On April 13, 2022 Versant Ventures reported the debut of Cimeio Therapeutics, a biotechnology company developing a novel approach to cell therapies. Versant has made a $50 million Series A commitment to Cimeio, which is the most recent start-up to emerge from the firm’s Ridgeline Discovery Engine in Basel, Switzerland (Press release, Versant Ventures, APR 13, 2022, View Source [SID1234612132]).

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Cimeio’s platform has the potential to transform the treatment of patients with rare genetic diseases, hematologic malignancies and autoimmune disorders. The company’s initial focus is on a novel approach to hematopoietic stem cell (HSC) transplants and adoptive cell therapy (ACT).

HSC transplants are the only curative treatments for certain debilitating and life-threatening diseases, but many patients are ineligible due to the intensive chemotherapy and radiation conditioning required. While targeted therapeutics have recently emerged as alternatives to harsh conditioning agents, these have fallen short due to the absence of sufficiently selective targets. Furthermore, there are few options for salvaging unsuccessful transplants or for dealing with residual or recurring disease.

Cimeio seeks to transform HSC transplant and ACT eligibility and outcomes with its cell-shielding technology and precisely paired immunotherapies. The company’s proprietary immunotherapies deplete diseased cells, while its cell-shielding technology protects healthy transplanted cells and allows them to engraft. Because the transplanted cells are shielded, the immunotherapy can continue to be safely administered post-transplant to boost engraftment or to treat minimal residual disease.

"Our Shielded-Cell & Immunotherapy Pairs represent a fundamentally new approach to cellular therapy," said Cimeio CEO Thomas Fuchs. "We believe our technology platform has the potential to significantly improve HSC transplant, and will one day allow it to be given as an outpatient procedure in some circumstances."

"Making cell therapies effective and practical for large numbers of patients is an important element of Versant’s company creation activities, and we are confident that Cimeio is well-positioned to use its powerful platform to generate a pipeline of cellular and paired immunotherapy candidates for a range of diseases," added Alex Mayweg, Ph.D., Managing Director at Versant and a Cimeio board member.

Cimeio Technology Platform

Cimeio’s shielding technology was discovered and developed in the labs of founder Lukas Jeker, M.D., Ph.D., Professor at the Department of Biomedicine, University of Basel, Head of Experimental Transplantation Immunology & Nephrology at the Basel University Hospital, and Senior Vice President of Gene Editing at Cimeio.

"We were able to specifically edit a cell surface receptor in a way that completely prevented antibody binding while keeping the receptor functional. This type of epitope editing could allow the shielding of any cell surface receptor, which gives our technology much broader application than removing a target entirely," said Dr. Jeker.

Cimeio uses gene editing tools to insert novel protein variants into HSCs or other types of cells, allowing the cells to maintain their function while making them resistant to depletion by the paired immunotherapy. Cimeio’s platform has effectively shielded cells from depletion mediated by antibodies, T-cell engagers, ADCs, and CAR-T cells in preclinical studies. The company is advancing its first programs towards clinical development in 2023.

Leadership and Operating Plans

Cimeio has built a leadership team of disease area and cell therapy veterans, and has assembled a scientific advisory board of gene editing and HSC transplant experts.

Management

Thomas Fuchs, CEO. Mr. Fuchs joined Cimeio from Genentech/Roche where he led its Hematology Franchise and was responsible for the portfolio strategy and life cycle management for the disease area.
Stefanie Urlinger, Ph.D., SVP Biology. Dr. Urlinger is a protein engineering expert who has led the discovery of dozens of antibodies during her time at Morphosys and iOmx, and works closely with academic founder Dr. Jeker.
Lukas Jeker, M.D., Ph.D., SVP Gene Editing. Dr. Jeker is a world leader in the field of applied genome editing and Professor of Experimental Transplantation Immunology & Nephrology at the University of Basel. Earlier in his career he conducted research in the lab of Jeff Bluestone at the University of California San Francisco as a post-doc and later an Assistant Adjunct Professor.
Daniel Stark, Ph.D., Chief Manufacturing Officer. Dr. Stark formerly was head of Manufacturing Science and Technology (MSAT) at Novartis Cell & Gene Therapies, where he oversaw CMC for Kymriah and Novartis’ pipeline of cellular therapies.
Thomas Winkler, M.D., CMO. Dr. Winkler is a physician-scientist who spent 11 years conducting basic and clinical research related to various benign and malignant hematological diseases, stem cell biology, and regenerative medicine at the National Institute of Health in Bethesda, MD, and later led early stage and pivotal studies at Agios and AstraZeneca.
Tristan Imbert, CFO. Mr. Imbert previously was Senior Vice President and CFO of Novartis Gene Therapies, where he oversaw the scale up and commercialization of Zolgensma.
SAB

