On October 29, 2020 Novocure (NASDAQ: NVCR) reported financial results for the quarter ended September 30, 2020, highlighting revenue growth and financial strength as well as the advancement of the company’s clinical and product development programs (Press release, NovoCure, OCT 29, 2020, View Source [SID1234569383]). Novocure is a global oncology company working to extend survival in some of the most aggressive forms of cancer by developing and commercializing its innovative therapy, Tumor Treating Fields.

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(1) Adjusted EBITDA is a non-U.S. GAAP measurement of earnings before interest, taxes, depreciation, amortization and share-based compensation.

(2) An "active patient" is a patient who is receiving treatment under a commercial prescription order as of the measurement date, including patients who may be on a temporary break from treatment and who plan to resume treatment in less than 60 days.

(3) A "prescription received" is a commercial order for Optune or Optune Lua that is received from a physician certified to treat patients for a patient not previously on Optune or Optune Lua. Orders to renew or extend treatment are not included in this total.

"We delivered another record quarter of financial performance with $133 million in net revenues and $0.09 in earnings per share," said William Doyle, Novocure’s Executive Chairman. "Our financial strength positions us well to invest in our commercial, clinical and engineering priorities to sustain long-term growth and maximize shareholder value. With readouts from key clinical trials in multiple indications anticipated over the next few years and an increased focus on technology innovation, our confidence in the potential of Tumor Treating Fields to extend survival in some of the most aggressive forms of cancer continues to build."

"We further strengthened our foundation for growth in Q3 with efforts underway to ensure organizational readiness for the company’s next chapter," added Asaf Danziger, Novocure’s Chief Executive Officer. "We ended the quarter with 3,361 active patients on therapy, adding to the more than 17,000 patients treated to-date, globally. With plans to expand access to our approved indications into additional markets, we remain focused on positioning our company to serve many more patients in the future."

Third quarter 2020 financial update

For the quarter ended September 30, 2020, net revenues were $132.7 million, representing 44% growth compared to the third quarter 2019.

In the United States, net revenues totaled $92.6 million in the quarter ended September 30, 2020, representing 51% growth compared to the same period in 2019.
In Germany and other EMEA markets, net revenues totaled $28.2 million in the quarter ended September 30, 2020, representing 15% growth compared to the same period in 2019.
In Japan, net revenues totaled $7.5 million in the quarter ended September 30, 2020, representing 57% growth compared to the same period in 2019.
In Greater China, net revenues totaled $4.3 million in the quarter ended September 30, 2020, representing 205% growth compared to the same period in 2019.
For the three months ended September 30, 2020, the increase resulted primarily from an increase of 610 active patients in our currently active markets, and a durable improvement in the net revenues booked per active patient, as well as an increase in collaboration revenues from our partnership with Zai Lab.

We recorded $10 million in revenues from Medicare fee-for-service beneficiaries billed under the coverage policy effective on September 1, 2019 in the third quarter 2020. We have gained a good understanding of how to ensure timely processing of Medicare claims and have sufficient experience to recognize approximately two-thirds of the expected contribution from Medicare beneficiaries. In the third quarter 2020, we also recognized approximately $8 million in incremental net revenues compared to the first two quarters of 2020 resulting from the successful appeal of previously denied claims for Medicare fee-for-service beneficiaries billed prior to established coverage.

Cost of revenues for the three months ended September 30, 2020 was $28.4 million compared to $22.9 million for the same period in 2019, representing an increase of 24%. The increase in cost of revenues was primarily due to the cost of shipping transducer arrays to a higher volume of commercial patients and increasing shipments of equipment to Zai Lab, partially offset by benefits of ongoing efficiency initiatives and scale. Gross margin was 79% for the three months ended September 30, 2020 compared to 75% for the three months ended September 30, 2019.

Research, development and clinical trials expenses for the three months ended September 30, 2020 were $32.8 million compared to $18.8 million for the same period in 2019, representing an increase of 75%. This was primarily due to an increase in clinical trial and personnel expenses for our phase 3 pivotal and post-marketing trials, an increase in development and personnel expenses to support our product development programs, increased investments in preclinical research and the expansion of our medical affairs activities.

Sales and marketing expenses for the three months ended September 30, 2020 were $29.4 million compared to $23.8 million for the same period in 2019, representing an increase of 23%. This was primarily due to an increase in personnel and professional services costs to support our growing commercial business and reimbursement efforts and an increase in marketing expenses related to the launch of Optune Lua for MPM.

General and administrative expenses for the three months ended September 30, 2020 were $27.1 million compared to $22.7 million for the same period in 2019, representing an increase of 19%. This was primarily due to an increase in personnel costs, insurance premiums and professional services.

Net income for the three months ended September 30, 2020 was $9.3 million compared to net income of $1.9 million for the same period in 2019.

At September 30, 2020, we had $234.5 million in cash and cash equivalents and short-term investments, a decrease of $91.6 million compared to $326.1 million at December 31, 2019. The decrease in our cash, cash equivalents and short-term investments was primarily due to the prepayment of the 2018 credit facility in the amount of $150 million, partially offset by the cash flow from operations and the exercise of options and proceeds from the issuance of shares.

The 2018 credit facility prepayment included $150 million in principal repayment and $3 million in prepayment premium, plus accrued and unpaid interest and expenses payable through the payoff date. The un-amortized issuance costs in the amount of $0.5 million that were fully amortized upon the repayment and the prepayment premium were included in the Company’s third quarter 2020 finance expenses.

Third quarter 2020 operating statistics

There were 3,361 active patients at September 30, 2020, representing 22% growth compared to September 30, 2019, and 3% growth compared to June 30, 2020.

In the United States, there were 2,218 active patients at September 30, 2020, representing 19% growth compared to September 30, 2019.
In Germany and other EMEA markets, there were 902 active patients at September 30, 2020, representing 23% growth compared to September 30, 2019.
In Japan, there were 241 active patients at September 30, 2020, representing 51% growth compared to September 30, 2019.
Additionally, 1,371 prescriptions were received in the quarter ended September 30, 2020, representing 4% growth compared to the same period in 2019, and a 4% decrease compared to the quarter ended June 30, 2020. In the quarter ended September 30, 2020, 1,117 Optune prescriptions were written for patients with newly diagnosed glioblastoma.

