On October 27, 2022 Merck & Co reported its Q3 results for 2022 (Presentation, Merck & Co, OCT 27, 2022, View Source [SID1234622484]).

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On October 27, 2022 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and financial results for the third quarter ending September 30, 2022 (Press release, PTC Therapeutics, OCT 27, 2022, View Source [SID1234622482]).

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"Our mission is to build an enduring biopharmaceutical company that treats diseases with significant unmet medical needs," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "We continue to achieve our ambitious goals for 2022 of generating strong revenue growth while advancing our broad and deep pipeline to continue to fulfill this vision."

Key Corporate Updates:

The Duchenne muscular dystrophy (DMD) franchise continued to show strong growth, with third quarter total net product revenue of $131 million, or 15% year-over-year growth.
Translarna (ataluren) total net product revenue was $77 million, with growth coming from new patients in existing geographies and continued geographic expansion
Emflaza (deflazacort) total net product revenue was $55 million, driven by new patient starts, broader access, continued high compliance and appropriate weight-based dosing
Evrysdi milestone of $50 million from Roche achieved for surpassing annual net sales of $750 million.
Strategic financing with Blackstone to grow pipeline. As part of the partnership, Blackstone provides PTC with an initial $350 million in capital at close, with an option for an additional $650 million in funding.
Key Clinical and Regulatory Updates:

PTC submitted a type II variation to EMA to convert the conditional marketing authorization for Translarna to a standard marketing authorization. PTC expects a CHMP opinion in the first half of 2023.
PTC submitted a meeting request to the FDA to gain clarity on the regulatory pathway for the NDA for Translarna in the US. While the FDA has provided initial written feedback that Study 041 does not provide substantial evidence of effectiveness, PTC is planning follow up discussions with the agency to understand whether the evidence in the ITT population in Study 041 along with confirmatory evidence from other studies could support approval.
PTC held a type C meeting with the FDA to discuss the details of a potential submission package for Upstaza. The FDA asked for additional bioanalytical data in support of comparability between the drug product used in the clinical studies and the commercial drug product. PTC is currently working with the FDA to address this request and expects to submit a BLA for Upstaza in the first half of 2023.
PTC continues to make progress in additional ongoing registration-directed clinical studies:
The APHENITY Phase 3 trial of sepiapterin (PTC923) for PKU, with results anticipated by the end of the fourth quarter
The MIT-E Phase 2/3 vatiquinone trial for mitochondrial disease associated seizures, with results anticipated in the first quarter of 2023
The MOVE-FA Phase 3 vatiquinone trial for Friedreich ataxia, with results anticipated in the second quarter of 2023
Enrollment in the PIVOT-HD Phase 2 trial of PTC518 for Huntington’s disease is active and ongoing at study sites in Europe and Australia. Enrollment in the US is paused as the FDA has requested additional data to support trial conduct. Data from the first 12 weeks of the placebo-controlled trial anticipated in the first half of 2023.
Third Quarter 2022 Financial Highlights:

