Can-Fite to Harness Artificial Intelligence to Develop Novel Anti-Cancer Drugs

On October 26, 2023 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address oncology and inflammatory diseases, reported that it entered into an agreement with Collaborations Pharmaceuticals, Inc. (CPI) to develop anti-cancer drugs utilizing artificial intelligence (AI) and machine learning (ML) techniques (Press release, Can-Fite BioPharma, OCT 26, 2023, View Source [SID1234636366]). This project will aim to develop a next-generation A3 adenosine receptor drug agonists that significantly reduce the development time and cost of bringing such drugs to market.

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CPI will utilize, apply and use AI and ML tools, including their MegaSyn generative AI method, to design new molecules with high affinity and selectivity to the A3AR Can-Fite target. CPI will also perform the chemical synthesis of the newly designed molecules with the ultimate goal of developing novel and robust anti-cancer drug candidates. Can-Fite will perform the testing of the biological anti-cancer effects and validate the molecular mechanism of the novel, chemically synthesized drug candidates.

"Our vision is to deliver in silico small molecule drug candidates in a better and faster way to patients via a collaboration with Collaborations Pharmaceuticals. Our accumulated experience of bringing anti-cancer drugs which target the A3AR from lab to patients will be implemented into this AI drug development project," stated Prof. Pnina Fishman, Executive Chairman and CSO at Can-Fite.

"We are delighted that Can-Fite chose our team of experts for this AI-led drug discovery collaboration and look forward to demonstrating what our technology can do," said Sean Ekins, PhD, DSc., CEO and Founder of Collaborations Pharmaceuticals, Inc. We also look forward to complementing their outstanding scientific approach with our integrated technology platform and ability to generate novel and selective molecules."

Bristol Myers Squibb Reports Third Quarter Financial Results for 2023

On October 26, 2023 Bristol Myers Squibb (NYSE:BMY) reported results for the third quarter of 2023, which reflect significant pipeline progress and advances in the company’s portfolio renewal strategy (Press release, Bristol-Myers Squibb, OCT 26, 2023, View Source [SID1234636365]).

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"My excitement for the company’s future is centered on the diversification of our business, the breadth of our new product portfolio and the strength of our pipeline," said Giovanni Caforio, M.D., board chair and chief executive officer, Bristol Myers Squibb. "I am proud of what we have achieved together and look forward to what the dedicated people of our company will continue to accomplish for patients."

Christopher Boerner, Ph.D., executive vice president and chief operating officer and CEO-elect, Bristol Myers Squibb, added the following:

"I want to thank Giovanni for his tremendous leadership and commitment not only to patients, but also to strengthening our company. During the third quarter, we continued to grow our in-line and new product portfolio. We remain focused on accelerating commercial performance, advancing our pipeline and harnessing our financial flexibility to pursue business development opportunities that benefit patients."

Third Quarter

$ amounts in millions, except per share amounts

2023

2022

Change

Change
Excl. F/X**

Total Revenues

$10,966

$11,218

(2)%

(3)%

Earnings per share – GAAP*

0.93

0.75

24%

N/A

Earnings per share – Non-GAAP*

2.00

1.99

1%

N/A

* GAAP and Non-GAAP earnings per share include the net impact of Acquired IPRD charges and licensing income, which decreased by $0.03 per share
in the third quarter of 2023 compared to an increase of $0.02 per share in the third quarter of 2022.

** See "Use of Non-GAAP Financial Information".
THIRD QUARTER FINANCIAL RESULTS
All comparisons are made versus the same period in 2022 unless otherwise stated.

