Another Fabry flop, courtesy of 4D

 Amid a narrowing pipeline of gene therapy candidates for Fabry disease, new doubts are creeping in about one of the contenders, 4D Molecular’s 4D-310. An update yesterday detailed three cases of atypical haemolytic uremic syndrome (aHUS) among six patients who have received the project so far – one of which was classified as grade four. This adverse event was seen in one patient in the initial data drop in 2021. 4D is now pausing enrolment into its current studies, although a pivotal trial is being planned. The group will also abandon the corticosteroid regimen it has been using for immunosuppression, in favour of a rituximab-sirolimus regimen that it hopes will prevent aHUS. In addition, 4D reported intriguing cardiac efficacy data from three patients in the phase 1/2 US Inglaxa study, but this was clearly not enough to reassure investors, who sent the group’s stock down 10% yesterday. More details on 4D-310’s efficacy and safety will be presented at the World Symposium in February. This meeting is also set to feature further results on Sangamo’s ST-920, which currently looks like the Fabry gene therapy project to beat – although the bar is low.

The Fabry gene therapy data so far
Project	Company	Trial	Dose(s)	AGA activity	Note
4D-310	4D Molecular	Inglaxa & Asia-Pacific trial	1x1013vg/kg (lowest dose)	Feb 2022: 10 to 14-fold of mean normal in 2 pts; Jan 2023: cardiac data in 3 US pts	Three cases of aHUS across 6 pts in US & Asia-Pacific trials; enrolment paused
ST-920	Sangamo	Staar	0.5x1013, 1x1013, 3x1013 & 5x1013vg/kg	Oct 2022: 2 to 30-fold of mean normal in 9 pts; 4 pts withdrawn from ERT	Study now in dose-expansion phase
FLT190	Freeline	Marvel-1	7.5x1011vg/kg (lowest dose)	Nov 2021: subtherapeutic & near normal in 2 pts*; Nov 2022: levels "sustained" for 1-3yrs	Dosing in 2nd cohort (1.5x1012vg/kg) began Sep 2022
Note: all studies ph1/2; *both pts experienced myocarditis; AGA=alpha-galactosidase A; ERT=enzyme-replacement therapy. Source: company releases. https://www.evaluate.com/vantage/articles/news/trial-results-snippets/another-fabry-flop-courtesy-4d

Agilent upped to Overweight from Equal Weight by Wells Fargo

 Target $170

https://finviz.com/quote.ashx?t=A&ty=c&ta=1&p=d

BriaCell Announces New Patent Issuance, Bria-OTS™ IND Timing Guidance

 

• BriaCell’s new patent protects its novel personalized off-the-shelf cell-based technology for breast cancer and multiple other cancer indications until 2040.

• BriaCell expects to initiate its Bria-OTS™ clinical study under an Investigational New Drug Application (IND) in the first half of 2023, in accordance with FDA guidance.

• Two additional clinical sites are now active in Phase II Bria-IMT™ study to broaden patient access: Carle Cancer Institute in Urbana, Illinois and the American Oncology Network, LLC (AON) in Baltimore, Maryland.

https://finance.yahoo.com/news/briacell-announces-patent-issuance-bria-140000423.html

Sarissa: Add Directors and Remove Chairman of Amarin

 Sarissa Capital Management LP ("Sarissa") today made the following statement on Amarin Corporation plc (NASDAQ: AMRN):

At the last shareholder meeting, the shareholders, the owners of the company, loudly expressed their lack of faith in the board and management. Sarissa sought to place shareholder representatives on the board and engaged with the company in what turned out to be months of simply lip service by the board, led by its Chairman Per Wold-Olsen. Despite Sarissa having a track record of creating significant shareholder value in healthcare companies, including those with cardiovascular drugs such as The Medicines Company, Amarin’s board rejected adding any shareholder representatives to the board in favor of their own candidates, including today’s addition of a board candidate without any input from Sarissa, Amarin’s largest shareholder.

https://finance.yahoo.com/news/sarissa-capital-submits-notice-call-183400808.html

DermTech Melanoma Test (DMT) Is Recommended for Coverage by TRICARE

 DermTech, Inc. (NASDAQ: DMTK) ("DermTech" or the "Company"), a leader in precision dermatology enabled by a non-invasive skin genomics platform, today announced that the Defense Health Agency’s Lab Joint Working Group has recommended the Company’s foundational assay included in the DermTech Melanoma Test (DMT) for coverage by TRICARE. TRICARE is the health care program for uniformed service members, retirees and their families around the world.

This coverage recommendation makes the foundational assay of the DMT available to TRICARE’s approximately 9 million members and beneficiaries of the Military Health System. The DMT is the first non-invasive approach to enhance melanoma detection with a greater than 99 percent negative predictive value (NPV).

https://finance.yahoo.com/news/dermtech-announces-foundational-assay-dermtech-133000036.html

Prenetics Owned ACT Genomics 1st Asia Company Cleared for Comprehensive Genomic Profiling Test

 

• Prenetics has completed the majority stake acquisition of ACT Genomics to spearhead its entry into to the US$80+ billion global precision oncology market

• FDA market authorization advances personalized cancer care in Asia

• Clearance sets the stage for continued R&D investment into early detection / liquid biopsy

https://finance.yahoo.com/news/prenetics-owned-act-genomics-receives-130500625.html

Biora Therapeutics Receives Pre-IND Feedback from FDA

 Biora Therapeutics, Inc. (Nasdaq: BIOR), the biotech company that is reimagining therapeutic delivery, today announced feedback from the United States Food and Drug Administration (FDA) on its clinical development plans for its PGN-600 program, and provided program updates.

For Biora’s Targeted Therapeutics Platform, which is focused on treatment of ulcerative colitis (UC), the company remains on track for an IND filing for its PGN-600 program followed by clinical trial initiation. During Q4 2022, Biora continued its engagement with the FDA with a pre-IND supplemental Type C filing requesting agency feedback on its proposed PGN-600 clinical development plans, including the company’s proposed approach to toxicity studies and other aspects of its clinical plan.

“The recent Type C response from the FDA further strengthens our confidence in our plans to enter the clinic during the first half of 2023 with IND filing followed by trial initiation in Q2, and data readouts anticipated in Q3,” said Adi Mohanty, Chief Executive Officer of Biora Therapeutics. “Based on previous studies, we know that achieving the appropriate concentration of tofacitinib in the tissue of the large intestine is correlated with endoscopic improvement, and we believe that reducing systemic exposure to tofacitinib could be a paradigm shift in the ability to help a substantial percentage of patients. Our planned clinical trial should provide insights on these very important tissue and plasma levels, along with the safety of our approach. With this critical data anticipated during the phase 1 trial, we look forward to progressing a program that could have significant impact on patient care for ulcerative colitis.”

Biora Therapeutics has previously shown the strong potential of its Targeted Therapeutics platform to help patients with ulcerative colitis (UC) through data demonstrating that:

• Higher tissue levels of tofacitinib in UC patients are correlated with endoscopic improvement.

• Biora’s device can accurately deliver liquid payload to the correct location in the colon of healthy volunteers.

• Biora's device can accurately identify colon entry and deliver liquid payload to the site of disease in active ulcerative colitis patients, despite a challenging environment of inflammation, bleeding, and highly variable motility.

• Biora’s device can function as intended when administered both with and without food.

For Biora’s Systemic Therapeutics program, the company has been transitioning from early concept to a clinical-ready device. With several of the key device upgrades implemented, the company expects to report data from preclinical studies on its next-generation device during Q1 and Q2 of 2023.

https://finance.yahoo.com/news/biora-therapeutics-receives-pre-ind-140000531.html