Tiziana Life Sciences Ltd. (Nasdaq: TLSA) (“Tiziana” or the “Company”), a biotechnology company developing breakthrough neuro-immunomodulation therapies, today announced “first patient dosed” in its Phase 2a study comparing two doses of intranasal foralumab and placebo in patients with non-active secondary-progressive multiple sclerosis (na-SPMS). Six investigational centers have been recruited for this double-blind, placebo-controlled trial, with up to 18 patients per treatment arm. The primary endpoint of the trial will be the change in microglial activation based on PET scans. Clinical evaluations include the Expanded Disability Status Scale (EDSS), QoL assessments, and the Modified Fatigue Impact Scale (MFIS), which assess parameters that are essential to a patient’s everyday life. Novel immuno-biomarkers will be measured also and assessed for predictive relevance. Central review of PET scans and images is an integral component of this study.
Tuesday, December 19, 2023
Investors still aren’t sold on UniQure’s gene therapy for Huntington’s
Shares of UniQure dipped more than 10% Tuesday morning after the biotechnology company released more data from a small study testing an experimental gene therapy for Huntington’s disease.
The new results include up to 30 months of follow-up from 39 patients enrolled in the study, which is evaluating two doses of UniQure’s therapy, called AMT-130. The company said there’s evidence both doses are generally safe and can preserve or improve neurological function in patients with the disease, which progressively impairs movement and cognition.
While UniQure leadership describes the results seen so far as “very promising,” analysts on Wall Street have found them puzzling. The low dose, for example, has appeared more effective and less variable than the high dose. How long either stays effective is also unclear. Nevertheless, UniQure intends to engage with regulators early next year to talk through the data it’s collected and “potential strategies for ongoing development of AMT-130.”
Huntington’s is caused by mutations in the gene responsible for making the similarly named “huntingtin” protein. While helpful to brain function in its normal form, the protein, when mutated, bunches together into toxic clumps that damage nerve cells.
AMT-130 is designed to silence that malfunctioning gene, and has become one of UniQure’s most closely watched programs.
In its update, UniQure said neurological function in patients given the high dose of AMT-130 was preserved or improved 18 months after treatment, compared to the start of the study. That was measured by a scoring system known in short as cUHDRS, which assesses the motor function, brain function and behavior of people with Hungtinton’s.
In the low dose arm, two other scales found neurological function was preserved at 30 months, according to UniQure.
The company added that, across all three measurements, AMT-130 showed “favorable trends” on the expected rate of decline compared to a natural history cohort. The cohort, which UniQure created with assistance from a nonprofit research organization, includes 31 patients with early Huntington’s disease who were similar to the participants in the AMT-130 study. Drugmakers use natural history data to better understand how a disease progresses without intervention.
Activist Investor Icahn Targets Illumina Board Amid GRAIL Divestiture
Activist investor Carl Icahn is at it again—planning to give the boot to Illumina’s board of “legacy conflicted directors.” Icahn disclosed his plans in a letter to shareholders Monday after the company said it would divest GRAIL, according to Reuters.
Illumina announced Sunday it was throwing in the towel on GRAIL, following a drawn out antitrust battle with regulators over the cancer testing company’s acquisition. On Friday, the U.S. Fifth Circuit Court of Appeals agreed there was evidence the deal was anti-competitive.
The $7 billion acquisition of GRAIL has been Icahn’s greatest complaint against Illumina. The gene-sequencing company went forward with the buy despite opposition from antitrust regulators. The legal woes that followed caused a 75% drop in Illumina’s share price, losing $55 billion in value for its shareholders, Icahn stated in Monday’s letter in which he applauded the decision to divest GRAIL but said that alone is not enough.
In June 2023, Illumina’s then-CEO Francis deSouza was pushed out of the helm. At the time, Icahn attempted to have three of his representatives placed on the board, but only one was elected.
“It would be a great mistake to allow the legacy conflicted directors to influence Illumina given their history of reckless decision making and value destruction,” Icahn's letter said, while clarifying that the conflicted directors are those worried about personal liability for the “billions of dollars they have cost the company through their negligence.”
According to Reuters, only four directors—Scott Ullem, Teno, Stephen MacMillan and the company's new CEO Jacob Thaysen—played no role in recent decisions. In his letter, Icahn did not divulge details of his plans but could target as many as seven directors for removal.
Icahn also filed a lawsuit against the board in October 2023, for breach of fiduciary duties by the directors. The 87-year-old activist had never sued a company’s board of directors prior to this.
