Triumvira Immunologics Announces First Patient Dosed in Phase II of TACTIC-2 Cell Therapy Trial for the Treatment of HER2+ Gastric and GEJ Cancers

On October 26, 2023 Triumvira Immunologics, a clinical-stage company developing novel, targeted autologous and allogeneic T cell therapeutics that co-opt the natural biology of T cells to treat patients with solid tumors, reported that the first patient has been dosed in Phase II of its Phase I/II study TACTIC-2 (NCT04727151) investigating the safety and efficacy of autologous TAC-T cell lead asset, TAC01-HER2, in targeting HER2 in relapsed or refractory gastric and gastroesophageal junction (GEJ) tumors (Press release, Triumvira Immunologics, OCT 26, 2023, View Source [SID1234636387]). TAC01-HER2 is a novel cell therapy based on genetically engineered autologous T cells expressing a T-cell Antigen Coupler (TAC) that recognizes human epidermal growth factor receptor 2 (HER2).

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"This marks a significant milestone for our company, building upon the determination of the recommended Phase II dose, identifying gastric and gastroesophageal cancer patients as targets for the Phase II registration supporting study and the positive benefit we observed during the Phase I part of TACTIC-2," said Deyaa Adib, M.D., Chief Medical Officer of Triumvira Immunologics. "Despite considerable advances in the oncology field, HER2-positive gastric and gastro-esophageal cancers remain difficult to treat, and new therapeutic options are urgently needed especially in later treatment lines in a growing patient segment. Our TAC technology offers a novel approach that works by leveraging the natural signaling pathways of endogenous TCRs and modifying T cells into TAC T cells with demonstrated success in the treatment of these tumors. We are committed to providing clinically meaningful therapeutic benefits to this patient population with high unmet medical needs."

TACTIC-2 is an open-label, multicenter Phase I/II study that aims to establish safety, maximum tolerated dose (MTD) or recommended phase 2 dose (RP2D), pharmacokinetic profile and efficacy of TAC01-HER2 as a monotherapy, and in combination with pembrolizumab, in subjects with HER2 positive gastric and gastroesophageal adenocarcinoma.

Theratechnologies Announces Pricing of US$25 Million Public Offering of Common Shares and Concurrent Private Placement

On October 26, 2023 Theratechnologies Inc. ("Theratechnologies" or the "Company") (Nasdaq: THTX; TSX: TH), a biopharmaceutical company focused on the development and commercialization of innovative therapies, reported that it has priced its previously announced public offering (the "Public Offering") of 12,500,000 common shares of the Company (the "Common Shares") at a public offering price of US$1.00 per Common Share (the "Offering Price") (Press release, Theratechnologies, OCT 26, 2023, View Source [SID1234636386]). The gross proceeds of the Public Offering are expected to be approximately US$12,500,000, before deducting the underwriting discounts and commissions and other estimated offering expenses. The Company has also granted the underwriter a 30-day option (the "Option") to purchase up to 1,875,000 Common Shares at the Offering Price, less underwriting discounts and commissions.

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Cantor Fitzgerald & Co. is acting as sole bookrunner for the Public Offering.

In connection with the Public Offering, the Company entered into a subscription agreement with Investissement Québec for a concurrent private placement (the "Concurrent Private Placement") of 9,118,184 Common Shares and 3,381,816 fully-funded, non-voting subscription receipts, exchangeable into Common Shares on a one-for-one basis (the "Exchangeable Subscription Receipts") in lieu of Common Shares, in each case, at the Offering Price, for US$12,500,000 aggregate gross proceeds, less a capital commitment fee of 1.5% payable to Investissement Québec. The component of the Concurrent Private Placement in the form of Exchangeable Subscription Receipts is designed to ensure that, following completion of the Public Offering and the Concurrent Private Placement, Investissement Québec does not have beneficial ownership or control over more than 19.9% of the issued and outstanding Common Shares and therefore is not a "control person" within applicable Canadian securities laws.