Total revenues were $217.1 million for the third quarter of 2022, compared to $138.7 million for the third quarter of 2021.
Total revenue includes net product revenue across the commercial portfolio of $134.2 million for the third quarter of 2022, compared to $115.6 million for the third quarter of 2021. Total revenue also includes royalty and collaboration revenue of $82.9 million in the third quarter of 2022, compared to $23.1 million for the third quarter of 2021.
Translarna net product revenues were $76.6 million for the third quarter of 2022, compared to $67.2 million for the third quarter of 2021. These results reflect an increase in net product sales from new patients in existing markets as well as continued geographic expansion.
Emflaza net product revenues were $54.8 million for the third quarter of 2022, compared to $47.1 million for the third quarter of 2021. These results reflect new patient starts, broader access, continued high compliance and appropriate weight-based dosing.
Roche reported Evrysdi 2022 year to date sales of approximately CHF 793 million, resulting in royalty revenue of $32.9 million to PTC in the third quarter of 2022, as compared to $13.1 million for the third quarter of 2021. Also in the third quarter of 2022, PTC recorded a sales milestone of $50 million for the achievement of $750 million in worldwide annual net sales from Evrysdi. This sales milestone was recorded as collaboration revenue.
Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $165.5 million for the third quarter of 2022, compared to $130.8 million for the third quarter of 2021. The increase reflects additional investment in research programs and advancement of the clinical pipeline.
Non-GAAP R&D expenses were $150.4 million for the third quarter of 2022, excluding $15.1 million in non-cash, stock-based compensation expense, compared to $117.8 million for the third quarter of 2021, excluding $13.0 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $80.1 million for the third quarter of 2022, compared to $69.3 million for the third quarter of 2021. The increase reflects our continued investment to support commercial activities, including expanding our commercial portfolio.
Non-GAAP SG&A expenses were $66.5 million for the third quarter of 2022, excluding $13.6 million in non-cash, stock-based compensation expense, compared to $56.4 million for the third quarter of 2021, excluding $12.8 million in non-cash, stock-based compensation expense.
Change in the fair value of deferred and contingent consideration was $5.3 million for the third quarter of 2022, compared to $10.8 million for the third quarter of 2021. The change in fair value of deferred and contingent consideration is related to the fair valuation of potential future consideration to be paid to former equity holders of Agilis Biotherapeutics, Inc. (Agilis) in connection with PTC’s acquisition of Agilis, which closed in August 2018.
Net loss was $109.3 million for the third quarter of 2022, compared to net loss of $133.6 million for the third quarter of 2021.
Cash, cash equivalents, and marketable securities was $288.4 million as of September 30, 2022, compared to $773.4 million as of December 31, 2021.
Shares issued and outstanding as of September 30, 2022, were 71,854,892.
PTC Updates Full Year 2022 Financial Guidance as Follows:

PTC anticipates total revenues for the full year 2022 to be between $710 and $750 million, compared to previous guidance of between $700 and $750 million.
PTC anticipates net product revenues for the DMD franchise for the full year 2022 to be between $490 and $500 million, compared to previous guidance of between $475 and $495 million.
PTC anticipates GAAP R&D and SG&A expenses for the full year 2022 to be between $925 and $965 million, compared to previous guidance of between $915 and $965 million.
PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2022 to be between $810 and $850 million, compared to previous guidance of between $800 and $850 million, both excluding estimated non-cash, stock-based compensation expense of $115 million.
Non-GAAP Financial Measures:

In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management’s opinion, these non-GAAP financial measures are useful to investors and other users of PTC’s financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company’s future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below.

Today’s Conference Call and Webcast Reminder:

To access the call by phone, please click here to register and you will be provided with dial-in details. To avoid delays, we recommend participants dial in to the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investor section of the PTC website at View Source A replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for 30 days following the call.

On October 27, 2022 Seagen Inc. (Nasdaq:SGEN) reported that financial results for the third quarter ended September 30, 2022 (Press release, Seagen, OCT 27, 2022, View Source [SID1234622481]). The Company also highlighted PADCEV (enfortumab vedotin-ejfv), TUKYSA (tucatinib), ADCETRIS (brentuximab vedotin) and TIVDAK (tisotumab vedotin-tftv) commercial and development accomplishments, as well as progress across its deep and diverse oncology pipeline.

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"Seagen delivered strong performance in the third quarter with growth across all sources of revenue, including total product net sales for both the quarter and year-to-date compared to the same periods in 2021," said Roger Dansey, M.D., interim CEO and Chief Medical Officer. "We also made substantial clinical development progress so far this year, reflected in the submission of multiple supplemental regulatory applications towards our goal of broadening the number of patients who could benefit from our medicines, and entered into two important strategic corporate development deals. We look forward to reporting data from across our pipeline throughout the coming year."