Bristol Myers Squibb posted third quarter revenues of $11.0 billion, a decrease of 2%, or 3% when adjusted for foreign exchange, due to lower sales of Revlimid, partially offset by our new product portfolio and in-line products.
U.S. revenues decreased 4% to $7.6 billion in the quarter primarily due to lower sales of Revlimid resulting from generic erosion and, as previously disclosed, an increase in the number of patients receiving free drug product for Revlimid, and to a lesser extent Pomalyst, from the Bristol Myers Squibb Patient Assistance Foundation, a separate and independent 501(c)(3) entity to which BMS donates products. This was partially offset by our new product portfolio and in-line products.
International revenues increased 2% to $3.3 billion in the quarter. When adjusted for foreign exchange impacts, international revenues increased 1%, primarily due to Opdivo and our new product portfolio, partially offset by lower average net selling prices.
On a GAAP basis, gross margin decreased from 79.0% to 77.1% and on a Non-GAAP basis, decreased from 79.8% to 77.3% primarily due to product mix and lower hedge settlement gains.
On a GAAP basis, marketing, selling and administrative expenses increased 4% to $2.0 billion in the quarter primarily due to higher advertising and promotion costs to support new product launches, partially offset by the cash settlement of Turning Point Therapeutics, Inc. ("Turning Point") unvested stock awards in 2022. On a Non-GAAP basis, marketing, selling and administrative expenses increased 4% to $1.9 billion in the quarter, primarily due to higher advertising and promotion costs to support new product launches.
On a GAAP basis, research and development expenses decreased 7% to $2.2 billion in the quarter due to lower clinical grants and supplies and cash settlement of Turning Point unvested stock awards in 2022. On a Non-GAAP basis, research and development expenses decreased 4% to $2.2 billion in the quarter primarily due to lower clinical grants and supplies.
On a GAAP and Non-GAAP basis, Acquired IPRD increased to $80 million in the quarter from $30 million in the same period a year ago. On a GAAP and Non-GAAP basis, licensing income was $12 million in the quarter compared to $73 million in the same period a year ago.
On a GAAP basis, amortization of acquired intangible assets decreased 7% to $2.3 billion in the quarter, primarily due to the Abraxane marketed product right being fully amortized in the fourth quarter of 2022.
On a GAAP basis, effective tax rate changed from 27.2% to 9.5% in the quarter and on a Non-GAAP basis the effective tax rate changed from 16.9% to 11.6%, primarily due to changes in the IRS income tax guidance regarding deductibility of certain non-U.S. research and development expenses.
The company reported on a GAAP basis net earnings attributable to Bristol Myers Squibb of $1.9 billion, or $0.93 per share, in the third quarter compared to $1.6 billion, or $0.75 per share, for the same period a year ago.
The company reported on a Non-GAAP basis net earnings attributable to Bristol Myers Squibb of $4.1 billion, or $2.00 per share, in the third quarter compared to $4.3 billion, or $1.99 per share, for the same period a year ago.
The EPS results in the third quarter of 2023 also include the impact of lower weighted-average common shares outstanding.
THIRD QUARTER PRODUCT REVENUE HIGHLIGHTS

($ amounts in millions)

Quarter Ended
September 30, 2023

% Change from Quarter
Ended September 30,
2022

% Change from
Quarter Ended
September 30,
2022 Ex-F/X**

U.S.(c)

Int’l

WW(d)

U.S.(c)

Int’l

WW(d)

Int’l

WW(d)

In-Line Products

Eliquis

$

1,799

$

906

$

2,705

4%

(2)%

2%

(6)%

—%

Opdivo

1,352

923

2,275

9%

15%

11%

15%

11%

Pomalyst/Imnovid

610

262

872

(5)%

7%

(2)%

4%

(2)%

Orencia

719

206

925

5%

2%

5%

1%

5%

Sprycel

406

111

517

1%

(30)%

(8)%

(29)%

(8)%

Yervoy

362

217

579

12%

8%

11%

6%

10%

Mature and other products (a)

191

285

476

—%

(12)%

(7)%

(11)%

(7)%

Total In-Line Products

5,439

2,910

8,349

4%

2%

3%

—%

3%

New Product Portfolio

Reblozyl

200

48

248

28%

41%

31%

35%

29%

Abecma

69

24

93

(8)%

(25)%

(13)%

(28)%

(14)%

Opdualag

162

4

166

93%

N/A

98%

N/A

98%

Zeposia

96

27

123

92%

42 %

78%

32%

75%

Breyanzi

77

15

92

*

67 %

*

67%

*

Onureg

30

13

43

25%

63 %

34%

50%

31%

Inrebic

19

10

29

12%

*

38%

*

33%

Camzyos

67

1

68

*

N/A

*

N/A

*

Sotyktu

62

4

66

*

N/A

*

N/A

*

Total New Product Portfolio

782

146

928

75%

38%

68%

31%

67%

Total In-Line and New Product Portfolio

6,221

3,056

9,277

10%

3%

8%

1%

7%

Recent LOE Products (b)

Revlimid

1,226

203

1,429

(44)%

(19)%

(41)%

(18)%

(41)%

Abraxane

181

79

260

57%

27%

47%

39%

51%

Total Recent LOE Products

1,407

282

1,689

(38)%

(10)%

(35)%

(7)%

(35)%

Total Revenues

$

7,628

$

3,338

$

10,966

(4)%

2%

(2)%

1%

(3)%

* In excess of +100%

** See "Use of Non-GAAP Financial Information".

(a) Includes over-the-counter (OTC) products, royalty revenue and mature products.

