Search the Site

Twitter

test tubes

04/11/16 Catalyst Database Summary

publication date: Apr 11, 2016

04/11/16 Catalyst Summary

ICPT, DRRX, CYTR, PRTK, PGNX, FOLD, DVAX, RTRX, ACAD ,  ADMP, AMPE, AEZS, KERX, and PBYI  TEST

 

 

ICPT

       

Intercept Pharma

       

Price: $156.31

       

Market Cap: $3942

       

FDA Decision (NDA)

ICPT has a PDUFA decision goal date of 5/29/16 for NDA seeking approval for the treatment of primary biliary cirrhosis (PBC) with FDA Fast Track and US/EU Orphan Drug status. On 4/7, the FDA advisory panel voted 17-0 in favor of approval.

         

DRRX

       

Durect

       

Price: $1.26

       

Market Cap: $145

       

Pivotal Phase 3 Clinical Trial

DRRX has a (ClinicalTrials.gov ID NCT02574520) with results originally expected in late 2016-early 2017 for pivotal Phase 3 (PERSIST) trial. It is being evaluated for post-op (gallbladder removal) extended pain control following single instillation within surgical incision to support a planned NDA resubmission in response to February14 CRL following mixed results in previous Phase 3 trials and return of US (Hospira) and European (Takeda) rights from previous partners. In early April16, DRRX reported FDA letter advising changes to trial design including use of standard bupivacaine as active control which will add time and cost to complete trial with update on new timeline pending completion of study protocol changes.

         

CYTR

       

CytRx

       

Price: $3.14

       

Market Cap: $217

       

Phase 2 and 3 Clinical Trials

CYTR has a (ClinicalTrials.gov ID NCT02049905) for fully enrolled pivotal Phase 3 second-line sarcoma trial under SPA with progression-free survival (PFS) primary outcome data expected in June16 after target events achieved in early April16. It also has an ongoing Phase 2 brain cancer (GBM) trial with overall survival (OS) data expected 2nd half of 2016. CYTR also expects to report Phase 2 lung cancer (SCLC) data during 2nd half of 2016. It expects to complete Phase 1B trials during 2nd half of 2016 evaluating combination regimens for the treatment of advanced sarcomas and other solid tumors. It has FDA Orphan Drug status for GBM, SCLC, and ovarian cancer. CYTR plans to begin clinical trials for DK049 during 2nd half of 2016 (novel formulation of anti-cancer drug gemcitabine designed for targeted and increased tumor drug levels).

         

PRTK

       

Paratek Pharma

       

Price: $15.11

       

Market Cap: $266

       

Pivotal Phase 3 Clinical Trials under SPA

PRTK has a (ClinicalTrials.gov ID NCT02378480) with topline data expected around end of June16 for first of two fully enrolled (as of early April16) pivotal Phase 3 trials under SPA for treatment of acute bacterial skin and skin structure infections (ABSSSI) vs. approved antibiotic ZYVOX (linezolid). It also has (CT.govID NCT02531438) for second Phase 3 trial with topline data expected during 2nd half of 2017 for the treatment of community-acquired bacterial pneumonia (CABP), FDA QIDP and has Fast Track status for ABSSSI, CABP and cUTI.

         

PGNX

       

Progenics Pharma

       

Price: $4.76

       

Market Cap: $348

       

FDA Decision (NDA)

PGNX has a PDUFA decision goal date of 7/19/16 under standard review and three month extension announced in early April16 to review responses to FDA info requests for NDA seeking approval for an oral tablet formulation in adults with opioid-induced constipation (OIC) with chronic non-cancer pain. It has FDA approved for OIC as a subcutaneous injection and is partnered with $VRX. PGNX will receive $50M for FDA approval, 15-19% royalties, $10M sales milestone payment for first $100M in sales, and 60% of any cash received by $VRX for ex-US licensing agreements.

         

FOLD

       

Amicus Therapeutics

       

Price: $8.11

       

Market Cap: $956

       

FDA New Filing (NDA)

On 4/1/16, FOLD received positive CHMP opinion for MAA filing to EMA seeking EU approval for the treatment of Fabry disease in patients with amenable mutations (includes 269 Fabry mutations or approximately 50% of entire patient population) with final approval expected within 67 days by early June. It will provide update on NDA filing timeline after planned FDA meeting during 2nd quarter of 2016.

         

DVAX

       

Dynavax

       

Price: $21.76

       

Market Cap: $834

       

FDA Decision (BLA Resubmission)

DVAX has a PDUFA decision goal date of 9/15/16 under six-month Class 2 review for BLA resubmission in response to CRL issued by FDA in February13 seeking approval for immunization against hepatitis B virus (HBV) infection in adults age 18+.