PRODUCTS HIGHLIGHTS

PADCEV

Presented Positive Results for the Combination of PADCEV and KEYTRUDA (pembrolizumab) as First-Line Treatment for Advanced Urothelial Cancer at the European Society for Medical Oncology Congress; Submitted Supplemental Biologics License Application (sBLA) to U.S. Food and Drug Administration (FDA): In September 2022, Seagen, Astellas and Merck announced the presentation of data from the phase 1b/2 EV-103 clinical trial (also known as KEYNOTE-869) Cohort K evaluating PADCEV in combination with Merck’s anti-PD-1 therapy KEYTRUDA as first-line treatment in patients with unresectable locally advanced or metastatic urothelial cancer who are ineligible to receive cisplatin-based chemotherapy. The results for the combination demonstrated an encouraging overall response rate of 64.5% and a manageable safety profile. The median duration of response was not reached. The results served as the basis for a sBLA submitted to FDA in October 2022 under the FDA’s Accelerated Approval Program.
TUKYSA

TUKYSA in Combination with Trastuzumab Granted Priority Review by FDA for Previously Treated HER2-Positive Metastatic Colorectal Cancer: In September 2022, FDA accepted for Priority Review the supplemental New Drug Application (sNDA) seeking accelerated approval for TUKYSA in combination with trastuzumab for adult patients with HER2-positive colorectal cancer who have received at least one prior treatment regimen for unresectable or metastatic disease. The sNDA submission is based on the results of the pivotal phase 2 MOUNTAINEER trial and the FDA target action date is January 19, 2023.
ADCETRIS

Submitted to the FDA Overall Survival Data with ADCETRIS Combination for Patients with Previously Untreated Advanced Hodgkin Lymphoma: In September 2022, longer-term follow-up data from the phase 3 ECHELON-1 clinical trial were submitted in a sBLA to FDA for inclusion in the label. The data demonstrated that ADCETRIS in combination with chemotherapy resulted in a 41% reduction in risk of death versus standard of care in patients with previously untreated advanced Hodgkin lymphoma.
NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Hodgkin Lymphoma Updated Elevating ADCETRIS Combination to Category 1, Preferred Recommendation: In September 2022, based on the overall survival benefit of ADCETRIS in combination with chemotherapy that was demonstrated in the ECHELON-1 trial, the NCCN Guidelines were updated elevating the ADCETRIS combination to Category 1, Preferred treatment option for adults with previously untreated Stage III or IV Hodgkin lymphoma with no known neuropathy. Category 1, Preferred is the highest recommendation by NCCN, indicating that based upon high-level evidence, there is uniform NCCN consensus that the intervention is appropriate.
TIVDAK

Entered into Regional Strategic Collaboration with Zai Lab: In September 2022, Seagen announced an exclusive collaboration and license agreement with Zai Lab for the development and commercialization of TIVDAK in mainland China, Hong Kong, Macau, and Taiwan.
PIPELINE PROGRAMS

Entered into Worldwide License Agreement with LAVA Therapeutics: In September 2022,Seagen entered into an agreement with LAVA Therapeutics to develop and commercialize LAVA-1223, a preclinical gamma delta bispecific T-cell engager for EGFR-expressing solid tumors. Seagen received an exclusive global license for LAVA-1223 and has the opportunity to exclusively negotiate rights to apply LAVA’s proprietary GammabodyTM platform on up to two additional tumor targets.
Presenting Data Highlighting Novel Targeted Therapies at Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 37th Annual Meeting: Initial phase 1 clinical data will be presented on SGN-B6A, a novel antibody-drug conjugate (ADC) in development for solid tumors. In addition, Seagen will present preclinical research from several other early-stage programs, including SGN-BB228, an Anticalin-based bispecific antibody. The SITC (Free SITC Whitepaper) Annual Meeting is taking place November 8-12, 2022.

For additional information on Seagen’s pipeline, visit www.seagen.com/science/pipeline.
CORPORATE HIGHLIGHTS

Published Annual Corporate Responsibility Report: In October 2022, Seagen published its second annual Corporate Responsibility Report providing an update on its ESG (environment, social, and governance) efforts, achievements and future commitments. The report is available on the Seagen website at www.seagen.com.
THIRD QUARTER AND NINE-MONTHS 2022 FINANCIAL RESULTS

Revenues: Total revenues for the third quarter and nine months ended September 30, 2022 were $510 million and $1,434 million, respectively, compared to $424 million and $1,145 million for the same periods in 2021. Revenues in the 2022 periods reflected higher net product sales across the Company’s commercial portfolio.

Note: Sum of product sales may not equal total net product sales due to rounding. Percent change reflects actual (unrounded) values. NM = Not meaningful.