(b) Recent LOE Products includes products with significant expected decline in revenue from a prior reporting period as a result of a loss of exclusivity.

(c) Includes Puerto Rico.

(d) Worldwide (WW) includes International (Int’l) and U.S.

THIRD QUARTER PRODUCT REVENUE HIGHLIGHTS

In-Line Products
Revenues for in-line products in the third quarter were $8.3 billion compared to $8.1 billion in the prior year period. In-line products revenue was largely driven by:

Opdivo worldwide revenues increased 11% reported and when adjusted for foreign exchange. U.S. revenues increased 9% to $1.4 billion compared to the prior year period primarily due to higher demand. International revenues were $923 million compared to $804 million in the prior year period, representing an increase of 15% reported and when adjusted for foreign exchange, primarily due to higher demand as a result of launches for additional indications and core indications.
Eliquis worldwide revenues increased 2% compared to the prior year period. U.S. revenues were $1.8 billion compared to $1.7 billion in the prior year period, representing an increase of 4% primarily due to higher demand, partially offset by GTN adjustments in 2023. International revenues were $906 million compared to $926 million in the prior year period, representing a decrease of 2%, primarily driven by lower average net selling prices and generic erosion in Canada and the U.K.
New Product Portfolio

New product portfolio worldwide revenues increased to $928 million compared to $553 million in the prior year period, representing a growth of 68%, primarily driven by higher demand across the portfolio, including for Opdualag, Sotyktu, Camzyos, Reblozyl, Zeposia and Breyanzi.
Recent LOE Products

Revlimid worldwide revenues declined by 41% compared to the prior year period, primarily due to generic erosion and, as previously disclosed, an increase in the number of patients receiving free drug product from the Bristol Myers Squibb Patient Assistance Foundation, a separate and independent 501(c)(3) entity to which the company donates products.
PRODUCT AND PIPELINE UPDATE
Bristol Myers Squibb recently achieved significant regulatory and clinical milestones, including an important U.S. regulatory approval for Reblozyl in first-line, MDS-associated anemia. In addition, the company achieved strong results from a Phase 3 study evaluating subcutaneous nivolumab and received two key approvals—from the U.S. Food and Drug Administration (FDA) and the European Commission for Opdivo in stage IIB or IIC melanoma. The company also announced initial data from a Phase 3 trial evaluating a perioperative regimen including Opdivo in non-small cell lung cancer, as well as positive Phase 2 results evaluating its potential first-in-class LPA1 antagonist in progressive pulmonary fibrosis.

Cardiovascular

Category

Asset

Milestone

Clinical & Research

Camzyos (mavacamten)

Data from the EXPLORER-LTE cohort of the MAVA-LTE study showed sustained improvements in left ventricular outflow tract obstruction, symptoms and NT-proBNP levels in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM) based on a cumulative 120-week analysis.

In addition, long-term follow-up results from the Phase 3 VALOR-HCM LTE trial demonstrated the consistent impact of oral treatment for severely symptomatic obstructive HCM patients by showing that nearly nine out of 10 patients treated with Camzyos have continued in the trial without septal reduction therapy at either 40 or 56 weeks of treatment.

Eliquis (apixaban)

Results presented by the Bristol-Myers Squibb-Pfizer Alliance from ATHENS, a retrospective real-world data study, demonstrated that switching from Eliquis to rivaroxaban in non-valvular atrial fibrillation patients was associated with a higher risk of stroke/systemic embolism and major bleeding than those who continued Eliquis.

Oncology

Category

Asset

Milestone

Regulatory

Opdivo(nivolumab)

The FDA approved the supplemental Biologics License Application for Opdivo as a monotherapy in the adjuvant setting for the treatment of eligible patients with completely resected stage IIB or IIC melanoma. The approval is based on results from the CheckMate -76K trial.

The European Commission approvedOpdivo as a monotherapy for the adjuvant treatment of adults and adolescents 12 years of age and older with stage IIB or IIC melanoma who have undergone complete resection. The approval is based on results from the Phase 3 CheckMate -76K trial.