In the United States, 955 prescriptions were received in the quarter ended September 30, 2020, representing a 4% increase compared to the same period in 2019.
In Germany and other EMEA markets, 330 prescriptions were received in the quarter ended September 30, 2020, representing 4% growth compared to the same period in 2019.
In Japan, 86 prescriptions were received in the quarter ended September 30, 2020, representing 2% growth compared to the same period in 2019.
Third quarter 2020 non-U.S. GAAP measures

We also measure our performance based upon a non-U.S. GAAP measurement of earnings before interest, taxes, depreciation, amortization and shared-based compensation ("Adjusted EBITDA"). We believe Adjusted EBITDA is useful to investors in evaluating our operating performance because it helps investors compare the results of our operations from period to period by removing the impact of earnings attributable to our capital structure, tax rate and material non-cash items, specifically share-based compensation.

Adjusted EBITDA was $37.3 million for the three months ended September 30, 2020, an increase of $17.2 million, or 85%, from $20.1 million for the three months ended September 30, 2019. This improvement in fundamental financial performance was driven by net revenue growth coupled with an ongoing commitment to disciplined management of expenses.

Anticipated clinical milestones

Data from phase 2 pilot HEPANOVA trial in advanced liver cancer (Q1 2021)
Data from phase 2 pilot EF-31 trial in gastric cancer (2021)
Interim analysis of phase 3 pivotal LUNAR trial in non-small cell lung cancer (2021)
Interim analysis of phase 3 pivotal PANOVA-3 trial in locally advanced pancreatic cancer (2021)
Interim analysis of phase 3 pivotal INNOVATE-3 trial in recurrent ovarian cancer (2021)
Data from phase 3 pivotal METIS trial in brain metastases (2022)
Data from phase 2 pilot EF-33 trial with high-intensity arrays in recurrent glioblastoma (2022)
Final data from phase 3 pivotal LUNAR trial in non-small cell lung cancer (2023)
Final data from phase 3 pivotal PANOVA-3 trial in locally advanced pancreatic cancer (2023)
Final data from phase 3 pivotal INNOVATE-3 trial in recurrent ovarian cancer (2023)
Conference call details

Novocure will host a conference call and webcast to discuss second quarter 2020 financial results at 8 a.m. EDT today, Thursday, October 29, 2020. Analysts and investors can participate in the conference call by dialing 855-442-6895 for domestic callers and 509-960-9037 for international callers, using the conference ID 5453859.

The webcast, earnings slides presented during the webcast and the corporate presentation can be accessed live from the Investor Relations page of Novocure’s website, www.novocure.com/investor-relations, and will be available for at least 14 days following the call. Novocure has used, and intends to continue to use, its investor relations website, as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD.

On October 29, 2020 NOXXON Pharma N.V. (Euronext Growth Paris: ALNOX), a biotechnology company focused on improving cancer treatments by targeting the tumor microenvironment (TME), reported its interim 2020 results for the six months ended June 30, 2020 (Press release, NOXXON, OCT 29, 2020, View Source [SID1234569381]).

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"Despite the challenging operational environment posed by COVID-19, the NOXXON team, along with dedicated clinical researchers, was able to finalize the NOX-A12 plus immunotherapy trial in pre-treated microsatellite stable metastatic pancreatic and colorectal cancer patients. The final trial results including overall survival and the safety profile warrant further clinical development of NOX-A12 plus immunotherapy combinations. We also advanced our ongoing NOX-A12 plus radiotherapy trial in first-line brain cancer patients," said Aram Mangasarian, CEO of NOXXON. "In addition, the balance sheet of the company has been significantly strengthened and simplified, putting NOXXON in better negotiating position with potential industrial and financial partners."

Business Overview

NOXXON has been focused on clinical trials combining NOX-A12, its anti-CXCL12 tumor microenvironment targeting agent, in two distinct therapeutic combinations: 1) NOX-A12 plus immunotherapy (anti-PD1 checkpoint inhibitors) and 2) NOX-A12 plus radiotherapy. Each combination approach has a different underlying rationale and mechanism of action, and thus diversifies the risk of NOXXON’s clinical pipeline.

The combination approach of NOX-A12 plus standard of care radiotherapy is currently being tested in a dose escalation Phase 1/2 trial in newly diagnosed patients with aggressive brain cancer (glioblastoma) who would not benefit from standard of care chemotherapy and whose tumor cannot be fully resected by surgery. At multiple points in the trial, the independent Data Safety Monitoring Board (DSMB) reviewed safety and tolerability of the NOX-A12 combination and each time concluded that the trial should continue as planned. All patients in the low dose cohort completed six months of therapy in October 2020 and the data are very encouraging. Tumor volume reductions were observed in two of three patients during the six-month treatment, and in the third patient in the period after a second surgery following continued NOX-A12 treatment. Maximum tumor volume reductions were 6% and 60% for the first two patients. The third patient experienced 23% tumor volume reduction relative to the post-second surgery baseline. All patients in the mid dose cohort have been enrolled, with the first patient completing four months of combination therapy and other patients receiving their initial doses of NOX-A12 in October 2020. This means that six months of therapy for Cohort 2 will complete in April 2021.

NOXXON plans to advance the NOX-A12 plus radiotherapy combination in first-line brain cancer if the ongoing Phase 1/2 data warrant additional studies. NOXXON believes that such a pivotal trial following the current study could allow first filing of a market approval application for NOX-A12 in 2024 with first market approval targeted for 2025 if data are positive.

