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12.12.2017 07:06:22

In The Red: ONCE Dims, SYRS On Watch, OVID Waiting For STARS, JUNO Bleeds

(RTTNews) - The following are some of the stocks that lost the largest percentage in price today in the pharma/biotech sector.

1. Spark Therapeutics (ONCE)

Lost 34.97% to close Monday's (Dec.11) trading at $47.72.

News: The Company announced that preliminary data from its Phase 1/2 clinical trial of SPK-8011 in hemophilia A showed a 100 percent reduction in annualized bleeding rate and 98 percent reduction in annualized infusion rate in the first four participants.

But investors were disappointed with the mean factor VIII activity level associated with SPK-8011 as it pales in comparison to BioMarin Pharmaceutical Inc's (BMRN) Valoctocogene Roxaparvovec, which is also an investigational gene therapy for Hemophilia A.

As of the Dec. 6, 2017, data cutoff, two participants have received a single administration of SPK-8011 at an initial dose of 5 x 1011 vector genomes and have now been followed for more than 40 weeks and 30 weeks. During this time, after week 12, participant one has achieved sustained expression with a mean factor VIII activity level of 10%. Participant two has shown different kinetics of factor VIII expression. His factor VIII level, as of the data cutoff, is 37 percent, and the mean factor VIII level since week 12 is 16%, according to the Company.

In a phase 1/2 clinical study of BioMarin's Valoctocogene Roxaparvovec, three patients with the longest follow-up (at week 48), had Factor VIII activity levels of 49%.

2. Syros Pharmaceuticals Inc. (SYRS)

Lost 31.69% to close Monday's trading at $8.45.

News: The Company announced initial clinical data from its ongoing phase II trial of SY-1425 in genomically defined subsets of patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).

Since the data support the continued development of SY-1425 in combination with other therapies, the Company does not plan to pursue further development of SY-1425 as a single agent.

Enrollment in the phase II study of SY-1425 in a single-agent cohort in newly diagnosed AML patients who are not suitable candidates for standard chemotherapy is being stopped.

Clinical trials & Near-term catalysts:

-- A phase II study evaluating SY-1425 in combination with chemotherapy drug Vidaza (azacitidine) in newly diagnosed AML patients who are not suitable candidates for standard chemotherapy is underway. -- Syros recently added a cohort in relapsed or refractory AML and higher-risk MDS patients to evaluate SY-1425 in combination with anti-cancer drug Darzalex (daratumumab) and expects to begin enrolling patients in that cohort in early 2018. -- The initial clinical data from the two combination cohorts are expected in 2018.

3. Juno Therapeutics Inc. (JUNO)

Lost 14.34% to close Monday's trading at $50.24.

News: The Company presented updated data from the ongoing phase I TRANSCEND study of JCAR017. Looks like investors were not impressed by the results.

JCAR017 is the Company's CAR-T therapy candidate. The TRANSCEND study is a Phase 1 trial evaluating JCAR017 in adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL), primary mediastinal B-cell lymphoma, follicular lymphoma Grade 3B, and mantle cell lymphoma.

The core group in the trial includes 29 patients who received dose level two (DL2 = 100 million cells), 34 patients who received dose level one (DL1 = 50 million cells) and 4 patients who received dose level one twice, approximately 14 days apart.

According to the updated data, at Dose level 2, the 3-month overall response rate (ORR) was 74% and 3-month complete response (CR) rate was 68%. Of patients that have reached 6 months of follow-up, 50% were in complete response. Across doses, 80% of patients with CR at 3 months stayed in CR at 6 months, and 92% of patients in response at 6 months remained in response as of the October 9, 2017, data cutoff. Across doses, the median duration of response (DOR) was 9.2 months and median durability of CR was not reached, added the Company.

4. Arsanis Inc. (ASNS)

Lost 12.53% to close Monday's trading at $12.22.

News: No news

Recent event:

On November 16, 2017, the Company went public on the NASDAQ Global Market offering its shares at a price of $10 each.

Clinical trials & Near-term catalysts:

-- The Company's investigational lead product candidate is ASN100, which is currently in a Phase 2 clinical trial for the prevention of S. aureus pneumonia in high-risk, mechanically ventilated patients.

-- The trial is expected to be completed in December 2018.

5. Novan Inc. (NOVN)

Lost 11.16% to close Monday's trading at $4.30.

News: No news

Clinical trials & Near-term catalysts:

The Company's pipeline includes...

-- SB206, which is under phase II trial for genital warts; under phase I study for Molluscum and under phase I study for HPV -associated sexually transmitted infections. -- NVN3100, which is under pre-clinical testing for anal neoplasia and cervical neoplasia. -- SB414, which is under phase Ib study for psoriasis, and under phase Ib trial for atopic dermatitis. -- SB204, under late-stage development for acne. The Company plans to complete development of SB204 via 3rd party funding and execution. -- SB208, under phase II study for Onychomycosis Tinea Pedis.

6. Ovid Therapeutics Inc. (OVID)

Lost 10.99% to close Monday's trading at $10.45.

News: No news

Clinical trials & Near-term catalysts:

The Company's lead drug candidate is OV101, under phase II trial in adults and adolescents diagnosed with Angelman syndrome or Fragile X syndrome.

Angelman syndrome, which requires life-long care, is a rare genetic disorder characterized by developmental delay, lack of speech, seizures, and walking and balance disorders.

The data from the trial, dubbed STARS, is expected to be available in the second half of 2018.

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