Chairman Fyodor Urnov, Ph.D., is Scientific Director at the Innovative Genomics Institute and Professor at the University of California, Berkeley. Dr. Urnov’s research is focused on development and advancement to the clinic of novel approaches to treat human disease using CRISPR-based genome and epigenome editing.
Jeff Bluestone, Ph.D., President and CEO of Sonoma BioTherapeutics. Dr. Bluestone is one of the leading immunologists in the field of T cell activation and immune tolerance research that has led to the development of multiple immunotherapies.
Toni Cathomen, Ph.D., Director at the Institute for Transfusion Medicine and Gene Therapy and a Professor at the University of Freiburg. Dr. Cathomen’s research is focused on improving CRISPR-Cas platforms for therapeutic applications in human stem cells.
Corey Cutler, M.D., M.P.H., Medical Director of the Stem Cell Transplantation Program at the Dana Farber Cancer Institute and Associate Professor of Medicine at Harvard Medical School. Dr. Cutler’s research is focused on developing novel methods of acute and chronic graft-versus-host disease (GVHD) prophylaxis and therapy, transplant for the myelodysplastic syndromes, and decision theory in stem cell transplantation.
Suneet Agarwal, M.D., Ph.D., Co-Program Leader for the Stem Cell Transplant Center at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and Associate Professor of Pediatrics at the Harvard Medical School. Dr. Agarwal’s research is focused on developing innovative therapies using novel medications or a patient’s own cells to treat genetic blood disorders.
Cimeio is located in Boston, MA, and Basel, Switzerland, and is rapidly expanding its research and CMC teams to advance its three lead programs and expand its portfolio. Cimeio also has active research collaborations with Dr. Jeker’s lab, with Matt Porteus, M.D., Ph.D., at Stanford University, and with Dr. Cathomen’s research group.

On April 13, 2022 Theratechnologies Inc. (Theratechnologies, or Company) (TSX: TH) (NASDAQ: THTX), a biopharmaceutical company focused on the development and commercialization of innovative therapies, reported business highlights and financial results for the first quarter of fiscal year 2022 ended February 28, 2022 (Q1 2022) (Press release, Theratechnologies, APR 13, 2022, View Source [SID1234612127]).

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"Building on the strong momentum of the second half of 2021, our performance continues through to the new year, said Paul Lévesque, President and Chief Executive Officer. "To this end, we are making good headway across our execution goals as evidenced by the strong sales growth in Q1 2022, our historically weakest quarter. In EGRIFTA SV, we achieved 35% sales growth, while overall blended commercial sales grew in the double digits by 20%."

"The development of TH1902, our lead investigational peptide drug conjugate linked to docetaxel, a well-established and well-characterized cytotoxic agent used in the treatment of cancer, is also progressing well. We anticipate completing the 300mg/m2 dosing regimen for 6 patients with TH1902 before the end of April 2022, and we are now nearing the conclusion of this stage of the trial and establishing the recommended Phase 2 dose (RP2D). This is the last step prior to initiating the program’s larger basket study trial. Additionally, we recently presented new TH1902 in vivo preclinical data at the AACR (Free AACR Whitepaper) Annual Meeting. Theratechnologies also continues its partner out-licensing discussions for TH1902’s development rights in Greater China," concluded, Mr. Lévesque.

Recent Business Highlights

Pipeline Updates

TH1902 Study Update: Enrollment in the Phase 1 trial of TH1902 has picked up momentum in the past few weeks, and we now anticipate that all 6 patients required for the 300mg/m2 dosing level will be enrolled before the end of April. This dose is the equivalent to approximately 1.5 times the indicated therapeutic dose of docetaxel. The targeted delivery of TH1902, along with the rapid internalization of the drug in cancer cells could enable the accumulation of 7.5 to 10 times more cytotoxic agent in cancer cells than when administered alone. If the absence of dose limiting toxicities (DLT) is confirmed, this dose will become the recommended Phase 2 dose (RP2D). As previously discussed, once the RP2D is established, initiation of enrollment of the larger open label basket trial will begin immediately. The basket trial will further assess the safety and tolerability of TH1902. The preliminary anti-tumor activity of TH1902 will be evaluated for all patients as per the response evaluation criteria in solid tumors.