The Phase 1/2 trial studying the combination of NOX-A12 plus immunotherapy in metastatic pancreatic and colorectal cancer patients who had failed standard therapy reported final top-line data in September 2020. Both the NOX-A12 mechanistic data as well as the overall survival figures observed following treatment with the combination of NOX-A12 and anti-PD1 have been highly encouraging for the patient population treated in this study. The patients enrolled in the trial all had advanced disease with liver metastases and received on average their sixth-line of therapy in colorectal cancer and their fourth-line of therapy in pancreatic cancer. Despite the advanced disease and heavy pre-treatment, overall survival at one year was 20%, assessed using the Kaplan-Meier method. Notably, this group of longer-term survivors included two pancreatic cancer patients who had received their fourth-line of treatment.

NOXXON is planning to test the NOX-A12 plus immunotherapy combination in second-line pancreatic cancer with a dosing regimen of NOX-A12 optimized to induce anti-tumor immune responses. A two-step approach is planned for this indication with a first trial comparing two NOX-A12 chemotherapy combinations in second-line patients followed by a pivotal trial comparing the best combination to standard of care. With this approach, completion of the pivotal trial and filing of the first market approval application for this indication could be achieved in 2026 with approval targeted for 2027.

On the financing front, the company was able to raise €11.1 million net cash during the reporting period from a mix of private placements, convertible bonds and warrant exercises, thereby significantly strengthening its balance sheet.

NOXXON is closely monitoring the progress of COVID-19 and its potential impact on its operations. As requested by the European Medicines Agency (EMA), NOXXON has critically assessed the risks and benefits of therapy continuation and inclusion of new trial participants in its clinical trial of NOX-A12 combined with radiotherapy in first-line brain cancer patients. Following a thorough evaluation and discussion with the partners involved in the trial, it has been decided to continue both the treatment of enrolled patients and recruitment of additional patients. The safety of patients, hospital staff and employees, as well as the severity of the disease under study and the limited options currently available for treatment, were important factors in this decision. As there have been delays due to factors including COVID-19, NOXXON has added further centers to the trial to ensure adequate recruitment capacity to meet its targeted timelines. Overall, the impact on trial recruitment, the organization and the staff has been manageable.

The increased interest of investors in healthcare and the shift in the types of investors considering financing small-cap European biotech companies (particularly in France where over 150,000 new investors opened equity investment accounts according to the French regulator, the AMF), broadened the investor base of the capital market and had a positive impact on NOXXON’s ability to raise funds.

Business Highlights During First Half-Year of 2020

Significant strengthening of balance sheet – NOXXON raised €11.1 million net proceeds from multiple sources during the first half of 2020, including €7.3 million via private placements. The Dutch specialist fund Nyenburgh Investment Partners (NYIP) led the largest of the private placements announced on May 8, 2020. In addition, NOXXON has access to a remaining capacity of €16.2 million (nominal) from its convertible bonds financing with Atlas after this financing agreement was amended in October 2020.
Simplified capital structure – Increased price and liquidity during the reporting period allowed the conversion of the vast majority of outstanding warrants held by the investors Acuitas and Yorkville at the beginning of the period.
Timely advancement of NOX-A12 plus radiotherapy trial despite COVID-19– Phase 1/2 clinical trial of NOX-A12 plus radiotherapy in first-line brain cancer patients progressed well despite COVID-19. On April 2, 2020 NOXXON announced completion of patient recruitment for the first dose cohort in the Phase 1/2 brain cancer study of NOX-A12 plus radiotherapy. On April 24, 2020 the DSMB reviewed the available safety data from the low-dose group and validated recruitment of patients in the mid-dose group of NOX-A12. The recruitment of the first patient in the mid-dose group was announced on June 30, 2020.
More mature data from NOX-A12 plus immunotherapy trial –overall survival data from the Phase 1/2 NOX-A12 and immunotherapy combination trial in metastatic pancreatic and colorectal cancer patients supports the benefit to patients from NOX-A12 plus anti-PD-1 therapy. This data was presented by the principal investigator of the trial, Dr. Niels Halama, Head of Department of Translational Immunotherapy at the German Cancer Research Center (DKFZ), Heidelberg and Medical Oncologist at the German National Center for Tumor Diseases, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting on April 27, 2020.
New Supervisory Board Member – Oscar Izeboud joined the Supervisory Board of NOXXON on June 30, 2020. Oscar brings both a deep understanding of medicine and extensive experience in the financing and business side of biotechnology. While leading life science and healthcare investment banking at Kempen and NIBC, Oscar successfully closed more than 100 transactions, including seventeen IPOs and fifteen mergers or acquisitions. This experience combined with his operational biotech background makes him a valuable asset for NOXXON’s strategic development.
Business Highlights After June 30, 2020

July 2020 – NOXXON announced that the first brain cancer patient from the mid-dose cohort in the NOX-A12 plus radiotherapy study reached four weeks of treatment and that the DSMB confirmed safety and validated recruitment of additional patients.
September 2020 – Dr. Niels Halama presented final top-line clinical data from the Phase 1/2 NOX-A12 plus immunotherapy combination trial in colorectal and pancreatic cancer patients at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress 2020.
October 2020 – NOXXON announced that two of the three planned dose cohorts were fully recruited in the NOX-A12 plus radiotherapy clinical trial.
First-half 2020 Financial Results (IFRS)

NOXXON Pharma did not generate any revenues in the first half of 2020 (H1 2020). The Group – NOXXON Pharma N.V. and NOXXON Pharma AG – does not expect to generate any revenues from its product candidates in development until the Group either signs a licensing agreement or obtains regulatory approval and commercializes its products or enters into collaborative agreements with third parties.

Other operating income decreased to €33 thousand in H1 2020 (vs. €274 thousand in H1 2019). Other services provided in 2020 generated lower other operating income than the sale of raw materials and a partial waiver of management and Supervisory Board members concerning their receivables from remuneration due from the Group in H1 2019.

NOXXON dedicated its resources to research and development (R&D) and general and administrative (G&A) expenses. R&D expenses decreased to €942 thousand in H1 2020 (vs. €1,062 thousand in H1 2019). The decrease in R&D expenses was mainly driven by lower costs for drug manufacturing, service fees and other costs related to clinical trials and preclinical testing, patent costs and consulting services, partly offset by higher personnel expenses.