Clinical & Research

Opdivo

First results from the Phase 3 CheckMate -901 trial demonstrated that Opdivo in combination with cisplatin-based chemotherapy followed by Opdivo monotherapy demonstrated statistically significant and clinically meaningful improvements in the primary efficacy endpoints of overall survival and progression-free survival compared to standard-of-care cisplatin-based chemotherapy as a first-line treatment for patients with unresectable or metastatic urothelial carcinoma who are eligible for cisplatin-based chemotherapy.

First data disclosure from the Phase 3 CheckMate -77T trial evaluating the perioperative regimen of neoadjuvant Opdivo with chemotherapy followed by surgery and adjuvant Opdivo in patients with resectable stage IIA to IIIB non-small cell lung cancer (NSCLC) demonstrated a statistically significant and clinically meaningful improvement in the primary efficacy endpoint of event-free survival compared to neoadjuvant chemotherapy and placebo followed by surgery and adjuvant placebo.

Three-year follow-up results from exploratory analyses of the Phase 3 CheckMate -816 trial demonstrated sustained event-free survival (EFS) and promising overall survival trends with three cycles of Opdivo in combination with platinum-based chemotherapy for the neoadjuvant treatment of patients with resectable NSCLC, regardless of PD-L1 expression levels. Neoadjuvant Opdivo with chemotherapy also showed improvements in pathologic complete response (pCR) and major pathologic response (MPR) over chemotherapy alone in PD-L1 ≥1% and <1% patient populations.

Part B of the Phase 3 CheckMate -914 trial, evaluating Opdivo as an adjuvant treatment for patients with localized renal cell carcinoma who have undergone full or partial removal of the kidney and who are at a moderate or high risk of relapse, did not meet the primary endpoint of disease-free survival as assessed by Blinded Independent Central Review. The safety profile was consistent with previously reported studies of other Opdivo and Opdivo-based combinations in solid tumors.

Subcutaneous nivolumab

Results from the Phase 3 CheckMate -67T trial evaluating subcutaneous nivolumab in advanced or metastatic clear cell renal cell carcinoma demonstrated noninferior pharmacokinetics (co-primary endpoints) and objective response rate (key secondary endpoint) when compared to intravenous Opdivo. The company looks forward to discussing next steps for subcutaneous nivolumab with health authorities across multiple indications.

Opdivo+Yervoy

Six-year follow-up results from Part 1 of the Phase 3 CheckMate -227 trial demonstrated long-term, durable survival benefits of Opdivo plus Yervoy compared to chemotherapy in the first-line treatment of patients with metastatic NSCLC, regardless of PD-L1 expression levels.

repotrectinib

Updated results from the registrational Phase 1/2 TRIDENT-1 study demonstrated that repotrectinib, a next-generation ROS1/TRK tyrosine kinase inhibitor, continued to show high response rates and durable responses in patients with ROS1-positive locally advanced or metastatic NSCLC. The FDA granted Priority Review of the New Drug Application for repotrectinib and assigned a Prescription Drug User Fee Act goal date of November 27, 2023.

Hematology

Category

Asset

Milestone

Regulatory

Reblozyl(luspatercept-aamt)

The FDA approvedReblozyl for the treatment of anemia without previous erythropoiesis stimulating agent use (ESA-naïve) in adult patients with very low- to intermediate-risk myelodysplastic syndromes who may require regular red blood cell transfusions. The approval is based on interim results from the pivotal Phase 3 COMMANDS trial, expanding Reblozyl indication to the first-line setting regardless of ring sideroblast status and enabling treatment across a broader array of patients.

Immunology

Category

Asset

Milestone

Regulatory

LPA1 antagonist BMS-986278

The FDA granted BMS-986278, a potential first-in-class oral, lysophosphatidic acid receptor 1 (LPA1)antagonist, Breakthrough Therapy Designation for the treatment of progressive pulmonary fibrosis (PPF).

Clinical & Research

LPA1 antagonist

Results from the Phase 2 study evaluating BMS-986278 in patients with PPF demonstrated that twice-daily administration of 60mg of BMS-986278 over 26 weeks reduced the rate of decline in percent predicted forced vital capacity by 69% compared to placebo.