GRAIL will be divested from Illumina through a third-party sale or capital markets transaction, according to the company’s announcement Sunday. Illumina was fined $476 million by the European Commission for “knowingly and intentionally” violating the commission’s merger regulations.
Gilead, Compugen Ink Potential $848M Deal for Preclinical Immunotherapy
Gilead Sciences is making a $60 million upfront payment as part of an exclusive license agreement for one of Compugen’s assets in a deal valued at $848 million, the companies announced Tuesday.
Under the agreement, Gilead will exclusively license a preclinical antibody asset from Compugen that acts against the Il-18 binding protein, as well as the Israeli biotech’s COM503 drug candidate designed to block the interaction between IL-18 binding proteins and IL-18, releasing natural IL-18 into a tumor to stop cancer growth.
“IL-18 is one of the rare cytokines which is naturally inhibited by an endogenous binding protein, presenting a unique opportunity to use a blocking antibody to increase the local concentrations of IL-18 within the tumor where it can potentiate anti-tumor immune responses, thereby potentially overcoming the limitations of systemically administered cytokines,” Compugen CEO Anat Cohen-Dayag said in a statement.
Compugen will remain responsible for the preclinical development and eventual Phase I study of the asset. After that, Gilead will have the right to further develop and commercialize the COM503 candidate.
In addition to the upfront payment of $60 million, a $30 million milestone payment will be available to Compugen related to the IND clearance of COM503, which is expected to be completed in 2024. Compugen is also eligible for an additional $758 million in development, regulatory and commercial milestone payments and single-digit to low double-digit royalties on net sales globally for the asset.
Compugen’s share price jumped 169% before the opening bell on Tuesday morning.
Gilead has been working hard on the deal front in 2023. In November, through its subsidiary Kite, the company decided to widen its collaboration with Arcellx. The two companies will evaluate the cell therapy CART-ddBCMA as a lymphoma treatment. Kite is also beginning to negotiate a license for an additional multiple myeloma program from Arcellx. Gilead’s Kite made an upfront payment of $85 million in the deal.
In September, Gilead closed its $405 million acquisition of U.K.-based MiroBio, which is focused on restoring immune balance with agonists targeting immune inhibitory receptors. The buy provides Gilead with MiroBio’s proprietary discovery platform and portfolio of immune inhibitory receptor agonists.
Successful Treatment of Obstructive Sleep Apnea with ProSomnus Precision Oral Appliance Devices
ProSomnus, Inc. (NASDAQ: OSA) (the “Company”), the leading non-CPAP Obstructive Sleep Apnea (OSA) therapy™, today announced the publication of a study by the Cureus Journal of Medical Science, in which the Company’s precision oral appliance therapy (OAT) devices successfully treated patients’ OSA.
“The study underscored what we see daily in our practice at Star Sleep & Wellness. Our team continues to favor the ProSomnus® EVO® Sleep and Snore Device, as it not only garners the fewest number of patient complaints regarding fit and comfort, but also shows to be superior in resolving chief complaints such as partner discontent, fatigue and daytime sleepiness,” said Dr. Kent Smith, D-ABDSM, ASBA, a co-author of the study.
Compugen Eligible for $10 M Milestone in AstraZeneca Phase 3 Trial in Biliary Tract Cancer
Compugen Ltd. (Nasdaq: CGEN) (TASE: CGEN) a clinical-stage cancer immunotherapy company and a pioneer in computational target discovery, today announced it will be eligible to receive a milestone payment of $10 million from AstraZeneca (LSE/STO/Nasdaq: AZN), when the first patient is dosed in AstraZeneca's ARTEMIDE-Bil01 trial with rilvegostomig. Rilvegostomig is a PD-1/TIGIT bispecific antibody where the TIGIT component is derived from Compugen's clinical-stage anti-TIGIT antibody, COM902. The ARTEMIDE-Bil01 trial is expected to recruit about 750 subjects in more than 20 countries with biliary tract cancer who will be randomized to receive rilvegostomig or placebo with investigator choice chemotherapy as adjuvant treatment after resection with curative intent.
Innate Pharma Licenses 4th Natural Killer Cell Engager in Oncology to Sanofi
- Sanofi to exercise one option to license a new ANKET® program from Innate; After a research collaboration period, Sanofi will be responsible for all development, manufacturing and commercialization of the program
- Innate to receive €15m as option exercise payment