G&A expenses decreased to €988 thousand in H1 2020 (vs. €1,238 thousand in H1 2019). The decrease in G&A expenses was mainly driven by lower legal, consulting and audit fees, lower public and investor relations and related expenses, as well as lower other expenses, partly offset by higher personnel expenses.

Foreign exchange losses increased to €7 thousand in H1 2020 (vs. €2 thousand in H1 2019) as a result of increased volume of purchases denominated in currencies other than Euro in H1 2020.

Finance cost increased from nil in H1 2019 to €4,173 thousand in H1 2020. Finance cost in H1 2020 was predominantly due to the Atlas convertible bonds financing with respect to the issuance and conversion of convertible notes into equity and the recognition of compound derivative financial instruments, the exercise of warrants of the Yorkville equity line financing, the cashless exercise of all remaining Acuitas warrants outstanding and fair value adjustments of warrants outstanding.

Finance cost in H1 2020 was non-cash finance cost, except for €105 thousand, thereof for transaction costs of €103 thousand borne by the company in conjunction with the issuance of convertible bonds.

Finance income (all non-cash) increased to €154 thousand in H1 2020 (vs. €75 thousand in H1 2019). The increase was due to the derecognition gain of compound derivative financial instruments in connection with the Atlas convertible bonds financing in H1 2020.

As a result of the above factors, the Group’s loss before income tax increased to €5,923 thousand in H1 2020 (vs. €1,953 thousand in H1 2019). The net cash used in operating activities amounted to €1,811 thousand in H1 2020 vs. €2,687 thousand in H1 2019.

Outlook

NOXXON is making progress in its ongoing Phase 1/2 trial of NOX-A12 plus radiotherapy in first-line, inoperable brain cancer (glioblastoma) patients who are shown by biomarker analysis of their tumor tissue to be resistant to the current standard of care chemotherapy. Currently, two of the three planned dose cohorts are fully recruited. If study results are positive, NOXXON plans to seek advice from authorities under its EU/US orphan drug designation to confirm that its planned approach is acceptable to complete development and achieve market approval in brain cancer. NOXXON’s partnering goal for this combination is the identification of industrial partners that will finance additional clinical trials in brain cancer and other indications where radiotherapy is core to the standard of care. NOXXON anticipates that at least partial top-line clinical data including post-treatment follow-up from the trial will be required to close a partnership in this area.

NOXXON published more mature data from the NOX-A12 clinical trial in metastatic microsatellite stable pancreatic and colorectal cancer patients in April 2020 and has published final top-line data in September 2020. NOXXON believes that further clinical trials are warranted based on this data, in particular in pancreatic cancer, where it plans to focus its near-term efforts. The goal of NOXXON is to find industrial partners that will not only provide anti-PD1 therapy but also financial support to conduct a trial.

To prepare for future trials leading to approval of NOX-A12, NOXXON has made additional investment commitments for the manufacturing of drug supply for clinical trials.

NOXXON’s long-term strategic plans now include the following trials by indication:

NOX-A12 plus radiotherapy in Brain Cancer

Completion of the ongoing Phase 1/2 dose escalation trial, potentially with an expansion of the dose chosen for the pivotal trial. Trial completion planned for 2021 (without any expansion).
Pivotal trial of NOX-A12 combined with radiotherapy in first-line MGMT promoter unmethylated glioblastoma patients vs. standard of care (assuming ongoing Phase 1/2 trial data supports further development) planned initiation in 2022, with first market authorization application targeted for 2024 and approval targeted for 2025.
NOX-A12 plus immunotherapy in Pancreatic Cancer

Two-arm Phase 2 "pick the winner" trial testing NOX-A12 plus anti-PD1 antibody with two different standard of care chemotherapy regimens to determine the choice of regimen for the pivotal trial. Trial initiation planned for 2021 and completion in 2023.
Pivotal trial of NOX-A12 combined with immunotherapy and standard of care in second-line pancreas cancer vs. standard of care, with market authorization application targeted for 2026 and approval targeted for 2027.
The second clinical stage asset, NOX-E36, is also being prepared for the next clinical trial. Manufacturing of clinical supply has been contracted and is projected to be available in mid-2021. Pre-clinical work comparing combination strategies for NOX-E36 in solid tumors to identify the most promising approaches are also advancing. NOXXON plans to initiate the first clinical trial of NOX-E36 combinations testing safety in 2021.

NOXXON continues to evaluate other indications and therapeutic combinations in which to test NOX‑A12 and NOX-E36 as well as the relative priority of such indications for the overall corporate strategy.

The Group will carefully monitor its available cash and calibrate additional financings through various sources in order to ensure its development plans and, to the extent deemed appropriate, maintenance of a sufficient cash runway. Considering cash and cash equivalents as well as financial assets as of June 30, 2020 of €10.7 million and available, secured financing of €11.5 million (nominal) as well as a subsequent amendment to this financing agreement increasing its capacity by an additional €4.7 million (nominal) drawable at the company’s discretion and subject to customary conditions being met, cash reach of NOXXON will be into Q1 2022, including the above planned manufacturing and clinical trial commitments.

The Half-Year Financial Report 2020 can be downloaded from the NOXXON website.

On October 29, 2020 Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) ("Takeda") reported financial results for the first half of fiscal year 2020 (period ended September 30, 2020) (Press release, Takeda, OCT 29, 2020, View Source [SID1234569380]).

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TAKEDA PRESIDENT & CHIEF EXECUTIVE OFFICER CHRISTOPHE WEBER commented:

"Takeda’s performance in the first half of FY2020 demonstrates the resilience of our business model, the depth of our portfolio and the commitment of our employees, who continue to serve patients and communities globally while overcoming challenges created by the COVID-19 pandemic.