Sotyktu (deucravacitinib)

Results from the POETYK-PSO long-term extension trial of Sotyktu treatment in adult patients with moderate-to-severe plaque psoriasis demonstrated that clinical response was maintained at 73.2% for Psoriasis Area and Severity Index 75 with 3 years of continuous Sotyktu treatment. Sotyktu demonstrated a consistent safety profile with no increases in adverse or serious adverse events and no new safety signals.

Zeposia (ozanimod)

First interim readout from the Phase 3b ENLIGHTEN trial demonstrated that almost half of patients with early relapsing multiple sclerosis (RMS) have clinically meaningful improvement in cognitive functioning compared to baseline after one year of Zeposia treatment.

In addition, late-breaking data from the DAYBREAK and RADIANCE trials demonstrated that, after eight years of follow-up, 76% of patients treated with Zeposia for RMS were free of six-month confirmed disability progression. Findings also demonstrated treatment with Zeposia resulted in low rates of progression-independent relapse activity and relapse-associated worsening, key drivers of disease progression and permanent disability in multiple sclerosis.

Research and Development (R&D) Update
In September, Bristol Myers Squibb hosted an R&D Day highlighting its advancing pipeline and differentiated research platforms to support long-term sustainable growth. During the presentation, members of the company’s leadership team discussed:

The strengthening of Bristol Myers Squibb’s scientific leadership and the advancement of a promising pipeline;
An expectation of doubling registrational assets from six to 12 over the next 18 months;
More than 25 indication expansion opportunities on the horizon and nine high-potential early assets that are expected to advance in the company’s pipeline;
Differentiated research platforms that support long-term growth, including Cell Therapy and Targeted Protein Degradation;
Increased depth across the company’s oncology, hematology, immunology and cardiovascular therapeutic areas and a growing presence in neuroscience; and
Efforts to further increase and sustain the productivity of its R&D engine and bring treatments to patients faster.
Business Development
In October 2023, the company announced it had entered into a definitive merger agreement to acquire Mirati Therapeutics, Inc. ("Mirati"), a commercial-stage targeted oncology company. The pending acquisition, when complete, is expected to strengthen and diversify Bristol Myers Squibb’s oncology franchise, add KRAZATI (adagrasib), a best-in-class KRASG12C inhibitor currently approved in lung cancer, to its commercial oncology portfolio, and add MRTX1719, a potential first-in-class MTA-cooperative PRMT5 inhibitor in Phase 1 development. Bristol Myers Squibb also gains access to several promising clinical and pre-clinical stage assets, including additional KRAS inhibitors and enabling programs.

Capital Allocation
The company maintains a balanced approach to capital allocation focused on prioritizing investment for growth through business development, maintaining a strong balance sheet, growing the dividend and opportunistic share repurchases. Dividend decisions are subject to approval by the Board of Directors.

In August, the company announced that it had entered into accelerated share repurchase (ASR) agreements to repurchase, in aggregate, $4 billion of Bristol Myers Squibb common stock. The company anticipates that final settlement of these transactions will occur during the fourth quarter of 2023.
Environmental, Social & Governance (ESG)
As a leading biopharmaceutical company, we understand our responsibility extends well beyond the discovery, development and delivery of innovative medicines. Our evolving ESG strategy builds on a legacy of comprehensive and global sustainability efforts that seek to drive business value and positively impact patients, employees, communities and the planet.

In August 2023, the company published its latest ESG report, which details the company’s goals, strategies and performance across four ESG focus areas: ethics, integrity and quality; health equity and healthcare access; global inclusion and diversity; and environmental sustainability. Highlights include:
Increased access for underserved communities.
Progress toward global inclusion and diversity and health equity aspirational goals.
Expanded clinical trial diversity and advanced supplier diversity.
A reduced environmental footprint.
Strengthened ESG oversight and accountability.
Bristol Myers Squibb was inducted into the Billion Dollar Roundtable, joining other Fortune 100 companies that have invested $1 billion with diverse-owned suppliers.
Financial Guidance
Bristol Myers Squibb is revising its 2023 GAAP and Non-GAAP line item guidance as follows:

Adjusting total revenues for Revlimid to be approximately $6.0 billion.
Adjusting GAAP diluted EPS range to $3.68-$3.83 and raising midpoint of Non-GAAP diluted EPS range, with the new range being $7.50-$7.65.
Adjusting GAAP tax rate to approximately 11% and adjusting Non-GAAP tax rate to approximately 15.5%, primarily due to a reduction in previously estimated taxes resulting from changes in the income tax guidance regarding deductibility of certain non-U.S. research and development expenses.
Key 2023 GAAP and Non-GAAP line-item guidance assumptions are:

U.S. GAAP

Non-GAAP2

July
(Prior)

October
(Revised)

July
(Prior)

October
(Revised)

Total Revenues

(as reported)

Low single-digit decline

No Change

Low single-digit decline

No Change

Total Revenues

(excl. F/X)

Low single-digit decline

No Change

Low single-digit decline

No Change

Revlimid

~ $5.5 billion

~$6.0 billion

~ $5.5 billion

~$6.0 billion

Gross Margin %

~76%

No Change

~76%

No Change

Operating Expenses1

Low single-digit decline

No Change

Low single-digit decline

No Change

Tax Rate

~ 16%

~11%

~ 17.5%

~15.5%

Diluted EPS

$3.72-$4.02

$3.68-$3.83

$7.35-$7.65

$7.50-$7.65

1 Operating Expenses — MS&A and R&D, excluding Acquired IPRD and Amortization of acquired intangible assets.

2 See "Use of Non-GAAP Financial Information."

The 2023 financial guidance excludes the impact of any potential future strategic acquisitions, including the planned acquisition of Mirati, and divestitures, and any specified items that have not yet been identified and quantified and the impact of future Acquired IPRD charges, including the charge associated with the re-acquisition of rights for mavacamten in China and certain other Asian territories. To the extent we have quantified the impact of significant R&D charges or other income resulting from upfront or contingent milestone payments in connection with asset acquisitions or licensing of third-party intellectual property rights, we may update this information from time to time on our website www.bms.com, in the "Investors" section. GAAP and Non-GAAP guidance assume current exchange rates. The 2023 Non-GAAP EPS guidance is further explained under "Use of Non-GAAP Financial Information." The financial guidance is subject to risks and uncertainties applicable to all forward-looking statements as described elsewhere in this press release.

The company will no longer include GAAP financial guidance beginning with the presentation of the fourth quarter and year-end 2023 results.

Medium-Term Financial Targets
The company is updating its previously communicated medium-term targets:

July (Prior)

October (Revised)

Low-to-mid single digit revenue CAGR1 from 2020-2025

Reaffirms low-to-mid single digit revenue CAGR1 from 2020-2025

Low double-digit revenue CAGR1 Ex-Revlimid/Pomalyst from 2020-2025

Reaffirms low double-digit revenue CAGR1 Ex-Revlimid/Pomalyst from 2020-2025

$8-$10 billion growth from in-line brands2 from 2020-2025

Reaffirms $8-$10 billion growth from in-line brands2 from 2020-2025

$10-$13 billion from new product portfolio in 2025

Adjusts to >$10 billion revenue from new product portfolio in 2026

40%+ Non-GAAP operating margin through 2025

Adjusts Non-GAAP operating margin target to >37% through 2025

1 At constant exchange rates on a risk-adjusted basis.

2 Primarily I-O and Eliquis.

Conference Call Information
Bristol Myers Squibb will host a conference call today, Thursday, October 26, 2023, at 8:00 a.m. ET during which company executives will review the quarterly financial results and address inquiries from investors and analysts. Investors and the general public are invited to listen to a live webcast of the call at View Source." target="_blank" title="View Source." rel="nofollow">View Source

Investors and the public can register for the live conference call here. Those unable to register can access the live conference call by dialing in the U.S. toll-free 1-833-816-1116 or international +1 412-317-0705. Materials related to the call will be available at View Source prior to the start of the conference call.

A replay of the webcast will be available at View Source approximately three hours after the conference call concludes. A replay of the conference call will be available beginning at 11:30 a.m. ET on October 26, 2023, through 11:30 a.m. ET on November 9, 2023, by dialing in the U.S. toll free 1-877-344-7529 or international +1 412-317-0088, confirmation code: 3515954.

Blueprint Medicines Reports Third Quarter 2023 Results

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.

SEED Therapeutics DisclosesNovel Molecular Glue IND Candidate for Cancerat “Targeted Protein Degradation Think Tank” Symposium

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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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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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.

Ascendis Presents Updated and New TransCon™ IL-2 ??? Monotherapy and Combination Therapy Data Confirming Clinical Activity Across Tumor Types at ESMO 2023

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