Enrollment for the larger trial is expected to begin in this first half of 2022. An amendment to the Phase 1 protocol was submitted to the FDA to include the following solid tumor types: HR+ Breast Cancer, Triple Negative Breast Cancer, Ovarian Cancer, Endometrial Cancer, Melanoma (10 patients per arm) was submitted. In addition, one arm will be added to include Thyroid, Small Cell Lung, Prostate and potential other high Sortilin expressing cancers (15 patients in total). The original trial design consisted of 40 patients across a selection of solid tumors, including colorectal and pancreatic cancers. The plan is now to enroll a total of approximately 70 patients in the basket trial to evaluate the potential anti-tumor activity of TH1902.

To date, the Company has received and responded to the questions raised by the FDA and the Company does not expect to receive any additional questions before the April 15, 2022 deadline date by which time the amendments to the protocol will be deemed accepted and ready to be implemented.
TH1902 China Out-licensing and Partnership Strategy: Out-licensing development and commercialization rights for TH1902 in Greater China continues and are ongoing with a number of different pharmaceutical and biotech companies.
Scientific Poster Presentations: The Company presented three posters at the recently attended AACR (Free AACR Whitepaper) annual meeting, including new in vivo TH1902 preclinical data demonstrating tumor growth inhibition of human cancer stem-like cells (CD133+) in both triple-negative breast and ovarian cancers.
F8 sBLA filing: As previously announced, our intention was to file a supplemental Biologic License Application ("sBLA") for the F8 formulation ("F8") by the end of the first quarter of calendar 2022. In contrast to EGRIFTA SV which is reconstituted daily with sterile water for injection, the F8 formulation requires bacteriostatic water for injection ("BWFI"), since the reconstituted product is used for seven daily injections. We were recently informed by the sole global supplier of BWFI that its plant was recently inspected by the FDA, and that it was required to make modifications before being able to resume manufacturing and shipment of its BWFI. Although we believe a return to supply is planned for the fourth quarter of 2022, there is currently no firm timeline for reinitiating shipments, and, as such, this will cause a delay in the potential launch of the F8 formulation. Consequently, we have decided to delay the filing of the sBLA for the F8 formulation until we have greater clarity on the supply issues. As a result of this uncertainty related to the availability of the F8 formulation of tesamorelin, and since the dosing of patients in Phase 3 trial in non-alcoholic steatohepatitis ("NASH") is dependant on the availability of the F8, we have also decided to pause any external activities related to the planning of the trial until there is more clarity on the availability of BWFI. We plan on keeping investors informed as the supply of BWFI becomes more certain.

This does not affect the supply of EGRIFTA SV since this formulation does not require BWFI for reconstitution.
Commercial and Medical Affairs Updates

Strengthening of US Commercial and Medical Affairs Capabilities: In March 2022, Theratechnologies initiated the full deployment of its own internal field force as pandemic restrictions continue to abate, enabling increased physician engagement. Strong momentum created in the second half of 2021 provided the major impetus for this decision, which should increase employee engagement, reduce turnover, and allow recruitment of top-tier talent for our field force. Onboarding of all internal commercial and medical field force will be fully completed by the end of April, 2022.
Trogarzo Lifecycle Management: A sBLA was filed with the U.S. Food and Drug Administration ("FDA") in the fourth quarter of 2021 for the Company’s Intravenous ("IV") Push mode of administration of Trogarzo for the treatment of human immunodeficiency virus type 1 (HIV-1). We are pleased to announce that the FDA has accepted our filing and has provided a target action date of October 3, 2022 in accordance with the Prescription Drug User Fee Act (PDUFA). Theratechnologies and TaiMed are also evaluating an intramuscular (IM) mode of administration for Trogarzo within the TMB-302 study. Patient enrollment is progressing well, and we expect full enrollment to be achieved in the coming weeks, enabling completion of the study in the second half of 2022.
2022 Revenue Guidance
Theratechnologies affirms fiscal 2022 revenue to be in the range of $79 million and $84 million for full fiscal 2022, or growth of the commercial portfolio to be in the range of 13% and 20% as compared to the 2021 fiscal year.

