On April 28, 2022 AC Immune SA (NASDAQ: ACIU), a clinical-stage biopharmaceutical company pioneering precision medicine for neurodegenerative diseases, reported results for the quarter ended March 31, 2022, and provided a corporate update (Press release, AC Bioscience, APR 28, 2022, View Source [SID1234613164]).

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Prof. Andrea Pfeifer, CEO of AC Immune SA, commented: "We continue to make strong progress in pursuit of our mission to enable the earlier diagnosis, treatment, and ultimately prevention, of neurodegenerative diseases. Our recent AD/PDTM presentations exemplified the breadth and innovative excellence of our approaches, including ACI-12589 generating the first live images of alpha-synuclein in human brains. This critical breakthrough lays the groundwork for precision medicine, including a pathway for biomarker-based development for NeuroOrphan indications."

Prof. Pfeifer continued: "Throughout the remainder of 2022, we expect to report five additional clinical data readouts from vaccine, antibody, and diagnostic programs targeting Abeta and Tau, including top line results from the landmark Alzheimer’s prevention trial of anti-Abeta antibody crenezumab expected in the coming months. Each of the anticipated milestones represents an important opportunity for value creation and an affirmation of AC Immune’s position as a global leader in addressing neurodegenerative diseases. We look forward to executing on our plan in the year ahead and are pleased to be well financed through at least Q1 2024."

Q1 2022 and Subsequent Highlights

Reported the first live images of alpha-synuclein in the human brain with ACI-12589, AC Immune’s wholly-owned alpha-synuclein (a-syn) positron emission tomography (PET) tracer, at the AD/PDTM 2022 Conference. Clinical PET image analyses showed enhanced contrast and a-syn target specificity in patients with multiple system atrophy (MSA), as well as increased tracer retention in brain areas affected by MSA disease processes. Together with preclinical data also presented at AD/PDTM 2022, these analyses demonstrate ACI-12589’s potential to be the first non-invasive diagnostic for alpha-synucleinopathies (e.g. MSA).

Hosted a key opinion leader webinar on a-syn as a target in neurodegenerative diseases featuring Oskar Hansson, MD, PhD, of Skåne University Hospital and Lund University. To view the presentation and a replay of the webinar, click here.

Announced interim Phase 1b/2a trial data confirming the consistent safety and potent immunogenicity of ACI-35.030, a first-in-class phosphorylated-Tau (pTau) vaccine candidate. Data from the high-dose cohort showed the strong induction of antibodies selective for pTau and its aggregated form, enriched paired helical filaments (ePHF). The data also support ACI-35.030’s favorable safety profile and plans for its continued late-stage development.

Published new data on the optimized formulation of ACI-24 in the peer-reviewed journal Brain Communications, showing that the anti-Abeta vaccine was well tolerated in preclinical models and generated a broad polyclonal anti-Abeta response with high titers of antibodies against neurotoxic pyroglutamate Abeta (pyroGlu-Abeta), a major component of Abeta plaques. Optimized ACI-24 was also shown to have enhanced and sustained immunogenicity against another key pathological Abeta species, oligomeric Abeta, in preclinical studies presented at AD/PDTM 2022.

Expanded leadership with the appointment of Howard Donovan as Chief HR Officer and member to the Executive Committee. Mr. Donovan is an internationally experienced, commercially focused leader. He joins from the World Economic Forum, where he led People Services since 2015.

Joerg Hornstein, Chief Financial Officer, will leave in the second half of 2022 to pursue a new opportunity. AC Immune is well positioned with two members of the company’s proven Finance Leadership Team who will transition to new roles. Christopher Roberts is appointed Vice President, Finance and interim CFO. Julian Snow is appointed Vice President, U.S. Finance & Corporate Development.

Achieved and Anticipated 2022 Clinical Milestones

ACI-12589
a-syn-PET tracer Reported results from first-in-human study at AD/PD 2022 conference
ACI-35.030
anti-pTau vaccine Reported Phase 1b/2a interim analysis from highest dose group in Q1; Expect to disclose future late-stage development plans in H2 2022
ACI-24 (optimized)
anti-Abeta vaccine ACI-24 (optimized vaccine formulation) Phase 1b/2a first-patient-in (AD) in H1 2022
Phase 1b in AD readout and decision to move into DS expected in H2 2022
Crenezumab
anti-Abeta antibody Top line Phase 2 results from AD prevention trial in patients with autosomal dominant AD expected in H1 2022
Semorinemab
anti-Tau antibody Additional fluid biomarker data from the Phase 2 Lauriet study in mild-to-moderate AD expected in H2 2022
PI-2620
Tau-PET tracer Phase 2 and Phase 1 results in AD and progressive supranuclear palsy (PSP) respectively, expected in H2 2022
ACI-7104
anti-a-syn vaccine Initiation of Phase 2 trial in early PD expected in H2 2022

