Ipsen delivers solid sales growth in the first nine months of 2023 and confirms its full-year guidance

On October 26, 2023 Ipsen, a global specialty-driven biopharmaceutical company, reported its sales performance for the year to date and the third quarter of 2023 (Press release, Ipsen, OCT 26, 2023, View Source [SID1234636421]).

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PTC Therapeutics Provides Corporate Update and Reports Third Quarter Financial Results

On October 26, 2023 PTC Therapeutics, Inc., (NASDAQ: PTCT) reported a corporate update and financial results for the third quarter ending September 30, 2023 (Press release, PTC Therapeutics, OCT 26, 2023, View Source [SID1234636412]).

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"I am very proud of the progress all of our teams made this quarter," said Matthew Klein, M.D., Chief Executive Officer, PTC Therapeutics, Inc. "The recent Royalty Pharma transaction and restructuring initiatives that we implemented have put the company on a strong financial footing. We believe we are well positioned to deliver on our most promising opportunities for growth, including the potential sepiapterin revenue opportunity of more than $1 billion and the PTC518 HD program."

Key Corporate Updates:

Finalized strategic partnership transaction with Royalty Pharma, in which Royalty Pharma acquired additional royalties of Evrysdi for $1.0 billion upfront. The agreement included options for PTC to sell the remainder of its royalties of Evrysdi for up to $500 million or for Royalty Pharma to acquire half of such retained royalties for up to $250 million at a later date, less royalties received by PTC. PTC maintains all economics associated with up to $250 million in the remaining commercial sales milestones associated with Evrysdi global net sales.
Third quarter 2023 revenue for the DMD franchise was $136 million, supporting increasing 2023 DMD franchise revenue guidance to between $565 million and $595 million.
Translarna (ataluren) quarterly net product revenue was $69 million, with new patients in existing geographies and continued geographic expansion.
Emflaza (deflazacort) quarterly net product revenue was $67 million, with new patient starts and high compliance.
Key Clinical and Regulatory Updates:

For Translarna, following the negative opinion from the CHMP, the CHMP gave PTC the option to request re-examination of both opinions or only one opinion. PTC decided to pursue re-examination of the negative opinion on renewal of the conditional authorization only. In accordance with EMA guidelines, PTC expects the opinion from the re-examination procedure in late January 2024, with ratification of that opinion by the European Commission 67 days later.

For the United States, a type C meeting with the FDA for Translarna is scheduled for the fourth quarter of 2023.
PTC held a pre-NDA meeting in the third quarter with the FDA for sepiapterin in PKU to discuss the NDA submission. At the meeting, the FDA stated that the sepiapterin clinical safety and efficacy data supported NDA submission for the treatment of pediatric and adult PKU patients. It was requested that PTC complete an additional 26-week nonclinical mouse study to assess sepiapterin carcinogenicity potential prior to NDA submission. This nonclinical study was not initially required when sepiapterin was acquired from Censa, as the NDA submission was planned under the Section 505(b)(2) pathway. With PTC’s decision to file under the Section 505(b)(1) pathway, the 26-week study is considered a required NDA component needed to inform labeling and is typically completed prior to submission. PTC will continue to discuss with the FDA the potential to submit the mouse study results during the NDA review process. PTC now expects the NDA submission to occur no later than the third quarter of 2024; the submission could be submitted during the second quarter of 2024 if the nonclinical study report can be submitted during the review process.
PTC expects to submit an MAA to the EMA for sepiapterin for the treatment of PKU in the first half of 2024.
Enrollment in the PIVOT-HD study for PTC518 for Huntington’s disease continues outside of the US for both the stage 2 and early stage 3 cohorts. PTC expects the next data update to occur in the first half of 2024. This update will include 12-month data on the initial group of subjects for which data was reported in June of this year.
PTC had a type A meeting with the FDA to discuss the clinical safety data needed to enable enrollment of the PIVOT-HD trial at US study sites. At the meeting, the FDA stated that the existing three months of safety data could support 12-week dosing at 5mg and 10mg dose levels and that six months of clinical safety data demonstrating a similar favorable safety profile could support 12-month dosing in PIVOT-HD.
PTC had a type C written-response-only meeting with FDA for vatiquinone for Friedreich ataxia to determine whether the data from the MOVE-FA study would be sufficient to support an NDA for accelerated approval. In their written response, the FDA stated that while they see the value of upright stability as a clinically meaningful endpoint, they believed a confirmatory study would likely be needed to support NDA submission. PTC has requested a follow-up live meeting to address the issues raised by the FDA.
PTC is participating in a scientific advice procedure with the EMA to determine if the MOVE-FA data could support a conditional marketing authorization application in the EEA. PTC expects to have the outcome of this procedure in the first quarter of 2024.
PTC had an informal meeting with the FDA for Upstaza for AADC deficiency. The FDA stated that the data PTC has provided to support comparability between the clinical drug product and the intended commercial drug product were still not sufficient. The FDA did say that the available data from the ongoing clinical study in the US assessing the safety of the drug delivery cannula could be used to support a BLA for accelerated approval based on biomarker data demonstrating a treatment-related increase in de novo dopamine production. The FDA suggested that PTC conduct a pre-BLA meeting prior to BLA submission to review BLA contents. This meeting has been scheduled for December 2023, and pending the outcome, PTC expects to submit the BLA shortly thereafter.
Third Quarter 2023 Financial Highlights:

Total revenues were $196.6 million for the third quarter of 2023, compared to $217.1 million for the third quarter of 2022.
Total revenues include net product revenue across the commercial portfolio of $144.0 million for the third quarter of 2023, compared to $134.2 million for the third quarter of 2022. Total revenues also include collaboration, royalty and manufacturing revenue of $52.5 million in the third quarter of 2023, compared to $82.9 million for the third quarter of 2022.
Translarna net product revenue was $69.0 million for the third quarter of 2023, compared to $76.6 million for the third quarter of 2022. These results were due to new patients in existing geographies and continued geographic expansion, while the quarter over quarter decrease was due to the timing of bulk government orders.
Emflaza net product revenue was $67.4 million for the third quarter of 2023, compared to $54.8 million for the third quarter of 2022. These results reflect new patient starts and high compliance.
Roche reported Evrysdi 2023 year-to-date sales of approximately CHF 1,065 million, resulting in royalty revenue of $50.2 million to PTC for the third quarter of 2023, as compared to $32.9 million for the third quarter of 2022.
Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $164.2 million for the third quarter of 2023, compared to $165.5 million for the third quarter of 2022.
Non-GAAP R&D expenses were $150.2 million for the third quarter of 2023, excluding $14.0 million in non-cash, stock-based compensation expense, compared to $150.4 million for the third quarter of 2022, excluding $15.1 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $80.9 million for the third quarter of 2023, compared to $80.1 million for the third quarter of 2022.
Non-GAAP SG&A expenses were $67.9 million for the third quarter of 2023, excluding $13.0 million in non-cash, stock-based compensation expense, compared to $66.5 million for the third quarter of 2022, excluding $13.6 million in non-cash, stock-based compensation expense.
During the third quarter of 2023, PTC incurred additional reductions in workforce as part of the continued strategic portfolio prioritization, which resulted in a one-time charge of approximately $22.6 million recorded to R&D and SG&A expense.
The change in the fair value of deferred and contingent consideration was a loss of $1.5 million for the third quarter of 2023, compared to a gain of $5.3 million for the third quarter of 2022.
The net loss was $133.0 million for the third quarter of 2023, compared to a net loss of $109.3 million for the third quarter of 2022.
Cash, cash equivalents, and marketable securities was $294.8 million on September 30, 2023, compared to $410.7 million at December 31, 2022.
Shares issued and outstanding as of September 30, 2023, were 75,459,022.
PTC Updates Full Year 2023 Financial Guidance as Follows:

PTC anticipates total revenues for full-year 2023 to be between $940 million and $1.0 billion.
PTC anticipates net product revenue for the DMD franchise for full-year 2023 to be between $565 million and $595 million.
PTC anticipates GAAP R&D and SG&A expenses for full-year 2023 to be between $915 million and $965 million.
PTC anticipates Non-GAAP R&D and SG&A expenses for full year 2023 to be between $810 million and $860 million, excluding estimated non-cash stock-based compensation expense of $105 million.
PTC expects to incur $37 million of one-time expenses related to the achievement of clinical success-based milestones from previous acquisitions and expenses associated with a rights exchange agreement, which have already been paid in equity and cash.

