On April 28, 2023 AC Immune SA (NASDAQ: ACIU), a clinical-stage biopharmaceutical company pioneering precision medicine for neurodegenerative diseases, reported results for the first quarter ended March 31, 2023, and provided a corporate update (Press release, AC Immune, APR 28, 2023, View Source [SID1234630690]).

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Dr. Andrea Pfeifer, CEO of AC Immune SA, commented: "Recent accomplishments reaffirm our industry-leading position in developing vaccines and diagnostics for neurodegenerative diseases supporting the transition to targeted treatment and disease prevention. Our maturing pipeline’s substantial breadth offers multiple milestones in the near- and mid-term and we have the strong cash position needed to deliver them."

"We are delighted to see our Tau positron emission tomography (PET) tracer PI-2620 progress into the pivotal Phase 3 ADvance trial, with our partner, LMI. Similarly, we are excited by the initial positive data from the Phase 1b/2 ABATE trial of ACI-24.060 and the expansion as planned, to include individuals with Down syndrome. The trial is now advancing towards additional interim safety and immunogenicity data in both indications this year, as well as PET imaging analyses in H1 2024 that will evaluate ACI-24.060’s impact on amyloid plaques. With amyloid PET increasingly recognized as a surrogate marker of efficacy in AD, these analyses represent a significant potential de-risking event that could rapidly propel our wholly-owned vaccine towards a pivotal program."

Q1 2023 and Subsequent Highlights

ACI-24.060 positive initial interim safety and immunogenicity data were reported from the first, low dose Alzheimer’s disease (AD) cohort of the Phase 1b/2 ABATE trial of AC Immune’s wholly-owned anti-Abeta SupraAntigen vaccine candidate.
Initial interim ABATE data triggered the planned trial expansion to include individuals with Down syndrome (DS) and the initiation of evaluation of higher doses of ACI-24.060 in AD.
Interim safety and immunogenicity data from ABATE’s AD and DS cohorts are expected in the second half of 2023.
The first participant was imaged in the pivotal Phase 3 ADvance trial evaluating the Morphomer derived Tau-PET tracer, PI-2620, in AD. PI-2620 is being developed as part of a research collaboration between AC Immune and Life Molecular Imaging.
AC Immune’s therapeutic and diagnostic programs were featured in 10 presentations at the International Conference on Alzheimer’s & Parkinson’s Diseases (AD/PD 2023), highlighting the breadth of the company’s pipeline as well as its differentiated Precision Medicine approach to addressing neurodegenerative disease.
New grants that collectively provide more than USD 500,000 in additional non-dilutive capital to support the advancement of diagnostic programs targeting TDP-43 (TAR DNA-binding protein 43) were awarded to AC Immune by The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and the Target ALS Foundation.
Our webinar "Early Diagnosis and Prevention of Alzheimer’s Disease" featured presentations by invited Key Opinion Leaders, Kaj Blennow, MD, PhD, of University of Gothenburg and Sahlgrenska University Hospital, and Giovanni Frisoni, MD, of University of Geneva and the Memory Clinic at Geneva University Hospital. To view a replay of the webinar, click here.

Anticipated 2023 Milestones

ACI-24.060
anti-Abeta vaccine
Submission of an Investigational New Drug (IND) application to enable expansion of ABATE study to the U.S. expected in H1 2023
Additional interim safety and immunogenicity data from AD cohorts of ABATE study expected in H2 2023
Interim safety and immunogenicity data from DS cohort of ABATE study expected in H2 2023
ACI-7104.056
anti-a-syn vaccine
Update from Phase 2 VACSYN study in Parkinson’s disease expected in H2 2023
ACI-35.030
anti-pTau vaccine
Initiation of next trial in AD expected in H2 2023 (to be followed by milestone payment)
Semorinemab
anti-Tau antibody
Results from the open-label extension of the Phase 2 Lauriet trial in mild-to-moderate AD expected in H2 2023
Anti-TDP-43 antibody
Advancement of candidate into preclinical development (tox) expected in H2 2023
a-syn-PET tracer
Declaration of next clinical candidate for development in Parkinson’s disease expected in H2 2023
TDP-43-PET tracer
Clinical candidate declaration expected in H1 2023
Analysis of financial statements for the quarter ended March 31, 2023

Cash position: The Company had a total cash balance of CHF 105.4 million (CHF 122.6 million as of December 31, 2022), composed of CHF 57.4 million in cash and cash equivalents and CHF 48.0 million in short-term financial assets. The Company’s cash balance provides sufficient capital resources to progress into at least Q3 2024 without considering receiving potential future milestone payments.

R&D expenditures: R&D expenses in the period were CHF 13.9 million, compared with CHF 15.1 million for the comparable period in 2022, mainly driven by lower discovery and preclinical expenses.
Other operating income: The Company recognized CHF 0.4 million in grant income from Michael J. Fox Foundation and Target ALS.