"Our results were once again driven by underlying growth of our 14 global brands across the five key business areas, which enabled us to generate strong margins and cash flow. Our R&D Engine continued to advance our Wave 1 pipeline, with 7 new regulatory filings anticipated within the next 12 months, and we expanded our cell therapy capabilities, both of which will help drive Takeda’s future growth. We also exceeded our divestiture target, with more than $11 billion of non-core disposals announced since January 2019, enabling us to continue to rapidly reduce debt.

"Takeda continues to focus on developing potential therapies to treat COVID-19 with, for example, the enrollment of the first patients in the CoVIg-19 Plasma Alliance Phase 3 clinical trial — an outstanding achievement in such a short time.

"We are confirming our full-year management guidance and raising forecasts for free cash flow, reported operating profit and reported earnings per share, reflecting our confidence in Takeda’s growth momentum. This is borne out by today’s encouraging results, despite the challenges posed by the COVID-19 pandemic. I am proud of all that we continue to achieve at Takeda and look forward to continuing to build on our strengths in the second half of FY2020."

FINANCIAL AND BUSINESS HIGHLIGHTS

Results for H1 FY2020 Ended September 30, 2020 [1],[2],[3],[4]

(billion yen, except percentages and per share amounts)

REPORTED

CORE

UNDERLYING

H1 FY2020

vs. PRIOR YEAR

H1 FY2020

vs. PRIOR YEAR

Revenue

1,590.8

-4.2%

1,590.8

-4.2%

+0.5%

Operating Profit

215.6

+97.7%

507.6

-6.3%

+1.9%

Margin

13.6%

+7.0pp

31.9%

-0.7pp

31.6%

Net Profit

86.5

+15.8%

345.5

-9.2%

EPS (JPY)

55 yen

+7 yen

221 yen

-23 yen

-0.4%

Operating Cash Flow

392.0

+14.9%

Free Cash Flow (Non-IFRS)

425.5

-37.1%

1 Underlying growth compares two periods (quarters or years) of financial results under a common basis and is used by management to assess the business. These financial results are calculated on a constant currency basis and excluding the impact of divestitures and other amounts that are unusual, non-recurring items or unrelated to our ongoing operations.

2 Core Operating Profit represents net profit adjusted to exclude income tax expenses, the share of profit or loss of investments accounted for using the equity method, finance expenses and income, other operating expenses and income, amortization and impairment losses on acquired intangible assets and other items unrelated to Takeda’s core operations, such as purchase accounting effects and transaction related costs.

3 Free Cash Flow represents cash flows from operating activities, excluding acquisition of plant, property and equipment, and including proceeds from sales of plant, property and equipment, as further adjusted to exclude the acquisition of intangible assets and the acquisition of investments, and to include the proceeds from sales and redemption of investments and proceeds from sales of business, net of cash and cash equivalents divested.

4 Further information on certain of Takeda’s Non-IFRS measures is posted on Takeda’s investor relations website at View Source

Takeda delivered a resilient performance in H1 FY2020
Reported revenue, at JPY 1,590.8 billion (~$15.1B)1, was impacted primarily by foreign exchange and divestitures, however Takeda delivered underlying revenue growth of 0.5% in the first half of FY2020, consistent with full year guidance of "low-single-digit growth".

We delivered reported operating profit of JPY 215.6 billion (~$2.0B)1, which grew 97.7%, reflecting lower purchase price accounting (PPA) and integration costs. Core operating profit, which adjusts for PPA and non-recurring items, declined year-on-year to JPY 507.6 billion (~$4.8B)1 owing to foreign exchange impact and divestitures. The core operating profit margin was 31.9%. Underlying core operating profit margin, which adjusts for the impact of foreign exchange and divestiture effects, grew to 31.6% year-on-year, driven by synergies and OPEX efficiencies.

Takeda’s reported net profit was JPY 86.5 billion, a 15.8% increase compared with the same period in the prior year.2 This is attributable mainly to lower purchase price accounting and integration costs.

Operating cash flow increased by 14.9% to JPY 392.0 billion. Free cash flow, which also reflects capital expenditures and proceeds from asset sales, was JPY 425.5 billion (~$4.0B)3, with the year-on-year growth rate impacted by the JPY 375.5 billion cash received for Xiidra in July 2019. Further de-leveraging in H1 led to a 3.7x net debt/adjusted EBITDA ratio at the end of the period.

The overall impact of the global spread of COVID-19 on Takeda’s consolidated financial results for the six-month period ended September 30, 2020 was not material. An adverse effect on revenue has been observed in some of our therapeutic areas, such as Neuroscience, for reasons such as patients visiting their medical care providers less frequently for non-life-threatening and chronic diseases. However, we have seen expansion of certain products with a more convenient administration profile. Voluntary suspension of certain business activities such as business travel and events in response to COVID-19 led to lower spending, which resulted in limited impact on Takeda’s profit. We continue to drive towards achieving our key deliverables in FY2020, while recognizing the potential for delays due to the pandemic, as detailed in Takeda’s Quick Report for the quarter ended September 30, 2020, released today. For the latest Takeda communications regarding COVID-19, please click here to visit the COVID-19 Information Center on Takeda’s website.

COMMERCIAL UPDATES ACROSS OUR FIVE KEY BUSINESS AREAS
Takeda’s five key business areas — Gastroenterology, Rare Diseases, Plasma-Derived Therapies, Oncology, and Neuroscience — with JPY 1,298.9 billion of reported revenue representing approximately 82% of total H1 revenues – delivered year-on-year underlying revenue growth of 4.0%. Our 14 global brands, with reported revenue of JPY 595.9 billion in aggregate, delivered a 15.4% increase in underlying revenue growth compared to a year before.

Gastroenterology
The Gastroenterology franchise with JPY 379.8 billion in reported revenue represented 24% of sales, spearheaded by exceptional growth through expanded patient share of gut-selective ENTYVIO in the U.S., EU, and Japan.

