Gen­Sight has a gene ther­a­py mys­tery on its hands, which is about the last thing it want­ed at the end of Phase III.

As­sess­ing the 48-week da­ta from its first piv­otal test of a treat­ment for Leber Hered­i­tary Op­tic Neu­ropa­thy — or LHON, a rare ge­net­ic dis­ease that caus­es blind­ness — in­ves­ti­ga­tors pulled out a mean­ing­ful im­prove­ment in sight in their one treat­ed eye.  

Bernard Gilly

The mys­tery: The oth­er eyes that re­ceived a sham ther­a­py did just as well with their eye­sight. Ex­act­ly as well.

That makes the Phase III study of GS010 a fail­ure, and rather than stay to try and solve the mys­tery, in­vestors bailed, dri­ving down the Eu­roNext stock $SIGHT down by 34%.

One of the rea­sons why the ear­ly gene ther­a­py pi­o­neers like Gen­Sight picked hered­i­tary eye dis­eases to go af­ter first is that the eye is a con­tained unit, lim­it­ing off-tar­get ef­fects. But Gen­Sight’s Paris-based CEO Bernard Gilly says that as on­ly a frac­tion of pa­tients his­tor­i­cal­ly see a spon­ta­neous im­prove­ment in eye­sight, he’s won­der­ing if the ther­a­py in­ject­ed in­to one eye de­liv­ers an un­ex­pect­ed ben­e­fit to the oth­er.

At the same time, Gen­Sight tout­ed a hit on a reti­nal bio­mark­er for the dis­ease, which they claimed as ev­i­dence that the ther­a­py had an im­pact sep­a­rate from the sham.

“The fact that struc­tur­al mea­sures of the reti­na showed such a large sta­tis­ti­cal dif­fer­ence with treat­ment is com­pelling and ob­jec­tive ev­i­dence that this gene ther­a­py pro­tects the in­tegri­ty of many reti­nal gan­glion cells from the dam­age of LHON,” com­ment­ed com­pa­ny co-founder José-Alain Sa­hel.

That’s a messy ar­gu­ment, with the com­pa­ny ar­gu­ing that it ap­peared the ther­a­py im­proved sight for both eyes, but on­ly hit the sec­ondary bio­mark­er in the treat­ed eye. 

The com­pa­ny — which tried and failed to make it on­to Nas­daq — will be left look­ing for more da­ta to an­a­lyze from its next two Phase III stud­ies, which are up­com­ing. But easy ex­pla­na­tions will be hard to come by.

More than two years into the Covid-19 pandemic, we’re not done seeing its disruption of biotech.

That’s according to Caladrius execs, who cited the pandemic as one reason for suspending its Phase IIb trial of a cell therapy as a treatment for coronary microvascular dysfunction (CMD). The company plans to go ahead on an interim analysis before deciding what to do with the program, which was built on a licensing deal with Shire in 2018.

Vanda Pharmaceuticals is making a name for itself, at least in terms of suing the FDA.

The DC-headquartered firm on Monday filed its latest suit against the agency, with the company raising concerns over the FDA’s failure to grant a fast track designation for Vanda’s potential chronic digestive disorder drug tradipitant, which is a neurokinin 1 receptor antagonist.

Specifically, Vanda said FDA’s “essential point” in its one-page denial letter on the designation pointed to “the lack of necessary safety data,” which was “inconsistent with the criteria for … Fast Track designation.”

SpringWorks Therapeutics thinks it has cemented the backbone for its first “pipeline-in-a-product” oncology treatment and will send it to the FDA before the clock strikes 2023 with a Phase III win on Tuesday.

The oral gamma secretase inhibitor, dubbed nirogacestat, beat placebo on the primary goal of progression-free survival in adults with progressing desmoid tumors.

The soft-tissue tumors can lead to long-lasting pain, disfigurement and amputation, and there are currently no approved meds for the rare oncology indication. The tumors typically impact patients aged 20 to 44 years old and disproportionately affect women at rates 2 to 3 times higher, with up to a total of 1,650 new cases diagnosed in the US annually, according to SpringWorks.