The Company will also enter into an investor rights agreement, pursuant to which Investissement Québec will be entitled to nominate one director to the Company’s board of directors for as long as it holds 50% of the Common Shares purchased pursuant to the Concurrent Private Placement. Copies of the subscription agreement, the exchangeable receipt agreement setting forth the terms and condition of the Exchangeable Subscription Receipts and the investor rights agreement, when available, will be filed on SEDAR+ at www.sedarplus.ca. Summaries of the subscription agreement and the exchangeable receipt agreement and a copy of the investor rights agreement, when available, will be filed on EDGAR at www.sec.gov.

Assuming completion of the Public Offering and the Concurrent Private Placement for US$12,500,000 and US$12,500,000 aggregate gross proceeds, respectively, but assuming that the Option is not exercised, Investissement Québec will beneficially own approximately 19.9% (25.4% if the Exchangeable Subscription Receipts were to be exchanged into Common Shares) of the issued and outstanding Common Shares as of the date of closing. If the Option is exercised in full, Investissement Québec will beneficially own approximately 19.1% (24.5% if the Exchangeable Subscription Receipts were to be exchanged into Common Shares) of the issued and outstanding Common Shares as of the date of closing.

A preliminary prospectus supplement (the "Prospectus Supplement") to the Company’s short form base shelf prospectus dated December 14, 2021 (the "Base Shelf Prospectus") was filed with the securities regulatory authorities in each of the provinces of Canada as well as with the U.S. Securities and Exchange Commission (the "SEC") as part of its registration statement on Form F-10 (the "Registration Statement") under the U.S.-Canada multijurisdictional disclosure system ("MJDS"). The Public Offering will be made in Canada only pursuant to the Prospectus Supplement and Base Shelf Prospectus and in the United States only pursuant to the Registration Statement, containing the Prospectus Supplement and the Base Shelf Prospectus, filed with the SEC under the MJDS. Copies of the Prospectus Supplement and the Base Shelf Prospectus are available on SEDAR+ at www.sedarplus.ca and on EDGAR at www.sec.gov, and a copy of the Registration Statement is available on EDGAR at www.sec.gov. Copies may also be obtained from Cantor Fitzgerald & Co., Attention: Capital Markets, 110 East 59th Street, 6th Floor, New York, New York 10022, or by e-mail at [email protected].

Completion of the Public Offering and Concurrent Private Placement is expected to occur by October 31, 2023, subject to customary closing conditions, including the listing of the Common Shares, and the Common Shares underlying the Exchangeable Subscription Receipts, on the Toronto Stock Exchange and the submission of notice to the Nasdaq Capital Market. The closing of the Public Offering and the Concurrent Private Placement are conditional upon each other.

The Company intends to use the net proceeds of the Public Offering and the Concurrent Private Placement for general corporate purposes, which may include working capital, general and administrative expenses, commercialization expenses, repayment of outstanding debt under its credit facility with certain funds and accounts for which Marathon Asset Management, L.P. acts as investment manager, and potential acquisitions or in-licensing of commercial products.

Prospective investors should read the Prospectus Supplement, Base Shelf Prospectus and Registration Statement before making an investment decision.

No securities regulatory authority has either approved or disapproved the contents of this news release. This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any province, state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such province, state or jurisdiction.

QIAGEN and Myriad Genetics partner to advance companion diagnostics development for cancer

On October 26, 2023 QIAGEN (NYSE: QGEN; Frankfurt Prime Standard: QIA) and Myriad Genetics (NASDAQ: MYGN) reported a new master collaboration agreement to develop companion diagnostic tests in the field of cancer (Press release, Qiagen, OCT 26, 2023, View Source [SID1234636385]).

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The partnership aims to deliver innovative services and products to pharmaceutical companies, enabling the development and commercialization of proprietary cancer tests for the U.S. clinical market, and providing distributable companion diagnostic test kits for the global market.

