On October 27, 2022 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, reported that it will host a conference call on Wednesday, November 2, 2022, at 5:00pm ET to discuss its financial results and corporate update for the quarter ended September 30, 2022 (Press release, Ultragenyx Pharmaceutical, OCT 27, 2022, https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-host-conference-call-third-quarter-2022-financial [SID1234622480]).

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The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call, please register by clicking on the following link (registration link), and you will be provided with dial-in details. The replay of the call will be available for one year.

On October 27, 2022 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that it has entered into a strategic financing collaboration with funds managed by Blackstone (NYSE: BX) to support its mission to reach a steady-state of delivering at least one therapy every 2-3 years to continue to bring transformative medicines to patients globally (Press release, PTC Therapeutics, OCT 27, 2022, View Source [SID1234622479]). As part of the collaboration, funds managed by Blackstone Life Sciences and Blackstone Credit will provide PTC with an initial $500 million commitment, including $350 million in low-cost, low-dilution capital at close.

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"This strategic financing will support the acceleration of PTC’s robust and diversified pipeline, business development opportunities and general corporate purposes," said Emily Hill, Chief Financial Officer, PTC Therapeutics. "Our strong internal drug development and commercialization efforts, record of business development and value creation, combined with Blackstone’s network and life sciences expertise, puts PTC in a strong position to continue to execute our mission."

This collaboration with Blackstone capitalizes on PTC’s multiple innovative therapeutic platforms and its proven track record of delivering transformative treatments to patients living with rare diseases. The customized senior secured term loan investment by Blackstone is collateralized by Translarna, Emflaza, Upstaza, sepiapterin, and vatiquinone.

Specifically, the transaction is comprised of the following components:

$300 million of senior secured debt at 7.25% + SOFR for a seven-year term
$50 million purchase of PTC common stock
$150 million delayed draw debt for up to 18 months
$500 million in potential credit facility or other investment capital to support business development opportunities, subject to mutual agreement between Blackstone and PTC
Commenting on the arrangement, Kiran Reddy, MD and Craig Shepherd, Senior Managing Directors with Blackstone Life Sciences said, "This transaction demonstrates our unique ability, in partnership with Blackstone Credit, to combine deep domain expertise and flexible scale capital in the life sciences, helping to grow impressive companies like PTC Therapeutics as they optimize their capital structure and advance important treatments for patients. PTC has a deep track record of bringing life-changing therapies to patients and we are excited to support them as they launch important initiatives with the goal of commercializing new medicines."

Brad Colman, Senior Managing Director with Blackstone Credit added, "The collaboration represents how Blackstone can support world-class life sciences and healthcare companies – providing critical resources to help grow their businesses. Our collaboration will enable PTC to study new potential medicines and brings them a secure, long-term capital solution."

Advisors
WilmerHale served as legal counsel to PTC Therapeutics. Ropes & Gray LLP served as legal counsel to Blackstone.

On October 27, 2022 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare and genetically defined diseases, and Sagard Healthcare Partners ("Sagard") reported that Agios has sold its rights to 5% royalties on U.S. net sales of Servier’s TIBSOVO (ivosidenib tablets) to Sagard for a one-time payment of $131.8 million (Press release, Agios Pharmaceuticals, OCT 27, 2022, View Source [SID1234622478]). TIBSOVO is an oral, targeted therapy approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with an isocitrate dehydrogenase-1 (IDH1) mutation with acute myeloid leukemia (AML) or cholangiocarcinoma (bile duct cancer). In 2021, Agios completed the sale of its oncology portfolio – including TIBSOVO – to Servier. Agios retains its rights to a potential future milestone payment of $200 million for vorasidenib, as well as 15% royalties on U.S. net sales of vorasidenib.

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"As a company solely focused on making an impact for people with rare and genetically defined diseases, this non-equity funding provides us with increased financial flexibility to continue to advance our mission," said Brian Goff, chief executive officer at Agios. "As the result of a competitive process, we achieved an attractive valuation for the TIBSOVO royalty and identified an agile and collaborative financial partner in Sagard Healthcare Partners. As we execute our PYRUKYND commercial launch, advance multiple pivotal studies across thalassemia, sickle cell disease and pediatric pyruvate kinase deficiency, and build a sustainable pipeline through both internally and externally discovered assets, this deal enhances our ability to stay laser-focused on unlocking the greatest impact for people living with genetically defined diseases."