Rare Diseases
The Rare Diseases franchise with JPY 295.4 billion in reported revenue represented 19% of sales, with the hereditary angioedema portfolio experiencing double digit growth driven by continued strong performance and successful launches of TAKHZYRO. The competitive landscape in Rare Hematology, including a -19% decline in ADVATE sales partially driven by ADYNOVATE and competitive uptake, was in line with our expectations. Additionally, Takeda is working closely with the FDA on a proposed plan to resupply NATPARA in the U.S. and anticipates that the required device modifications and product testing will likely delay availability beyond 2020.

PDT Immunology
PDT Immunology with JPY 205.9 billion in reported revenue represented 13% of sales, driven by strong Gammagard-Liquid demand in the U.S. and subcutaneous IG worldwide. Albumin sales decreased versus H1 last year (-13%) due to phasing and high FY19 H1 sales as a result of supply dynamics in China following a blackout period. However, we expect this to recover in H2, driven by demand and capacity expansion.

Oncology
Oncology with JPY 210.0 billion in reported revenue represented 13% of sales as our portfolio continues to expand indications in metastatic non-small cell lung cancer, chronic myeloid leukemia, myeloma, and ovarian cancer.

Neuroscience
Neuroscience with JPY 207.8 billion in reported revenue represented 13% of sales, with slowing momentum attributable to COVID-19 stay-at-home restrictions that reduced patient visits and diagnoses and created opportunities for discontinuing medication. A normalization of sales was noted toward the end of Q2.

Global Brand Highlights

Business areas

Product

Reported Product Revenue

JPY (billion)

Year-over-Year Underlying

Revenue Growth

Gastroenterology

ENTYVIO

207.0

+25.8%

Rare Diseases

TAKHZYRO

43.7

+45.5%

PDT Immunology

Immunoglobulin

162.7

+ 14.2%

Oncology

NINLARO

44.4

+ 19.2%

Oncology

ALUNBRIG

4.3

+30.2%

COST SAVINGS AND DIVESTITURES
Operational efficiencies and cost savings supported margin performance and we are on track to achieve our targeted annual run rate of $2.3 billion in cost synergies by the end of FY2021. Takeda is deleveraging rapidly, with a net debt/adjusted EBITDA ratio of 3.7x at the end of Q2, down from 3.8x in March 2020. We are on course to meet our medium-term deleveraging goal of 2x within FY2021-FY2023.

Takeda exceeded its $10B non-core asset divestiture target and has announced 10 deals since January 2019 to date for a total aggregate value of up to ~$11.3 billion, including:

Agreement to divest Takeda Consumer Healthcare Company Limited to Oscar A-Co KK, a company controlled by funds managed by The Blackstone Group Inc. and its affiliates for a total value of JPY 242.0 billion. The transaction is expected to close by March 31, 2021, subject to customary legal and regulatory closing conditions. (Press release)
Agreement to divest non-core assets in Europe and Canada to Cheplapharm for approximately $562 million, subject to customary legal and regulatory closing conditions (Press Release)
Agreement to divest its TACHOSIL Fibrin Sealant Patch to Corza Health, Inc. Takeda will receive €350 million in cash upon closing of the transaction expected by March 31, 2021, which is subject to customary legal and regulatory closing conditions. (Press release)
Additionally, Takeda exceeded its $700 million target for incremental cash from real estate and securities, receiving $1.1 billion to date.

PIPELINE UPDATE: MOMENTUM IN OUR DYNAMIC R&D GROWTH ENGINE
Takeda has built a world-class R&D engine leveraging our internal research capabilities, while also actively engaging with innovative ecosystems around the world to translate science into highly innovative medicines. The main drivers for targeted new product launches are 12 unique New Molecular Entities (NMEs) in Wave 1, which represent several potential best-in-class / first-in-class therapies targeted for launch by FY2024 with aggregate potential peak sales of more than $10 billion.

As announced in the Q1 FY2020 results release, Takeda has seven of these potential Wave 1 NME filings targeted for the next 12 months: TAK-721, TAK-609, CoVIg-19, TAK-003, mobocertinib, pevonedistat and maribavir.

During Q2, Takeda’s R&D engine continued to advance its Wave 1 pipeline and expanded its capabilities in cell therapy. Wave 1 Q2 highlights include:

TAK-721 completed the rolling NDA submission and remains on track to be the first FDA-approved agent to treat eosinophilic esophagitis.
The CoVIg-19 Plasma Alliance began enrolling patients in the ITAC Phase 3 clinical trial to evaluate the safety, tolerability and efficacy of its investigational anti-coronavirus H-Ig medicine for treating hospitalized adults at risk for serious complications of COVID-19. (Press release)
TAK-003 is on track for a regulatory filing for Dengue vaccine in endemic countries in Asia and Latin America, and in the EU in Q4 FY2020.
Mobocertinib (TAK-788) demonstrated ten-month follow-up results from the Phase 1/2 trial presented at the virtual European Society for Medical Oncology Conference (ESMO) (Free ESMO Whitepaper) and achieved a duration of response (DoR) of more than one year in the trial’s study population of patients with epidermal growth factor receptor (EGFR) Exon20 insertion+ metastatic NSCLC (mNSCLC). (Press release)
Soticlestat (TAK-935/OV935): Our Phase 2 ELEKTRA Study of Soticlestat in partnership with Ovid Therapeutics Inc. met its primary endpoint for reducing seizure frequency in children with Dravet Syndrome or Lennox-Gastaut Syndrome. (Press release)
TAK-994, the first oral OX2R agonist, is in phase 2 enrolling NT1 and NT2 patients. Final data is targeted for 2H FY21.
Other notable updates
We expanded our cell therapy manufacturing capabilities in Boston, Massachusetts to support next-generation clinical programs including TAK-007, TAK-940 and TAK-102. This expansion provides end-to-end research and development capabilities and will accelerate Takeda’s efforts to develop next-generation cell therapies, initially focused on oncology with potential to expand into other therapeutic areas. Five collaborative oncology cell therapy programs are expected to be in clinical development by the end of FY2021 and Takeda has already initiated clinical studies testing its CAR-T candidates, TAK-940 and TAK-102 in Q2. (Press release, website)

We are excited about the transformative medium-term potential of a number of our major R&D programs, including:

TAK-999: Takeda and Arrowhead Pharmaceuticals entered into a collaboration to develop TAK-999, a first-in-class GalNAc based RNAi designed to treat the underlying cause of Alpha-1 Antitrypsin-Associated Liver Disease. (Press release)
TAK-981, a first-in-class small molecule inhibitor of sumoylation that activates type 1 interferon signaling and lymphocyte activation, is being explored in more than 10 expansion cohorts with adaptive trial designs in a broad range of tumor types with a high unmet need. Emerging data demonstrated the therapy is well tolerated and responses have been observed in single-agent dose-escalation in solid tumors and in combination with rituximab in Non-Hodgkin’s lymphoma.
Takeda will share further pipeline progress at our upcoming Wave 1 Pipeline Market Opportunity call on December 8, 2020.