Monte Rosa Therapeutics Announces $25 Million Registered Direct Offering, Priced At- the-Market Under Nasdaq Rules

On October 26, 2023 Monte Rosa Therapeutics, Inc. (Nasdaq: GLUE), a clinical-stage biotechnology company developing novel molecular glue degrader (MGD)-based medicines, reported that it has raised $25 million at-the-market from a life sciences-dedicated investor (Press release, Monte Rosa Therapeutics, OCT 26, 2023, View Source [SID1234636411]).

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The company has entered into a definitive agreement for the issuance and sale of pre-funded warrants to purchase 10,000,400 shares of its common stock at an offering price of $2.4999 per pre-funded warrant, which represents the closing price for the common stock on October 25th less the $0.0001 per share exercise price for each pre-funded warrant. The pre-funded warrants will be immediately excercisable at a nominal exercise price of $0.0001 per share and may be exercised at any time until the pre-funded warrants are exercised in full. The closing of the offering is expected to occur on or about October 30, 2023, subject to the satisfaction of customary closing conditions.

The gross proceeds to Monte Rosa from this offering are expected to be approximately $25 million, before deducting certain offering expenses. Monte Rosa intends to use the net proceeds from the offering towards its further advancement of its clinical and late preclinical pipeline, including MRT-2359, its orally bioavailable, GSPT1-directed MGD, MRT-6160, its orally bioavailable VAV1-directed MGD, and its NEK7 program, to key value inflection points.

The securities above are being offering pursuant to a shelf registration statement on Form S-3 (333-266003) that was filed with the Securities and Exchange Commission (the "SEC") on July 1, 2022, and was declared effective by the SEC on July 13, 2022. The offering is being made only by means of the written prospectus and prospectus supplement that form a part of the registration statement. A prospectus supplement relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov.

This press release does not constitute an offer to sell or the solicitation of offers to buy any of the securities being offered, and shall not constitute an offer, solicitation or sale of any security in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

BioMimetix Announces Acceptance of Late-Breaking Abstract and Oral Presentation of BMX-001 Clinical Data at the 2023 Society for Neuro-Oncology Annual Meeting

On October 26, 2023 BioMimetix JV, LLC, a clinical-stage biotechnology company developing metalloporphyrins, a novel drug class for the treatment of cancer patients, reported that data from its Phase 2 study of lead candidate, BMX-001, for the treatment of high-grade glioma (HGG), has been selected as a late breaking oral presentation at the 2023 Society for Neuro-Oncology (SNO) Annual Meeting taking place on November 15-19, 2023 in Vancouver, Canada (Press release, BioMimetix Pharmaceutical, OCT 26, 2023, View Source [SID1234636401]).

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In line with conference publication guidelines, Late-Breaking Abstracts will be made public at 7:00 a.m. PT on the first day of the meeting, Friday, November 17, 2023 and at View Source

Details of the oral presentation are as follows:

Title: Results of BMX-HGG study: a multi-institutional, randomized phase 2 clinical trial of concurrent chemoradiation with or without BMX-001 in patients with newly diagnosed high-grade glioma
Abstract Number: LTBK-09
Date and Time: Friday, November 17, 2023, from 9:05 – 9:15 a.m. PT
Session: Plenary I, Exhibit Hall C
Presenter: Katherine Peters, M.D., Duke University Medical Center

Takeda Announces FY2023 H1 Results; Updates Full-Year Forecasts While Remaining on Track Towards Management Guidance

On October 26, 2023 Takeda (TOKYO:4502/NYSE:TAK) reported financial results for the first half of fiscal year 2023 (six months ended September 30, 2023), updating reported and Core forecasts and reconfirming Management Guidance for Core change at CER (Press release, Takeda, OCT 26, 2023, View Source [SID1234636400]).

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Takeda chief executive officer, Christophe Weber, commented:
"We continue to pursue our vision to discover and deliver life-transforming treatments, making further progress in the first half of our fiscal year to address unmet medical needs and provide new treatment options to improve patient outcomes and quality of life. Our pipeline is robust and we anticipate a number of important milestones in the second half of the fiscal year, including potentially up to three new product approvals in the U.S., for TAK-755, fruquintinib, and TAK-721.