"Agios is a pioneer in cellular metabolism with a track record of developing innovative, targeted therapies for patients with tremendous unmet need," said David MacNaughtan, Partner & Head of Sagard Healthcare Partners. "We are pleased to invest in TIBSOVO, which represents an important therapeutic approach for patients with hematologic malignancies with an IDH1 mutation."

Wilmer Cutler Pickering Hale and Dorr LLP served as legal advisor to Agios. Mintz, Levin, Cohn, Ferris, Glovsky & Popeo, PC acted as legal advisor to Sagard on the transaction.

On October 27, 2022 PharmaMar Group (MSE: PHM) reported total revenues of €141.4 million in the first nine months of 2022, compared with €140.3 million in the same period last year. Of total revenues, recurring revenues (sales plus royalties) increased by 3% to €119.3 million during the first nine months of the year (Press release, PharmaMar, OCT 27, 2022, View Source [SID1234622477]). Of these revenues, sales of Yondelis (trabectedin) continue to grow, with gross sales up 6% to September 30th. Despite pressure on prices, net sales rose of almost 1% to €52.2 million.

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Revenues from Zepzelca (lurbinectedin) under the "early access" program totaled €13.8 million as of September 30th (vs. €23.3 million as of September 2021). This amount includes the impact of the entry into force in France of the regulations governing the prices of drugs under the Temporary Authorization for Use (ATU) system, under which lurbinectedin is distributed in that country, and which has led to significant discounts since the beginning of the year.

Raw material sales to our partners of both trabectedin and lurbinectedin in the first nine months significantly increased to €17.8 million, 32% higher compared to the same period last year.

Royalty revenues to September 30th also grew significantly to €35.4m1, an increase of 30% compared to the same period in 2021.

The Group’s non-recurring revenues, which are those from licensing agreements, totaled €22.1 million in the first nine months of the year (vs. €24.4 million at September 2021). These revenues relate to the recognition in income of the amounts received in 2020 as a result of the lurbinectedin license agreement with Jazz Pharmaceuticals ($300 million), which are recognized in the income statement depending on the degree of progress of the contractual commitments.

PharmaMar Group R&D expenditure as of September 30th 2022 was €57.1 million, 23% more than in the same period of the previous year. This increase is due to the progress of developments in the different research areas, where, among other trials, the Company is currently conducting 4 Phase III trials. Of the total amount allocated to R&D, the oncology area is the one with the highest investment, with a total amount of €48.4 million, compared to the €40.1 registered in the same period of the last year.

At September 30th 2022, the PharmaMar Group generated €40.4 million in cash from operating activities. Total debt also decreased by €5.8 million to €39.8 million in the period, which again marks a new 20-year low in debt. Thus, at September 30th 2022, the Group’s cash and cash equivalents position stood at €241 million compared to €212.6 million at the end of the previous year, and net cash totaled €201.2 million, representing a 20% increase since the beginning of the current year.

On September 27th, the Board of Directors of PharmaMar Group decided to discontinue the diagnostics business, which was conducted through its wholly-owned subsidiary Genomica, S.A.U. Consequently, it agreed to initiate the corresponding procedures for the dissolution and liquidation of Genomica, S.A.U., subject to the legally or conventionally applicable procedures for negotiation with the employees.

Genomica closed the past fiscal year 2021 with negative operating and investment cash consumption of €3.2 million, revenues of €5.2 million and a net loss of €3.2 million, which reduced the consolidated operating margin by 4%.

Although the discontinuation of this activity will contribute to improving PharmaMar Group’s operating margin in coming years, at September 30th 2022, the discontinuation process had a negative impact of €4.6 million on the income statement, as a result of adding the necessary provisions for closure costs to the losses from the diagnostics activity in that period.

Despite the costs related to the discontinuation of Genomica, PharmaMar Group reported a net profit of €43.4 million in the first nine months of 2022.

Results conference call for analysts and investors

PharmaMar will host a conference call for analysts and investors on Friday, October 28th, 2022, at 13:00 (CET). The numbers to connect to the teleconference are: +34 91 901 16 44 (from Spain), +1 646 664 1960 (from the US or Canada) or +44 20 3936 2999 (other countries). Participants’ access code: 250969.

The teleconference and the recording of the webcast can be accessed on PharmaMar’s website by visiting the Events Calendar section of the Company’s website at www.pharmamar.com.

Johnson & Johnson has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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