Net Product Sales: The increases in net product sales for the third quarter of 2022 compared to the same period in 2021 were driven by continued commercial execution. ADCETRIS growth was related to greater use in frontline advanced Hodgkin lymphoma, increase in diagnosis rates, and favorable pricing dynamics. PADCEV growth was a result of additional eligible patients in the second-line, post-checkpoint maintenance setting for metastatic urothelial cancer. TUKYSA performance reflects competitive dynamics in its current indication. TIVDAK commercialization began in the U.S. following FDA approval in September 2021.
Royalty Revenues: Royalty revenues were primarily driven by sales of ADCETRIS outside the U.S. and Canada by Takeda as well as royalties from sales of Polivy (polatuzumab vedotin) by Roche and Blenrep (belantamab mafodotin) by GSK, which are ADCs that use Seagen technology.
Collaboration and License Agreement Revenues: The increase in collaboration and license agreement revenues was driven by a $30 million upfront license fee from Zai Lab in the third quarter of 2022 related to a regional collaboration for development and commercial for TIVDAK. In addition, the increase reflects clinical milestones and payments from other ADC collaborators, royalty contribution from Astellas’ sales of PADCEV in its territory, as well as higher amounts of drug product supplied to a collaborator for the year-to-date in 2022.
Cost of Sales: Cost of sales for the third quarter and year-to-date in 2022 were $108 million and $302 million, respectively, compared to $83 million and $225 million for the same periods in 2021. The increases were primarily driven by higher sales of our medicines and the related gross profit share amounts owed to collaboration partners, which were $71 million and $189 million in the third quarter and year-to-date in 2022, respectively, compared to $45 million and $116 million for the same periods in 2021. Cost of sales also reflects amortization of TUKYSA acquired in-process technology costs, third-party royalties owed for PADCEV and TUKYSA net product sales, and cost of products sold.

Research and Development (R&D) Expenses: R&D expenses for the third quarter and year-to-date in 2022 were $385 million and $987 million, respectively, compared to $459 million and $924 million for the same periods in 2021 reflecting continued investment in clinical development of the Company’s approved drugs and pipeline programs, and a $50 million upfront fee to LAVA Therapeutics in the third quarter of 2022. The 2021 periods included the $200 million upfront license payment owed to RemeGen related to our agreement to obtain exclusive rights to disitamab vedotin.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses for the third quarter and year-to-date in 2022 were $210 million and $605 million, respectively, compared to $180 million and $505 million for the same periods in 2021. The increases in 2022 were driven by ongoing commercialization efforts, costs from legal proceedings with Daiichi Sankyo, and other corporate activities.

Non-cash, share-based compensation expense for the nine months ended September 30, 2022 was $157 million, compared to $121 million for the same period in 2021.

Net Loss: Net loss for the third quarter of 2022 was $191 million, or $1.03 per diluted share, and net loss for the year-to-date in 2022 was $462 million, or $2.51 per diluted share.

Net loss for the third quarter of 2021 was $294 million, or $1.61 per diluted share, and net loss for the year-to-date in 2021 was $500 million, or $2.75 per diluted share.

Cash and Investments: As of September 30, 2022, Seagen had $1.8 billion in cash and investments.

2022 FINANCIAL OUTLOOK

Seagen anticipates 2022 revenues, operating expenses and other costs to be in the ranges shown in the table below. Updates reflect changes in expectations for net product sales, royalties, and collaboration and license agreement revenues, as well as R&D expenses, and SG&A expenses.

Conference Call Details

Seagen management will host a conference call and webcast with supporting slides to discuss its third quarter 2022 financial results and provide an update on business activities. The event will be held today at 1:30 p.m. Pacific Time (PT); 4:30 p.m. Eastern Time (ET). The live event will be simultaneously webcast and available for replay from the Seagen website at investor.seagen.com. Investors may also participate in the conference call by calling 844-763-8274 (domestic) or 412-717-9224 (international). The conference ID is 10171976. Supporting slides are available on the Seagen website at investor.seagen.com under the Investors section. A webcast replay will be archived on the Company’s website investor.seagen.com, under the Investors section.

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.