KEY CORPORATE INITIATIVE
Takeda is accelerating its digital transformation through a collaboration with Accenture and AWS that will leverage cloud and data-driven insights to improve our productivity across our value chain, increase operational agility, reduce technology costs, and develop the workforce of the future.

We are investing in developing capabilities and hiring new talent in emerging fields of data and digital. We will also train employees in cloud, advanced analytics, robotic process automation (RPA), agile and innovation sprint methodologies. By moving 80% of applications to the cloud, we will remove non-differentiating technology, reduce our internal data center footprint, and decrease capital expenditures. (Press release)

FIGHTING COVID-19 UPDATE
Guided by our values, Takeda’s response to COVID-19 has focused on protecting the health and safety of our employees, striving to ensure our medicines are available to patients who rely on them and playing our part to reduce transmission and support the communities where our employees live and work. Takeda has also undertaken a number of efforts to help the world respond to COVID-19, including:

Novavax and Takeda Announce Collaboration for Novavax’ COVID-19 Vaccine Candidate in Japan

Press release

Takeda Expands COVID-19 Vaccine Supply in Japan Through Partnership with Moderna and Government of Japan

Press release

First Patient Enrolled in NIH Phase 3 Trial to Evaluate Potential COVID-19 Hyperimmune Medicine

Press release

CoVIg-19 Plasma Alliance Builds Strong Momentum Through Expanded Membership and Clinical Trial Collaboration

Press release

Members of the COVID R&D Alliance and Quantum Leap Healthcare Collaborative Enroll First Patients in I-SPY COVID Trial

Press release

FY2020 GUIDANCE

Updating Full-Year Reported Forecast; Core and Underlying Guidance Confirmed

Takeda has solid growth momentum heading into H2 2020 and potential for accelerated underlying growth and achieving an underlying core operating profit margin in the mid-30s over the medium term.
Core and underlying guidance for FY2020 remains unchanged. Takeda upgraded its reported operating profit, reported net profit, and reported EPS forecast for FY2020 to reflect assumptions for one-time gains from several announced divestitures that were not included in the previous forecast but are now expected to be recognized within the current fiscal year, with the exception of the sale of shareholdings in Takeda Consumer Healthcare Company Limited.4 The reported revenue forecast for FY2020 has been reduced from JPY 3,250.0 billion to 3,200.0 billion, owing primarily to the impact of foreign exchange.

Key assumptions in FY2020 forecast
Company guidance reflects management’s expectations for continued business momentum across Takeda’s five key business areas, underlying revenue growth of our 14 global brands, and accelerated realization of cost synergies.

FY2020 guidance also reflects the following key assumptions, including (i) that there will not be an additional 505(b)2 competitor for subcutaneous VELCADE launched in the U.S. within FY2020; (ii) includes the impact of divestitures disclosed by Takeda as through October 29, 2020, with the exception of the divestment of Takeda Consumer Healthcare Company; and (iii) management’s current expectations regarding COVID-19.

Based on currently available information, Takeda believes that its financial results for FY2020 will not be materially affected by COVID-19 and, accordingly, Takeda’s FY2020 forecast reflects this belief. However, the situation surrounding COVID-19 remains highly fluid, and future COVID-19-related developments in FY2020, including new or additional COVID-19 outbreaks and additional or extended lockdowns, shelter-in-place orders or other government action in major markets, could result in further or more serious disruptions to Takeda’s business, such as slowdowns in demand for Takeda’s products, supply chain related issues or significant delays in its clinical trial programs. These events, if they occur, could result in additional impacts on Takeda’s business, results of operations or financial condition, as well as resulting in significant deviations from Takeda’s FY2020 forecast.

On October 29, 2020 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported that it will host a live conference call and webcast at 4:30 p.m. ET on Thursday, November 5, 2020 to report its third quarter 2020 financial results and provide a corporate update (Press release, Syros Pharmaceuticals, OCT 29, 2020, View Source [SID1234569378]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

To access the live conference call, please dial 866-595-4538 (domestic) or 636-812-6496 (international), and refer to conference ID 1088286. A webcast of the call will also be available on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 30 days following the presentation.

On October 29, 2020 Blueprint Medicines Corporation (NASDAQ:BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported financial results and provided a business update for the third quarter ended September 30, 2020 (Press release, Blueprint Medicines, OCT 29, 2020, View Source [SID1234569377]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"Following our tremendous progress in the third quarter, Blueprint Medicines is in a very strong position, with two precision therapies now approved and significant near-term growth opportunities expected across our portfolio," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "In recent months, we bolstered our commercial foundation with the U.S. approval and launch of GAVRETO, and we are now preparing to file our supplemental NDA for AYVAKIT in advanced systemic mastocytosis, marking an important step toward delivering a much-needed new therapy to patients across the systemic mastocytosis disease spectrum. In parallel, we continued to strengthen our pipeline with the presentation of compelling preclinical proof-of-concept data for BLU-945, a potentially best-in-class therapy for treatment-resistant EGFR-driven lung cancer, that support our plan to initiate clinical development of BLU-945 in the first half of next year."