Analysis of Financial Statements for the Quarter Ended March 31, 2022

Cash Position: The Company had a total cash balance of CHF 173.8 million, composed of CHF 57.8 million in cash and cash equivalents and CHF 116.0 million in short-term financial assets. This compares to a total cash balance of CHF 198.2 million as of December 31, 2021. The Company’s cash balance provides enough capital resources to progress through at least Q1 2024 without consideration of potential incoming milestone payments.

R&D Expenditures: R&D expenses increased by CHF 1.8 million for the three months ended March 31, 2022, to CHF 15.1 million.
Discovery and preclinical expenses (- CHF 0.6 million): The Company decreased expenditures across a variety of its discovery and preclinical programs, led by ACI-24 for DS as this program advances into clinical development.

Clinical expenses (+ CHF 0.9 million): The Company increased expenditures across multiple clinical programs, predominantly for ACI-24 for DS and ACI-7104 as the program prepares to enter Phase 2 testing in early PD patients in H2 2022.

Other non-allocated (+ CHF 1.5 million): The Company’s other non-allocated R&D expenditure increased by CHF 0.9 million related to the reallocation of certain IT and facilities costs and IT related investments as well as CHF 0.6 million across various other cost centers.

G&A Expenditures: For the three months ended March 31, 2022, G&A decreased by CHF 0.2 million to CHF 4.2 million. This decrease is predominantly related to a CHF 0.7 million reallocation of certain IT and facilities expenditures made in Q1 2022 that were not reclassified in the prior period.

Other Operating Income: The Company recognized CHF 0.5 million in grant income for R&D activities performed under our Michael J. Fox Foundation for Parkinson’s Research (MJFF) and Target ALS grants, an increase of less than CHF 0.1 million compared to the prior period.

IFRS Loss for the Period: The Company reported a net loss after taxes of CHF 18.8 million for the three months ended March 31, 2022, compared with a net loss of CHF 16.7 million for the comparable period in 2021.
2022 Financial Guidance

For the full year 2022, the Company expects its total cash burn to be in the range, CHF 75 million to CHF 80 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, 2022 Immunicom, Inc., a clinical-stage biotechnology company developing a breakthrough technology platform for immuno-oncology, reported that data from its ongoing clinical investigation, Protocol CP7-005 [NCT04004910], at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Conference in New Orleans (Press release, Immunicom, APR 28, 2022, View Source [SID1234613180]). The trial data, presented by Principal Investigator, Prof. Piotr Wysocki, detailed continued encouraging data for the Company’s LW-02 Cartridge Immunopheresis treatment used with chemotherapy.

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Expanding on Part A data where LW-02 Cartridge Immunopheresis was used as a monotherapy, results from Immunicom’s Part B & Extension—where Immunopheresis is combined with various chemotherapy regimens—continue to demonstrate effectiveness, safety, and tolerability. Median overall survival (OS) extended beyond six months in patients treated greater than four weeks. In addition, the incidence of brain metastases appears to be less than would otherwise be expected in this heavily pre-treated mTNBC population, and further analyses are underway to confirm this observation.

Immunicom’s Immunopheresis subtractive therapy selectively removes sTNF-Rs from plasma through therapeutic apheresis—a blood-filtering process like dialysis—using Immunicom’s unique LW-02 Cartridge (containing the Company’s proprietary, high-affinity, molecular ligand subtractive matrix). Removal of sTNF-R from plasma unleashes a patient’s natural TNF-α, an innate molecule in the body, to directly kill cancer tumor cells and upregulate the immune system to attack the tumor.

Commenting on the data, Dr. Wysocki stated, "We are seeing further confirmation that Immunopheresis is a potentially revolutionary treatment for cancer. In these difficult to treat late-stage cancer patients, the possibility of extending survival and preventing spread of the cancer to the brain, which is common in these patients, is especially promising and opens a new playing field in the battle against refractory cancers. Our longest surviving patient who had progressed three prior lines of treatment before being treated with Immunopheresis therapy had a robust response to treatment that lasted nine months. For this patient, disease progression occurred with reduced frequency of LW-02 Immunopheresis treatment, but a robust response was again observed on increasing the LW-02 treatment to three times per week, demonstrating the ability of LW-02 Immunopheresis therapy to produce a durable and sustained response now out to over 18 months."