IFRS loss for the period: The Company reported a net loss after taxes of CHF 17.5 million for the three months ended March 31, 2023, compared with a net loss of CHF 18.8 million for the comparable period in 2022.
2023 Financial guidance

Total cash burn guidance for the full year 2023 remains unchanged ranging from CHF 65 to CHF 75 million. The Company defines cash burn as operating expenditures adjusted to include capital expenditures and offset by significant non-cash items (including share-based compensation and depreciation expense).

On April 28, 2023 Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage oncology company developing first-in-class1 therapeutics that combine both targeted and immune-mediated mechanisms, reported positive early results from a global randomized Phase 1b/2 clinical study in which TPST-1120, Tempest’s small molecule PPAR⍺ antagonist, demonstrated clinically-meaningful improvement in multiple categories when combined with the standard-of-care regimen of atezolizumab and bevacizumab in a randomized comparison to atezolizumab and bevacizumab in the first-line treatment of patients with unresectable or metastatic hepatocellular carcinoma (HCC) (Press release, Tempest Therapeutics, APR 28, 2023, View Source [SID1234630687]).

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Data from 40 patients in the TPST-1120 arm randomized per protocol against 29 evaluable (30 total) patients in the control arm showed:

Unconfirmed responses of 30% for the TPST-1120 triplet arm (12/40) vs. 17.2% for the control arm (5/29), demonstrating a 74.4% relative improvement in objective response rate (ORR);
Confirmed responses of 17.5% for the TPST-1120 triplet arm (7/40) vs. 10.3% for the control arm (3/29), demonstrating a 69.9% relative improvement in confirmed ORR;
47.5% (19/40) of the TPST-1120 arm patients are on treatment vs. 23.3% (7/30) in the control arm;
80% (32/40) of the TPST-1120 arm patients are on study vs. 50% (15/30) in the control arm;
The addition of TPST-1120 was well tolerated, with safety data consistent with the control regimen; and,
The randomized arms were generally well balanced at baseline for prognostic factors.
In the IMbrave150 Phase 3 trial, confirmed ORR benefit correlated with overall survival (OS) benefit.2

The study was conducted in clinical collaboration with F. Hoffman La-Roche. Secondary endpoints include duration of response (DoR), progression free survival (PFS) and OS, which are immature as of the data cut and will be potentially available later in the year or next year. Enrollment began in fall of 2021 and the cutoff date for these data was February 8, 2023, which was greater than six weeks after the last patient enrolled and allows for all patients to have had at least one scan. Tempest expects the full data set to be presented by Roche at a medical meeting at a later date. ORR was determined by RECIST v1.1, and confirmed responses included at least two scans.

"These randomized data in first-line HCC are exciting and support the promise of TPST-1120 as an active small molecule for patients," said Stephen Brady, chief executive officer of Tempest. "HCC is a common and aggressive cancer where significant unmet need remains to improve care in the first line and beyond. We believe the improvements shown in the TSPT-1120 arm validate the hypothesis of targeting HCC with TPST-1120, as well as the mechanistic basis for combination with both a checkpoint inhibitor and VEGF inhibitor. We look forward to receiving more data this year, including with respect to potential biomarkers, and to the potential next steps of this program in HCC and other cancers of interest."

"HCC is one of the few tumor types with increasing mortality in the United States," said Mark Yarchoan, M.D., Associate Professor of Medical Oncology at Johns Hopkins School of Medicine. "Atezolizumab + bevacizumab is the current preferred frontline therapy, and the addition of TPST-1120 appears to be active and well tolerated. Response rates in HCC can vary significantly across studies, and therefore the use of a randomized, controlled study design instead of a historical control is a strength of this study. The numerically higher response rate and proportion of patients on study with the addition of TPST-1120 is promising. I believe that this is an active agent and I look forward to further clinical development of TPST-1120."

Conference Call & Webcast Information

Tempest will host a webcast conference call today, April 28, 2023 at 8:30 a.m. ET.

To join the conference call via phone and participate in the live Q&A session, please pre-register online here to receive a telephone number and unique passcode required to enter the call. The live webcast and audio archive of the presentation may be accessed on the investor section of the Tempest website at View Source The webcast will be available for replay for 30 days.

About the Randomized Clinical Trial

The Phase 1b/2 global randomized HCC study is part of Roche’s Morpheus program and evaluates TPST-1120 in combination with the standard-of-care regimen of atezolizumab and bevacizumab in patients with unresectable or metastatic HCC not previously treated with systemic therapy. To date, the trial has enrolled 70 patients to the 1120 arm and contemporaneous control arm at ~25 sites worldwide including in the United States and Europe, who received either the TPST-1120 combination or the standard-of-care regimen of atezolizumab and bevacizumab with primary efficacy endpoint of objective response rate, and key secondary endpoints including PFS and OS. Under the terms of the clinical collaboration agreement, Roche managed the study operations for this global, multicenter trial.