First Quarter Fiscal 2022 Financial Results

The financial results presented in this press release are taken from the Company’s Management’s Discussion and Analysis (MD&A) and interim consolidated financial statements (Interim Financial Statements) for the three-month period ended February 28, 2022 (First Quarter Fiscal 2022) which have been prepared in accordance with International Financial Reporting Standards (IFRS) as issued by the International Accounting Standards Board (IASB). The MD&A and the Interim Financial Statements can be found at www.sedar.com, on EDGAR at www.sec.gov and at www.theratech.com. Unless specified otherwise, all amounts in this press release are in U.S. dollars and all capitalized terms have the meaning ascribed thereto in our MD&A.

Revenue
Consolidated revenue for the three-month period ended February 28, 2022 was $18,557,000 compared to $15,430,000 for the same period ended February 28, 2021.

For the first quarter of fiscal 2022, net sales of EGRIFTA SV reached $11,704,000 compared to $8,688,000 in the first quarter of the prior year, representing an increase of 34.7% over the first quarter of 2021, due to the combined effect of a higher number of units sold and higher net selling price.

In the first quarter of fiscal 2022, Trogarzo net sales amounted to $6,853,000 compared to $6,742,000 for the same quarter of 2021, representing an increase of 1.6%. While unit sales were higher in both North America and Europe, revenue growth was impacted by greater rebates in Europe.

Cost of Sales
For the three months ended February 28, 2022, cost of sales increased to $6,099,000 from $5,411,000 in the same quarter in fiscal 2021, primarily due to the higher cost of goods sold. Cost of goods sold was $4,878,000 in the first quarter of 2022 compared to $4,190,000 for the same quarter the previous year. The increase in cost of goods sold was mainly due to higher sales. Cost of sales also included the amortization of the other asset of $1,221,000 in both Q1 fiscal 2022 and Q1 fiscal 2021.

R&D Expenses
R&D expenses amounted to $8,003,000 in the three-month period ended February 28, 2022 compared to $4,883,000 for the same period in 2021. The increase was largely due to higher spending in our oncology programs, increased spending in medical and patient education, as well as increased medical affairs spending in Europe.

Selling Expenses
Selling expenses amounted to $7,807,000 for the first quarter of 2022 compared to $6,158,000 for the same three-month period last year, reflecting the addition of key hires in North America and Europe, greater commercialization activities in both territories.

The amortization of the intangible asset value for the EGRIFTA and Trogarzo commercialization rights is also included in selling and market development expenses. As such, we recorded an expense of $795,000 for both the first quarter of fiscal 2022 and 2021.

General and Administrative Expenses
General and administrative expenses amounted to $4,368,000 for the three months ended February 28, 2022 compared to $3,562,000 for the first quarter of 2021. The increase in general and administrative expenses was mainly associated with an overall increase in business activities and increased activity in Europe.

Net Finance Costs
Net finance costs for the three months ended February 28, 2022 were $1,285,000 compared to $1,332,000 for the comparable period of 2021. Net finance costs in the first quarter of 2022 and 2021 included interest of $802,000 on the senior convertible notes issued in June 2018.

Net finance costs also included accretion expense of $517,000 in the first quarter of 2022, compared to $581,000 for the comparable period in 2021.

Net Loss
Given the increase in revenue and the increased expenses for the three months ended February 28, 2022, net loss for the period was $9,032,000, compared to $5,922,000 for the same period last year.

Liquidity and Financial Position
We ended the first quarter of fiscal 2022 with $34,283,000 in cash, bonds and money market funds.

During the first quarter of fiscal 2021, the Company completed a public offering for the sale and issuance of 16,727,900 units of the Company for a gross cash consideration of $46,002,000 including the full exercise of the over-allotment option. Share issue costs amounted to $3,385,000 resulting in net proceeds of $42,617,000.

Our current cash, bond and money market funds will be sufficient to fund the Company’s operations for the next twelve months. We are currently exploring alternatives to redeem the senior convertible notes issued in June 2018, which become due in June 2023.

For the three-month period ended February 28, 2022, operating activities used cash of $4,174,000 compared to $1,896,000 in the comparable period of fiscal 2021, primarily due to the increased loss in 2022.

In the first quarter of fiscal 2022, changes in operating assets and liabilities had a positive impact on cash flow of $69,000 (2021-negative impact of $3,332,000). These changes included a negative impact from higher accounts receivable, a decrease in accounts payables and accrued liabilities, and were offset by positive impacts from lower inventories and lower prepaid expenses and deposits.