Immunicom’s AACR (Free AACR Whitepaper) presentation underscores the burgeoning prospects for the Company’s Immunopheresis technology platform on multiple fronts in the fight against cancer and other immunologic disorders. The LW-02 Cartridge has received FDA Device Breakthrough Designation for treatment of solid malignancies and a CE Mark in Europe for use in chemo-refractory mTNBC. Three ongoing clinical trials continue to assess LW-02 Cartridge Immunopheresis as a monotherapy, and/or in combination with chemo- or immunotherapy, with other trials planned to initiate later in 2022.

Prof. Wysocki’s AACR (Free AACR Whitepaper) poster presentation is available on Immunicom’s website.

Subtractive Therapy – Immunopheresis and the LW-02 Cartridge

Immunicom employs a proprietary, high-affinity, molecular capture-ligand binding matrix within the LW-02 Cartridge to remove specific cytokine receptors, soluble TNF-Receptors 1 and 2 (sTNFR-1/2), which are shed by cancer cells into the extracellular tumor microenvironment. sTNF-Rs serve as decoys, binding tumor necrosis factor alpha (TNF-α) before it can bind to its membrane-embedded sTNF-Rs receptor to trigger several cell death pathways. The selective removal of decoy sTNF-Rs by the LW-02 Cartridge unleashes the patient’s immune system to identify and aggressively attack the cancer.

Immunopheresis, like dialysis, is a subtractive therapy that occurs outside the body, in contrast to conventional drug and biologics that are infused into the patient. Immunopheresis is thus much better tolerated than chemo- and immunotherapies, allowing for its use as an adjunct with these therapies, possibly in lower doses to reduce their toxicity.

On April 28, 2022 Corcept Therapeutics Incorporated (NASDAQ: CORT) reported it will report first quarter financial results and provide a corporate update on May 5, 2022 (Press release, Corcept Therapeutics, APR 28, 2022, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announce-first-quarter-financial-results-7 [SID1234613198]). The company will also host a conference call that day at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time).

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Conference Call Information
To participate, click the link below and enter your information. The link will become active 15 minutes prior to the scheduled start time.

Click to Join Meeting

Alternatively, you may dial 1-833-693-0540 from the United States or 1-661-407-1581 internationally approximately 15 minutes before the start of the call. The passcode will be 6942208.

A replay will be available through May 12, 2022, at 1-855-859-2056 from the United States and 1-404-537-3406 internationally. The passcode will be 6942208. You may also access a replay on the Investors / Past Events tab of www.corcept.com

On April 28, 2022 Kelonia Therapeutics, a biotech company revolutionizing in vivo gene delivery, launched with a $50 million Series A financing to usher in a new era of genetic medicines for a wide range of diseases (Press release, Kelonia Therapeutics, APR 28, 2022, Kelonia Therapeutics Launches with $50 Million Series A Financing to Pioneer Precision Targeted Genetic Medicines [SID1234640176]). Kelonia’s platform overcomes the central challenge that has prevented the full realization of gene therapy for patients. Despite life-changing responses, existing gene therapies are highly complex, costly, and limited by complicated treatment paradigms, tractable therapeutic applications, and dose-limiting toxicities. By enabling precisely targeted, highly efficient, manufacturable "off-the-shelf" in vivo gene delivery, Kelonia’s technology has the potential to dramatically expand the impact and reach of genetic medicines to every patient in need.

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Kelonia is backed by a strong syndicate of investors with a track record of successfully launching and building disruptive biotech companies. Alta Partners, Horizons Ventures, Venrock and other investors participated in the Series A round. The company will use the funding to redefine what’s possible for genetic medicines starting with an "off-the-shelf" chimeric antigen receptor (CAR) to treat hematologic cancer that may enable the unrivalled clinical benefit of CAR T without the typical toxicities and with the ease of access of conventional medicines. Additionally, the company will advance other programs for oncology and non-oncology indications, and further expand its gene delivery platform and capabilities.

"The cell and gene therapy field has been searching for solutions to durable in vivo genetic modifications regardless of whether applying gene editing, RNA expression or viral-mediated gene integration," said Kevin Friedman, Ph.D., President and Chief Scientific Officer of Kelonia. "At Kelonia, we believe we have found an in vivo gene delivery solution that is safe, effective, and manufacturable for broad therapeutic application. With our Series A funding and key strategic collaborations, we will advance our lead product candidate toward clinical studies and further optimize our technology to explore treating diseases never thought possible with genetic medicines."