Third Quarter 2020 Highlights and Recent Updates

AYVAKIT (avapritinib): systemic mastocytosis (SM)

●Announced positive top-line results from the Phase 1 EXPLORER and Phase 2 PATHFINDER clinical trials of AYVAKIT in patients with advanced SM. Consistent with previously reported EXPLORER trial results, the data showed profound reductions in mast cell burden, high overall response and complete remission rates, and durable clinical benefit, including prolonged median overall survival. AYVAKIT was generally well-tolerated, with an improved safety profile at the 200 mg once daily dose. Read the press release here.

AYVAKIT/AYVAKYT (avapritinib): gastrointestinal stromal tumor (GIST)

●Recorded $6.1 million in net product revenue during the third quarter of 2020 for AYVAKIT, which was approved by the U.S. Food and Drug Administration (FDA) in January 2020 for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.
●Received European Commission (EC) conditional marketing authorization for avapritinib under the brand name AYVAKYT as a monotherapy for the treatment of adult patients with unresectable or metastatic GIST harboring the PDGFRA D842V mutation and initiated the first commercial launch following EC approval in Germany. Read the press release here.

GAVRETO (pralsetinib): RET-altered cancers

●Received FDA approval of GAVRETO for the treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test and launched GAVRETO together with Genentech, a member of the Roche Group, in the U.S. in September. Read the press release here. Please click here to see the full Prescribing Information for GAVRETO and visit www.GAVRETO.com for more information on GAVRETO.
●Reported updated data from the ongoing ARROW clinical trial of GAVRETO in advanced RET-mutant medullary thyroid cancer (MTC) at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress

2020. The data showed durable responses and a well-tolerated safety profile for GAVRETO in patients with advanced RET-mutant MTC, with consistent clinical activity in patients across lines of therapy and regardless of RET mutation genotypes. Read the press release here.
●Announced acceptance of U.S. marketing application for GAVRETO for the treatment of patients with advanced or metastatic RET-mutant MTC and RET fusion-positive thyroid cancer. The FDA accepted the new drug application (NDA) for priority review under its Real-Time Oncology Review (RTOR) pilot program, and set an action date of February 28, 2021 under the Prescription Drug User Fee Act.

BLU-945: EGFR-mutated NSCLC

●Reported preclinical proof-of-concept data for BLU-945 at the ESMO (Free ESMO Whitepaper) Virtual Congress 2020. The data showed BLU-945 potently and selectively inhibited triple-mutant epidermal growth factor receptor (EGFR) harboring the most common on-target resistance mutations to standard treatments for EGFR-mutated NSCLC, resulting in robust anti-tumor activity in multiple lung cancer models. Read the press release here.
●Announced plans to develop BLU-945 as a monotherapy and in combination with other agents for the treatment of patients with treatment-resistant EGFR NSCLC.

Corporate:

●Strengthened the company’s leadership team with the appointment of Fouad Namouni, M.D., as President, Research & Development. In this new role, Dr. Namouni leads a joint research and development organization, overseeing all phases of product development from discovery through global registration under a unified portfolio vision.

Key Upcoming Milestones

The company expects to achieve the following near-term milestones:

●Submit a supplemental new drug application to the FDA for avapritinib for the treatment of patients with advanced SM in the fourth quarter of 2020.
●Obtain U.S. approval of GAVRETO for RET-altered thyroid cancers in the first quarter of 2021.
●Initiate a global Phase 1 dose-escalation trial for BLU-945 in EGFR-mutated NSCLC in the first half of 2021.

Third Quarter 2020 Financial Results

●Revenues: Revenues were $745.1 million for the third quarter of 2020, including $6.1 million of net product revenues from sales of AYVAKIT, $0.2 million of net product revenues from sales of GAVRETO and $738.8 million in collaboration revenues under the collaboration agreements with Roche and CStone. Blueprint Medicines recorded $9.1 million in collaboration revenues for the third quarter of 2019.
●Cost of Sales: Cost of sales was $0.1 million for the third quarter of 2020. Blueprint Medicines did not incur cost of sales in the third quarter of 2019, as no product sales were generated during that period.
●R&D Expenses: Research and development expenses were $74.2 million for the third quarter of 2020, as compared to $81.5 million for the third quarter of 2019. This decrease was primarily due to reimbursement from the global development cost sharing arrangement under the collaboration agreement with Roche for pralsetinib. Research and development expenses included $8.6 million in stock-based compensation expenses for the third quarter of 2020.
●SG&A Expenses: Selling, general and administrative expenses were $37.4 million for the third quarter of 2020, as compared to $25.6 million for the third quarter of 2019. This increase was primarily due to an increase in costs and personnel expenses associated with building Blueprint Medicines’ commercial infrastructure for commercialization of AYKAKIT and GAVRETO, partially offset by reimbursement for Roche’s share of the loss generated from the commercialization of GAVRETO in the U.S. under the collaboration for pralsetinib. Selling, general and administrative expenses included $11.0 million in stock-based compensation expenses for the third quarter of 2020.

●Net Income (Loss): Net income was $634.0 million for the third quarter of 2020, or a diluted net income per share of $11.16, as compared to a net loss of $94.3 million for the third quarter of 2019, or a diluted net loss per share of $1.93.
●Cash Position: As of September 30, 2020, cash, cash equivalents and investments were $1,355.9 million, as compared to $548.0 million as of December 31, 2019. This increase was primarily related to upfront payments of $775.0 million received in the third quarter of 2020 under Blueprint Medicines’ collaboration with Roche and $308.4 million in net proceeds received from Blueprint Medicines’ January 2020 follow-on underwritten public offering, partially offset by cash used in operating activities.

Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:30 a.m. ET today to discuss third quarter 2020 financial results and recent business activities. The conference call may be accessed by dialing (855) 728-4793 (domestic) or (503) 343-6666 (international), and referring to conference ID 2995408. A webcast of the call will 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 Conference

Blueprint Medicines will participate in the Jefferies Virtual London Healthcare Conference on November 17, 2020 at 2:55 p.m. ET. A live webcast of the 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.