The alliance between QIAGEN and Myriad brings together the respective strengths of each partner. Myriad leverages its CLIA-certified, CAP-accredited lab platform, assay development expertise and robust commercial infrastructure for clinical sample testing. QIAGEN will provide Sample to Insight solutions, including sample preparation, its PCR, digital PCR (using the QIAcuity system), QIAseq next-generation sequencing (NGS) technologies, instruments, as well as the QIAGEN Digital Insights portfolio of bioinformatics. Additionally, QIAGEN contributes GMP-certified product manufacturing capabilities and globally established commercial channels. The partnership also capitalizes on the combined FDA and worldwide regulatory expertise of both companies, providing seamless compliance and integration in clinical and companion diagnostic applications.

"We are pleased to collaborate with Myriad, combining their expertise in complex and proprietary lab-developed tests with our proficiency in distributable kit-based solutions, to offer a comprehensive global companion diagnostic approach. Our partnership aims to accelerate the advancement of cancer companion diagnostics, making them accessible to pharma partners worldwide," said Jonathan Arnold, Vice President, Head of Translational Science and Precision Diagnostics at QIAGEN. "Together, we share the common goal of improving patient care and guiding treatment decisions in oncology through our combined expertise and capabilities."

Our partnership with QIAGEN creates a comprehensive companion diagnostic development and commercialization solution for our pharma partners that will support the advancement of cancer care and personalized treatment decisions for patients worldwide," said Paul Bartel, Senior Vice President of Companion Diagnostics, Myriad Genetics. "As we grow Myriad’s presence globally in the companion diagnostics space, we are excited to partner with QIAGEN to increase access to these solutions, and help more patients and providers make effective therapeutic choices."

The initial project focus for the two companies will involve collaborating with pharma partners to develop assays utilizing next-generation sequencing workflows or QIAGEN’s digital PCR platform, QIAcuity.

Future projects are under consideration to explore advancing and kitting next-generation detection of measurable residual disease (MRD), which involves using circulating tumor DNA (ctDNA) assays to detect cancer that may remain in a patient’s body following treatment, and homologous recombination deficiency (HRD), a condition where cells are unable to repair damaged DNA effectively, potentially increasing the survival ability for cancers treated with chemotherapy.

MRD is a highly complex workflow, and both companies plan to collaborate in assessing the feasibility of integrating digital PCR to produce a kitted and standardized solution for diagnostic laboratories.

QIAGEN has master collaboration agreements to develop and commercialize companion diagnostics with more than 30 global pharma and biotech companies – a deep pipeline that will advance precision medicines in diverse disease indications, tailoring a patient’s treatment to the genetic profile identified by companion diagnostics testing. Myriad has provided testing support for hundreds of clinical trials, has obtained 10 companion diagnostic approvals from the FDA and PMDA, and anticipates that the QIAGEN partnership will drive the expansion of the Myriad portfolio of oncology products.

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 [SID1234636384]).

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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.
oTranslarna (ataluren) quarterly net product revenue was $69 million, with new patients in existing geographies and continued geographic expansion.
oEmflaza (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.

Promontory Therapeutics to Present Data on PT-112 Mechanism of Action at the Society for Immunotherapy of Cancer’s 38th Annual Meeting

On October 26, 2023 Promontory Therapeutics Inc., a clinical stage biotech company advancing immunogenic small molecule approaches in oncology, reported that it will present a poster demonstrating the molecular mechanism of its lead therapeutic candidate, PT-112, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 38th Annual Meeting (Press release, Promontory Therapeutics, OCT 26, 2023, View Source [SID1234636383]). SITC (Free SITC Whitepaper) is taking place November 1-5, 2023 in San Diego.

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PT-112 is the subject of ongoing Phase 2 clinical trials for metastatic castrate-resistant prostate cancer and thymic epithelial tumors, and a completed Phase 2a trial in non-small cell lung cancer.

Poster Session Details
Title: Molecular mechanisms of immunogenic cell death driven by PT-112
Abstract Number: 1106
Primary Category: Immune-Stimulants and Immune Modulators
Session Date/Time: Saturday, Nov. 4, 2023, 9:00 am – 8:30 pm PT
Session Location: Exhibit Halls A and B1 – San Diego Convention Center

For more information about Promontory Therapeutics and PT-112, visit www.PromontoryTx.com.