"Development setbacks with EXKIVITY and ALOFISEL, which impacted our reported profit in FY2023 Q2, highlight the inherent risk in research and development in the pharmaceutical business, but do not hinder our strategy for a return to growth in the near-term and our confidence in the long-term strength of the business remains firm."

Takeda chief financial officer, Costa Saroukos, commented:
"We are raising our full-year revenue and Core EPS forecasts to reflect updated foreign exchange and tax rate assumptions, while revising our reported operating profit and EPS forecasts to reflect large non-core items booked in our second quarter. These items do not reflect the underlying momentum of the business, and we remain on track to our Management Guidance, which is on a Core basis at CER.

"We continue to see strong performance of our Growth and Launch Products, which grew +13% at CER in the first half of the fiscal year and represent 42% of total revenue. While our Core revenue and profit performance in the first half continued to reflect the temporary headwinds we face this fiscal year, largely from generic competition, we believe our Growth and Launch Products will drive a return to revenue and profit growth in the near-term."

FINANCIAL HIGHLIGHTS

Results for FY2023 H1 Ended September 30, 2023

(Billion yen,
except
percentages and
per share
amounts)

REPORTED

CORE(c)

(Non-IFRS)(a)

FY2023 H1

vs. PRIOR YEAR

(Actual % change)

FY2023 H1

vs. PRIOR YEAR

(Actual % change)

vs. PRIOR YEAR

(CER % change(d))

Revenue

2,101.7

+6.4%

2,101.7

+6.4%

+1.4%

Operating Profit

119.2

-53.2%

588.8

-5.8%

-9.5%

Margin

5.7%

-7.2pp

28.0%

-3.6pp

Net Profit

41.4

-75.2%

407.7

-8.7%

-13.8%

EPS (yen)

27

-75.4%

261

-9.4%

-14.4%

Operating Cash Flow

291.3

-4.6%

Free Cash Flow
(Non-IFRS)(a)(b)

-71.1

N/A

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

(b) We define Free Cash Flow as cash flows from operating activities, subtracting acquisition of property, plant and equipment ("PP&E"), intangible assets and investments as well as removing any other cash that is not available to Takeda’s immediate or general business use, and adding proceeds from sales of PP&E, as well as from sales of investments and businesses, net of cash and cash equivalents divested.

(c) Core results adjust our reported results calculated and presented pursuant to IFRS to exclude the effect of items unrelated to Takeda’s core operations, such as, to the extent applicable for each line item, non-recurring items, purchase accounting effects and transaction related costs, as well as amortization and impairment of intangible assets and other operating income and expenses.

(d) CER (Constant Exchange Rate) change eliminates the effect of foreign exchange rates from year-over-year comparisons by translating Reported or Core results for the current period using corresponding exchange rates in the same period of the previous fiscal year.

FY2023 OUTLOOK
Updating Full-Year FY2023 Reported and Core Forecasts, and Reconfirming Management Guidance

Based on Takeda’s first half results and reflecting expected foreign exchange rates during the second half of FY2023, Takeda’s reported and Core forecasts have been revised from the original forecast.

(Billion yen except per share amounts)

FY2023 ORIGINAL
FORECAST
(May 2023)

FY2023 REVISED
FORECAST
(October 2023)

FY2023 MANAGEMENT
GUIDANCE
Core Growth at CER
(Non-IFRS)
(Unchanged from May 2023)

Revenue

3,840.0

3,980.0

Core Revenue

3,840.0

3,980.0

Low-single-digit % decline

Reported Operating Profit

349.0

225.0

Core Operating Profit

1,015.0

1,015.0

Low-10s % decline

Reported Net Profit

142.0

93.0

Reported EPS (yen)

91

59

Core EPS (yen)

434

447

Low-20s % decline

Free Cash Flow*

400.0-500.0

400.0-500.0

Annual Dividend per Share (yen)

188

188

*Free Cash Flow forecast reflects expenditures related to the acquisition of TAK-279 from Nimbus (JPY134.1 billion) and in-licensing of fruquintinib from HUTCHMED (JPY55.1 billion).

Additional Information About Takeda’s H1 Earnings Results
For more details on Takeda’s FY2023 H1 results and other financial information, including key assumptions in FY2023 forecast and management guidance, please visit: View Source

For more information on Takeda’s commercial progress across the five key business areas and pipeline updates, please visit: View Source