         

RTRX

       

Retrophin

       

Price: $14.8

       

Market Cap: $540

       

Phase 2 Clinical Trial

RTRX has a (ClinicalTrials.gov ID NCT01613118) for fully enrolled (as of late MARCH16) Phase 2 (DUET) trial for treatment of rare kidney disease (FSGS) (US and EU Orphan Drug status for this indication) with topline results expected during 3rd quarter of 2016. It is licensed from $LGND.

         

ACAD

       

ACADIA Pharma

       

Price: $31.78

       

Market Cap: $3526

       

FDA Decision (NDA) and Phase 2 Clinical Trial

ACAD has a PDUFA decision goal date of 5/1/16 under priority review seeking FDA approval for treatment of Parkinson's disease psychosis (PDP) with FDA Breakthrough Therapy designation for this indication. On 3/29, an FDA advisory panel voted 12-2 in favor of approval (overall benefit vs. safety risk profile for treatment of PDP). ACAD plans to file for approval in Europe (MAA and EMA) during 2nd quarter of 2016. It expects to report topline data during 2nd half of 2016 for Phase 2 Alzheimer’s disease (AD) psychosis trial.

         

ADMP

       

Adamis Pharma

       

Price: $6.52

       

Market Cap: $90

       

FDA Decision (NDA Resubmission)

ADMP has a PDUFA decision goal date of 6/4/16 under six-month Class 2 review for NDA resubmission seeking approval for novel formulation of approved drug/device product (e.g. EpiPen and others) for the emergency treatment of severe allergic reactions (anaphylaxis). In MARCH15, ADMP received CRL citing CMC issues related to volume of dose delivered by syringe and subsequently redesigned, tested, and validated its product candidate then discussed with FDA before commercial manufacturing scale-up. In MARCH16, ADMP acquired privately held US Compounding ($20M+ revenue past two years) with expected closing in 2nd quarter of 2016 and will issue 1.62M shares and assume secured debt.

         

AMPE

       

Ampio Pharma

       

Price: $3.2

       

Market Cap: $171

       

Pivotal Phase 3 Clinical Trial under SPA

AMPE has a (ClinicalTrials.gov ID NCT02556710) with results expected in mid-2016 for second of two pivotal Phase 3 (PIVOT) trials under SPA for treatment of knee osteoarthritis pain after a single injection into the knee joint.

         

AEZS

       

Aeterna Zentaris

       

Price: $3.6

       

Market Cap: $37

       

Pivotal Phase 3 Clinical Trial

AEZS has a (ClinicalTrials.gov ID NCT02558829) with expected completion during 3rd quarter of 2016 for confirmatory Phase 3 trial to support planned NDA resubmission by mid-2017 in response to CRL issued by FDA. In NOVEMBER14, AEZS is seeking FDA approval as oral test for adult growth hormone deficiency with FDA Orphan Drug status for this indication. In NOVEMBER15, AEZS conducted 1:100 reverse stock split.

         

KERX

       

Keryx Biopharma

       

Price: $4.93

       

Market Cap: $507

       

FDA New Filing (sNDA)

In late Marchch16, KERX reported positive pivotal Phase 3 data which met primary and secondary endpoints for treatment of iron deficiency anemia in pre kidney dialysis patients to support a planned sNDA filing during 3rd quarter of 2016 seeking expanded FDA approval. In September14, KERX received FDA approval to control serum phosphorus levels in patients with chronic kidney disease (CKD) on dialysis, and in September15, it received EU approval.

         

PBYI

       

Puma Biotech

       

Price: $30.78

       

Market Cap: $966

       

Phase 2 and 3 Clinical Trials, FDA New Filing (NDA)

PBYI expects to file NDA seeking FDA approval in mid-2016 as an extended adjuvant treatment of HER2(+) early stage breast cancer (BC) which was delayed from previous guidance of 1st quarter of 2016 in late MARCH16 to amend statistical analysis plan based on FDA feedback. PBYI expects to report additional Phase 2 data during 1st half of 2016 for same indication with loperamide prophylaxis to address diarrhea side effect along with Phase 2 data 1st half of 2016 from Mammaprint High subgroup. It expects to report Phase 2 data in mid-2016 from HER2(-) BC trial and Phase 3 data in late 2016-early 2017 for third-line treatment of HER2(+) BC, expected to report Phase 2 data in BC patients with brain mets and Phase 2 data in HER2 non-amplified BC during 4th quarter of 2016.