AUPH's Dry Eye Syndrome Study Fails, KPTI's SEAL Study Hits Goal, REGN Halts Rare Bone Disease Trial

(RTTNews) - Today's Daily Dose brings you news about Aurinia's disappointing results from its dry eye syndrome trial, CureVac's encouraging data from phase I study of COVID-19 vaccine candidate, Jounce discontinuing enrollment in NSCLC trial, Karyopharm's promising results from SEAL study of Xpovio, Regeneron pausing fibrodysplasia ossificans progressive trial on reports of deaths, and Santhera trimming its workforce.

Read on…

1. Aurinia's Dry Eye Syndrome Treatment Fails to Meet Study Goal

Shares of Aurinia Pharmaceuticals Inc. (AUPH) (AUP.TO) slumped over 10% in extended trading Monday, following disappointing data from its phase II/III study of Voclosporin ophthalmic solution in dry eye syndrome.

The phase II/III study, dubbed AUDREY, did not achieve statistical significance on its primary endpoint of a 10mm or greater improvement in Schirmer Tear Test (STT) at 4 weeks.

Based on the AUDREY trial results, the company has decided to suspend the development of Voclosporin ophthalmic solution for dry eye syndrome.

Voclosporin in oral formulation, proposed for the treatment of lupus nephritis, is under FDA review, with a decision expected on January 22, 2021.

AUPH closed Monday's trading at $15.50, down 1.15%. In after-hours, the stock fell 10.32% to $13.90.

2. CureVac to Initiate Pivotal trial of COVID-19 Vaccine Candidate before Year-end

CureVac N.V.'s (CVAC) ongoing phase I dose-escalation study evaluating the safety, reactogenicity and immunogenicity of CVnCoV, its investigational SARS-CoV-2 vaccine candidate, in healthy adults, has yielded positive results.

CVnCoV was generally well tolerated across all tested doses (2-12µg) and induced strong binding and neutralizing antibody responses in addition to first indication of T cell activation, the company noted.

A phase IIa clinical trial of the COVID-19 vaccine candidate in older adults in Peru and Panama is ongoing. The company is expected to initiate a pivotal phase IIb/III trial of CVnCoV before the end of this year.

CVAC closed Monday's trading at $47.08, down 3.49%.

3. Jounce Discontinues Enrollment In EMERGE Study

Jounce Therapeutics Inc. (JNCE) has decided not to continue further enrollment in its phase II trial of Vopratelimab, dubbed EMERGE, based on interim analysis.

EMERGE is a phase II clinical trial of Vopratelimab in combination with Ipilimumab in patients with non-small cell lung cancer (NSCLC) or urothelial cancer who have progressed on or after PD-1/PD-L1 inhibitor therapies. The trial was designed to enroll 40 patients with NSCLC and approximately 40 patients with urothelial cancer. According to the company, so far fifty-nine patients were enrolled in the EMERGE trial, 50 of whom are evaluable.

Early evaluation of the data from the EMERGE trial has indicated that the trial will not meet pre-specified interim criteria for continuation of enrollment.

The company also announced that it has dosed the first patient in its phase II trial evaluating a combination of Vopratelimab and JTX-4014 in immunotherapy naïve biomarker-selected NSCLC patients, dubbed SELECT.

JNCE closed Monday's trading at $6.82, down 18.03%.

4. Karyopharm's Xpovio Shows Promise in treating Liposarcoma

Shares of Karyopharm Therapeutics Inc. (KPTI) were up over 7% in extended trading Monday, following positive top-line results from the phase III portion of its phase II/III trial of XPOVIO in advanced unresectable dedifferentiated liposarcoma.

The phase II/III trial of XPOVIO in advanced unresectable dedifferentiated liposarcoma, dubbed SEAL, was initiated in 2016. The results from the phase II portion of the trial were reported in 2017. The phase II portion of the SEAL study met the primary endpoint, with patients in the XPOVIO arm achieving progression-free survival (PFS) of 5.5 months, compared to 2.7 months for placebo-treated patients, an increase of 2.8 months.

In the phase III portion of the SEAL study, the topline results of which were reported Monday, XPOVIO reduced the risk of disease progression or death by approximately 30%, compared to placebo.

The detailed results from the SEAL study will be presented virtually in an oral presentation at the Connective Tissue Oncology Society (CTOS) Annual Meeting on Friday, November 20, 2020.

The company plans to seek approval for XPOVIO in the indication of liposarcoma, a rare cancer of connective tissues, in the first quarter of 2021.

XOPVIO, known generically as Selinexor, is currently approved by the FDA for the treatment of relapsed or refractory multiple myeloma and relapsed or refractory diffuse large B-cell lymphoma (DLBCL). The drug is also under FDA review for the treatment of patients with multiple myeloma after at least one prior line of therapy, with a decision expected on March 19, 2021.

The net product sales of XPOVIO during the third quarter of 2020 were $21.3 million compared to $12.82 million in the year-ago quarter.

KPTI closed Monday's trading at $14.50, down 2.16%. In after-hours, the stock was up 7.79% at $15.63.

5. Regeneron Pauses LUMINA-1 trial after Fatal Serious Adverse Events

Regeneron Pharmaceuticals Inc. (REGN) has paused its phase II trial of investigational therapy Garetosmab in patients with fibrodysplasia ossificans progressive, dubbed LUMINA-1, following reports of deaths.

Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder in which muscles, tendons and ligaments are progressively replaced by bone, a process known as heterotopic ossification.

The fatal serious adverse events were reported in the trial during the open-label extension, during which all patients received Garetosmab. The company has not disclosed the number of deaths that have occurred during the extension phase of the trial.

In January of this year, the company had reported encouraging topline 28-week results from the 44-patient LUMINA-1 trial. During the 28-week treatment period, there were no deaths in the trial.

REGN closed Monday's trading at $548.58, down 0.92%.

6. Santhera Trims Headcount by More than 50; DMD Trial Data to Be Reported In Q2

As Santhera Pharmaceuticals Holding AG (SPHDF.OB) gears up to report Duchenne Muscular Dystrophy trial data, an organizational restructuring in order to better focus on its lead investigational asset has been implemented.

The lead drug candidate Vamorolone is currently in a pivotal phase IIb study in Duchenne Muscular Dystrophy patients, with a 6-month readout expected in the second quarter of 2021. If the trial results are positive, the company expects to make an NDA filing in the fourth quarter of 2021.

In order to refocus on Vamorolone and extend its cash reserves, the company has decided to trim its workforce by more than 50 positions to 47 full-time equivalent employees. This restructuring will result in one-time costs of approximately CHF 3 million against recurring annual cost reductions of CHF 10 million.

Kristina Sjöblom Nygren, the company's Chief Medical Officer and Head of Development, is leaving the company by this year-end owing to family reasons and to pursue other opportunities. A search is on to find a successor to Kristina.

SPHDF.OB closed Monday's trading at $3.57, unchanged from the previous day's close.

7. Stocks That Hit New Highs/Lows

Shattuck Labs Inc. (STTK) closed Monday's trading at a new high of $26.87, up 3.95%.

R1 RCM Inc. (RCM) closed Monday's trading at a new high of $18.59, up 3.74%.

Bio-Rad Labs Inc. (BIO) closed Monday's trading at a new high of $591.02, up 0.78%.

Progenity Inc. (PROG) closed Monday's trading at a new low of $4.27, down 14.43%.

Teligent Inc. (TLGT) closed Monday's trading at $0.47, down 10.30%.