About TPST-1120

TPST-1120 is an oral, small molecule, selective PPAR⍺ antagonist wholly-owned by Tempest. Tempest’s preclinical data suggest that TPST-1120 can kill tumor cells directly and target suppressive immune pathways in the tumor microenvironment. Both types of targeted cells can be dependent on fatty acid metabolism, which is regulated by the PPAR⍺ transcription factor. In extensive non-clinical studies, TPST-1120 as a monotherapy or in combination with other anti-cancer drugs resulted in significant reductions in tumor growth and stimulation of durable anti-tumor immunity. In a Phase 1 clinical trial in patients with heavily-pretreated advanced solid tumors, TPST-1120 as monotherapy and in combination with the PD-1 inhibitor nivolumab demonstrated tumor reduction (including according to RECIST criteria), as well as biomarker modulation. TPST-1120 was well-tolerated both as a monotherapy and in combination with nivolumab.

About Hepatocellular Carcinoma

HCC is an aggressive cancer with rising mortality and is projected to become the third leading cause of cancer death by 2030.3 Every year, more than 900,000 people worldwide are diagnosed with HCC.4 Incidence and mortality are highest in East Asia and are increasing in parts of Europe and the US.5 In the US, HCC represents the fastest-rising cause of cancer-related death.3

Nine out of ten cases of HCC are caused by chronic liver disease, which includes chronic hepatitis B and C infection, non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), alcohol-related liver disease (ALD) and cirrhosis resulting from these conditions.6

Even if diagnosed in the early stage, an estimated 70-80% of people with early-stage HCC experience disease recurrence following surgery.7 Early recurrence is associated with poorer prognosis and shorter survival.5,8 Tumor size, number of tumors, and portal vein invasion are associated with an increased risk of recurrence.

On April 28, 2023 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) reported that it will announce its financial results for the quarter ended March 31, 2023, on Monday, May 8, 2023, after the close of the U.S. financial markets (Press release, Y-mAbs Therapeutics, APR 28, 2023, View Source [SID1234630682]). The announcement will be followed by a conference call and webcast with the investment community on Tuesday, May 9, 2023, at 9:00 a.m. ET. Participating on the call from Y-mAbs will be Thomas Gad, founder, Chairman and Interim CEO; Bo Kruse, Chief Financial Officer; Sue Smith, Chief Commerical Officer and Vignesh Rajah, Chief Medical Officer.

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Conference call and webcast details:

Investors (domestic): 888-886-7786
Investors (international): 416-764-8658
Conference ID: 09065062
To access a live webcast of the update, please use this link.

On April 28, 2023 Adaptimmune Therapeutics plc (NASDAQ: ADAP), a leader in cell therapy to treat cancer, will report financial results and provide business updates for the first quarter ended March 31, 2023, before the US markets open on Friday, May 12, 2023 (Press release, Adaptimmune, APR 28, 2023, View Source [SID1234630681]). Following the announcement, the Company will host a live teleconference and webcast at 8:00 a.m. EDT (1:00 p.m. BST) that same day.

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The press release will be available in the investor section of Adaptimmune’s corporate website. A live webcast of the conference call and replay can be accessed at View Source Call in information is as follows: (800)- 319-4610 (US or Canada) or +1 (416)- 915-3239 (International and additional options available HERE).

On April 28, 2023 Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, reported equity awards on December 31, 2022 that were previously approved by the Compensation Committee of its Board of Directors under Sarepta’s 2014 Employment Commencement Incentive Plan, as a material inducement to employment to 18 individuals hired by Sarepta in April 2023 (Press release, Sarepta Therapeutics, APR 28, 2023, View Source [SID1234630680]). The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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The employees received, in the aggregate, options to purchase 9,975 shares of Sarepta’s common stock, and in the aggregate 5,125 restricted stock units ("RSUs"). The options have an exercise price of $122.77 per share, which is equal to the closing price of Sarepta’s common stock on April 28, 2023 (the "Grant Date"). One-fourth of the shares underlying each employee’s option will vest on the one-year anniversary of the Grant Date and thereafter 1/48th of the shares underlying each employee’s option will vest monthly, such that the shares underlying the option granted to each employee will be fully vested on the fourth anniversary of the Grant Date, in each case, subject to each such employee’s continued employment with Sarepta on such vesting dates.

One-fourth of the RSUs will vest yearly on each anniversary of the Grant Date, such that the RSUs granted to each employee will be fully vested on the fourth anniversary of the Grant Date, in each case, subject to each such employee’s continued employment with Sarepta on such vesting date.