Conference Call Details
A conference call and webcast will be held on April 13, 2022 at 8:30 a.m. (ET) to discuss the first quarter fiscal 2022 results and recent business highlights. The call will be hosted by Paul Lévesque, President and Chief Executive Officer of Theratechnologies, and other members of the management team.

The conference call can be accessed by dialing 1-844-400-1697 (toll free) or 1-703-736-7400 (International). The conference call will also be accessible via webcast here. An audio replay of the conference call will be available on the same day starting at 11:30 a.m. (ET) until April 20, 2022, by dialing 1-855-859-2056 (North America) or 1-404-537-3406 (International) and by entering the access code: 7843697. An archived webcast will also be available on the Company’s Investor Relations website under ‘Past Events’.

On April 13, 2022 Sonnet BioTherapeutics Holdings, Inc., (NASDAQ:SONN) a clinical-stage company developing targeted immunotherapeutic drugs, reported that dosing has been initiated in a Phase 1 clinical trial of SON-1010 (IL12-FHAB) in adult patients with advanced solid tumors (Press release, Sonnet BioTherapeutics, APR 13, 2022, View Source [SID1234612126]).

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SON-1010 is a proprietary version of human Interleukin 12 (IL-12), configured using Sonnet’s Fully Human Albumin Binding (FHAB) platform. The FHAB technology targets tumor and lymphatic tissue, providing a mechanism for dose sparing and an opportunity to improve the safety and efficacy profile of not only IL-12, but a variety of potent immunomodulators.

"We are excited to have dosed the first patient in our trial and to have initiated this novel approach to enhance the safety of cytokine immunotherapy," said Richard Kenney, M.D., Sonnet’s Chief Medical Officer. "Cytokines have shown great promise in animal models of cancer treatment for several decades, yet the developmental progress in human trials has typically been frustrated by toxicity before the therapeutic dose can be reached. Targeting the tumor by linking an albumin-binding domain, which also extends the cytokine half-life in the body, may be the key to inducing a successful local immune response in the tumor microenvironment."

Interleukin 12 can orchestrate a robust immune response to many cancers and pathogens. Given the types of proteins induced, non-small cell lung cancer, melanoma, head and neck cancer, sarcoma and several gynecological cancers are particularly relevant for this approach.

"As Sonnet’s first FHAB candidate to be dosed in a patient, this SON-1010 milestone represents a significant step forward in our full-spectrum approach to immunotherapy," said Pankaj Mohan, Ph.D., Sonnet Founder and Chief Executive Officer. Dr. Sant Chawla, the Principal Investigator at Sarcoma Oncology Center added, "This Phase 1 clinical trial will carefully assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of SON-1010. Despite recent progress in immunotherapy, there continues to be a large unmet medical need in cancer. We are very pleased to be part of this study, as our clinic treats patients with several different types of tumors that could benefit from this approach."

About the SB101 Phase 1 Trial

This first-in-human study is primarily designed to evaluate the safety of multiple ascending doses of SON-1010 and will be conducted at several sites across the United States. While the optimal dose is unknown at this stage, the potential to target tumors, the extended PK mechanism, and our preclinical data suggest the therapeutic dose may be lower compared to native human IL-12. The study, utilizing a standard 3+3 oncology design in at least five cohorts, should establish the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) using monthly subcutaneous injections of SON-1010. The primary endpoint explores the safety and tolerability of SON-1010, with key secondary endpoints intended to measure PK, PD, immunogenicity and anti-tumor activity. This study will form the basis for potential combinations with other types of immunotherapies and the future development of bispecific candidates using the FHAB platform.

On April 13, 2022 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported that the company will participate in investor meetings at the Kempen Life Sciences Conference being held in Amsterdam, April 20 – 21, 2022 (Press release, Hookipa Biotech, APR 13, 2022, View Source [SID1234612125]).

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On April 13, 2022 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday, April 27th, at 4:30 p.m. ET to discuss first quarter 2022 financial results and provide a general business update (Press release, BioMarin, APR 13, 2022, View Source,-April-27,-at-4-30pm-ET [SID1234612124]).

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Interested parties may access a live audio webcast of the conference call via the investor section of the BioMarin website, www.biomarin.com. A replay of the call will be archived on the site for one week following the call.