Based on discoveries made in the lab of Massachusetts Institute of Technology’s Michael Birnbaum, Ph.D., and leveraging pioneering research from leading scientists at the French National Centre for Scientific Research (CNRS), Kelonia’s in vivo gene delivery technology enables a few potent lentiviral vector-like particles armed with an adjustable targeting system to precisely, efficiently, and safely deliver payloads exactly where needed to treat a broad range of diseases. The company’s early applications combine oncology-targeted therapeutics, such as CAR and T cell receptor molecules, with Kelonia’s precision in vivo targeting technology. When used in concert, this combination enables potent and precise tumor targeting with limited "off-tumor" toxicity, which would otherwise be a concern. Administered directly in vivo as an "off-the-shelf" medicine, Kelonia’s transformational therapies in development for solid and hematologic tumors have the potential to democratize patient access to genetic medicines. Beyond oncology, the company will advance its technology to unlock delivery to previously hard-to-reach tissues, such as neurological, muscular or renal, to deliver different types of genetic cargo with the goal of radically transforming the treatment of diseases in these areas.

"It turns out, a relatively simple and elegant idea to de-target and redirect lentivirus-like particles based on recently published research from my lab can potentially provide a solution to in vivo gene delivery," said Dr. Birnbaum, Ph.D., Co-Founder of Kelonia. "I’m incredibly excited about the potential of Kelonia’s platform and team to vastly expand the utility of gene therapies to treat oncology, autoimmune disease, rare monogenic or other diseases currently intractable to gene therapies."

"Kelonia is combining the two crucial elements required to develop truly novel medicines: breakthrough biology and an exceptional team," said Bryan Roberts, Partner at Venrock. "Michael Birnbaum’s industrially robust platform affords a targeting specificity log orders better than anything else out there and the team has a stellar track record for translating groundbreaking scientific gene therapy discoveries into viable products that are transformative for patients."

Strategic Collaborations

In addition to the completion of its Series A, Kelonia has established strategic collaborations with Adimab and ElevateBio. With both collaborations already successfully underway, each of these outstanding partners brings differentiating capabilities that enable and accelerate the company’s vision to bring breakthrough genetic medicines to patients.

Adimab is the leading provider of therapeutic antibody discovery and engineering technologies. Kelonia will leverage Adimab’s expertise and proprietary technologies, across a range of applications, to access tissue-specific antibodies that enable unlocking precise in vivo gene delivery to different tissues as well as antibodies that can be leveraged within the therapeutic genetic cargo.

ElevateBio is a technology-driven company focused on powering transformative cell and gene therapies with multiple next-generation technology platforms and a fully integrated R&D and manufacturing facility. Through an expanding partnership, Kelonia will utilize ElevateBio’s lentiviral vector platform, process and analytical development expertise, and cGMP manufacturing capabilities to develop and advance novel manufacturing processes for Kelonia and manufacture of Kelonia’s products.

On April 28, 2022 ArcticZymes Technologies (OSE: AZT) reported sales of NOK 49.2 million (40.9) and an EBITDA of NOK 27.9 million (25.8) for the first quarter of 2022 (Press release, Biotec Pharmacon, APR 28, 2022, View Source [SID1234613094]).

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Highlights from Q1 2022

ArcticZymes Technologies (AZT) had Q1 sales of NOK 49.2 million growing by 21% (Q1 2021: NOK 40.9 million)
Positive EBITDA of NOK 27.9 million (Q1 2021: NOK 25.8 million)
Cash flow for Q1 was positive NOK 14.2 million (Q1 2021: NOK 23.2 million) giving a cash balance of NOK 214.6 million (Q1 2021: NOK 163.3 million)
Signed a new OEM supply deal with a leading global Life Science company
Established new application laboratory at ShareLab in Oslo

CEO Jethro Holter comments:

"We are delighted with the strong performance of our first quarter for 2022. The ArcticZymes team has delivered the best-ever quarterly performance achieving sales revenues of NOK 49.2 million and an EBITDA of NOK 27.9 million. Both Molecular Tools and Biomanufacturing were instrumental in delivering growth.

Furthermore, ArcticZymes is proud to expand its operational activities outside of Tromsø and strategically establish itself at ShareLab in Oslo. Initial activities at Sharelab will focus on application development."