at clinically relevant exposures in DLBCL, MM and CLL cell lines and patient-derived samples.
BMF-219 is the first investigational menin inhibitor in clinical development to show potential as a therapeutic agent in hematologic malignancies outside of MLLr and NPM1 mutated acute myeloid leukemia/acute lymphoblastic leukemia (AML/ALL) patients, specifically in subsets of DLBCL, MM and CLL patients.
Biomea continued site activation and patient enrollment for the dosing of BMF-219 across four liquid tumor cohorts in the COVALENT-101 study, including patients with AML/ALL, DLBCL, MM and CLL.
Next Anticipated Milestone:
On track to present initial clinical data of AML/ALL patients (including those with MLL rearrangement and NPM1 mutation) dosed in the COVALENT-101 study in the first half of 2023.
COVALENT-102 (BMF-219)
Presented strong and highly specific pan-KRAS anti-cancer activity of BMF-219 as a single agent across KRAS G12C, G12D, G12V and G13D mutant cell lines including in non-small cell lung cancer (NSCLC), colorectal cancer (CRC) and the most prevalent type of pancreatic cancer, PDAC.
BMF-219 is the first investigational menin inhibitor in development to enter clinical trials for the treatment of solid tumors. A targeted pan-KRAS inhibitor could have the potential to treat 25-35% of NSCLC, 35-45% of CRC, and approximately 90% of PDAC patients.
Biomea received FDA clearance of its IND in the fourth quarter of 2022 and has since initiated a Phase I/Ib clinical trial of BMF-219 as a monotherapy in patients who have unresectable, locally advanced, or metastatic NSCLC, CRC or PDAC with an activating KRAS mutation.
Next Anticipated Milestone:
On track to dose first patient in COVALENT-102 study in January 2023.
COVALENT-103 (BMF-500)
Presented data showing multi-fold higher potency and increased cytotoxicity of Biomea’s covalent FLT3 small-molecule inhibitor BMF-500 compared to the commercially available reversible, non-covalent FLT3 inhibitor gilteritinib, and complete, sustained tumor regression in mouse models of FLT3-ITD AML with maintenance of effect after cessation of therapy.
Next Anticipated Milestone:
On track to file IND for BMF-500 in the first half of 2023 to initiate COVALENT-103 study of the covalent FLT3 inhibitor in patients with acute leukemia.
DIABETES

COVALENT-111 (BMF-219)
Presented preclinical data highlighting the ability of BMF-219 in a Type 2 diabetes rat model to restore normal HOMA-B, a measure of pancreatic beta cell function, following only 4-weeks of treatment and to significantly lower HbA1c compared to active control, liraglutide, -3.5% vs -1.7%, respectively.
BMF-219 is the first investigational menin inhibitor in development to enter clinical trials for the improvement of glycemic control and insulin sensitivity in Type 2 diabetes patients.
Biomea completed the healthy volunteer portion of the Phase I/II COVALENT-111 study of BMF-219 in Canada. BMF-219 was well tolerated with an encouraging pharmacokinetic and pharmacodynamic profile in healthy volunteers and with no safety signals detected.
Biomea received FDA clearance in December 2022 to expand the Phase II portion of COVALENT-111 to sites in the U.S. and in January 2023 announced dosing of the first U.S. patient with Type 2 diabetes. The company continues to enroll Type 2 diabetes patients in the Phase II portion of the study in Canada as well.
Next Anticipated Milestones:
On track to present initial clinical data from the first two cohorts of the Phase II portion of the study by the end of Q1 2023, and to present details of the healthy volunteer (Phase I) portion of the study at a scientific medical meeting in 2023.
FUSION SYSTEM DISCOVERY PLATFORM

Developed two covalently binding small molecules (BMF-219 and BMF-500), each within 18 months from target identification to IND candidate, leveraging the proprietary FUSION System Discovery Platform and showing excellent preclinical profiles.
Next Anticipated Milestone:
On track to announce a third development candidate from the FUSION platform in the first half of 2023.

On January 09, 2023 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) reported preliminary, unaudited ORLADEYO (berotralstat) net revenue for the fourth quarter and full year 2022 and provided guidance for full year 2023 ORLADEYO net revenue and expected peak ORLADEYO sales (Press release, BioCryst Pharmaceuticals, JAN 9, 2023, View Source [SID1234626035]).

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"In our second year on the market we more than doubled our first year ORLADEYO sales and are more than a quarter of the way towards $1 billion in peak sales. This continues to be an exceptional launch of an oral rare disease drug and we expect this success to continue creating real value for patients and for shareholders this year and for many years to come," said Jon Stonehouse, president and chief executive officer of BioCryst.

Preliminary, unaudited ORLADEYO net revenue in the fourth quarter of 2022 was $70.7 million (+53 percent y-o-y). Preliminary, unaudited ORLADEYO net revenue for full year 2022 was $251.6 million (+105 percent y-o-y).

Fourth Quarter and Full Year 2022 ORLADEYO Revenue Dynamics
"The number of patients on therapy at the end of 2022 was in-line with our expectations as patients continued to switch to ORLADEYO in the fourth quarter. Revenue in the quarter lagged our expectations slightly as we saw fewer paid product shipments based on seasonal factors and lower conversion from free to paid product than expected. We see continued growth in 2023 in the U.S. and around the world as we advance toward $1 billion in peak ORLADEYO sales," said Charlie Gayer, chief commercial officer of BioCryst.

2023 Financial Outlook
The company expects full year 2023 global net ORLADEYO revenue to be no less than $320 million. As in 2022, due to the seasonal impact of managed care reauthorizations in the first quarter of the year, the company expects revenue in the first quarter of 2023 to be similar to revenue in the fourth quarter of 2022.

Presentation Tomorrow at 41st Annual J.P. Morgan Healthcare Conference
On Tuesday, January 10, 2023 at 6:00 p.m. ET, the company will present at the 41st 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 9, 2023 Bristol-Myers Squibb presented its corporate presentation (Presentation, Bristol-Myers Squibb, JAN 9, 2023, View Source [SID1234626039]).

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