Q4 2014 NDA/BLA Round-up Part I: So Deoxycholic Acid Can Remove My Fat?

For project 6 at PSIC, we shift our time horizon longer and decide to look at candidates which will have NDA/BLA filing due this quarter. This means the FDA decision will come 6-9 months later after our research.

For this round, we have 1 PDUFA around May and 14 NDA/BLA filing due Q42014. A round-up of these company and their drug candidates is summarized below:

Q4 2014 NDA/BLA Round-up Part I

Ticker $ Cap Drug Disease
KYTH 32.85 745M ATX101 (deoxycholic acid) Injectable treatment for the reduction of submental fat.
RLYP 18.72 636M PATIROMER (RLY5016) Hyperkalemiato, which is abnormally high blood K.
ACAD 26.06 2.6B Pimavanserin Parkinson’s disease psychosis.
JNJ 98.71 278B 3-month INVEGA SUSTENNA Schizophrenia

Developed by Janssen R&D

BDSI 15.95 786M BEMA Buprenorphine Chronic pain
LLY 62.58 70B CYRAMZA (ramucirumab)_BLA Metastatic colorectal cancer
LLY-2 62.58 70B Necitumumab_BLA Non-small cell lung cancer
Roche 283.9 195B Cobimetinib + Vemurafenib malignant melanoma


KYTH: Kythera Biopharmaceuticals

The company focuses on aesthetic medicine market

Pipeline has only one drug: ATX101

ATX-101 is a synthetic version of deoxycholic acid

ATX-101 is an injectable drug to reduce submental fat (commonly known as double chin)

ATX-101 filed FDA in US. The PDUFA date is May/13/2015

RLYP: Patiromer (RLY5016)

RLYP: Relypsa, Inc.

Relypsa focuses on nonabsorbed polymeric drugs to treat renal, cardiovascular and metabolic diseases.

The pipeline has two drugs, with Patiromer as a leading drug.

Patiromer is a non-absorbed, potassium-binding polymer.

Patiromer will bind K in colon to increase K secretion in the colon.

Patiromer is developed to treat hyperkalemia (abnormally high level of K in the blood).

NDA is expected in early Q4.

ACAD: Pimavanserin

ACAD: Acadia Pharmaceuticals Inc.

Acadia Pharmaceuticals focuses on neurological and related central nervous system disorders.

The pipeline has five drugs, with pimavanserin as its leading drug, developed for three indications. Pimavanserin in Parkinson’s disease psychosis is the first indication which went through phase 3 clinical trials. The other two indications are schizophrenia and Alzheimer’s Disease Psychosis, which are in phase 2 clinical trials.

Pimavanserin is a chemical entity as a selective serotonin inverse agonist, preferentially targeting 5-HT2A receptors.

Granted Breakthrough Therapy designation by FDA. Plan to file NDA at the end of 2014.

JNJ & ALKS: 3-month Invega Sustenna

JNJ: Johnson & Johnson
ALKS: Alkermes

A combination of previous approved drug and new delivery technology.

Invega Sustenna was approved for once monthly injection for treating schizophrenia.

Alkermes has developed a NanoCrystal technology, which enables solubility of poorly water-soluble compounds. (My understanding: Break down insoluble drug molecules into nano size particles and stabilize it in water as a suspension solution.)

JNJ has tested 3 month formulation (injection at once 3-month?) for Invega Sustenna + NanoCrystal technology.

Based on positive reviews from Independent Data Monitoring Committee, JNJ halted the Phase 3 study early and planned to file NDA at the end of 2014.

BDSI: BEMA Buprenorphine

BDSI: BioDelivery Sciences International, Inc.

BioDelivery Sciences International focuses on pain and addiction.

BioDelivery Sciences uses BEMA technology as its core technology for drug delivery.

BEMA is a small, bioerodible polymer film for mucosal membranes (inside mouth) delivery for drugs. Buprenorphine is an opioid receptor modulator that is used to treat opioid addiction in high dosages, and to control acute-to-moderate pain in lower doses. BEMA Buprenorphine is developed for treatment of moderate to severe chronic pain.

Based on BEMA, there are four drugs in its pipeline, with one on market, one approved by FDA in Jun 2014, one (BEMA Buprenorphine) ready to file NDA and one in Phase II study.

Positive outcome of the pre-NDA meeting. Expect to file NDA in late 2014.

LLY and DYAX: Cyramza (ramucirumab)

LLY: Eli Lilly
DYAX: Dyax Corp.

DYAX has a phage display library platform to display therapeutic protein/peptide (perticularly human mAb) on phage and screen against potential therapeutic targets. Ramucirumab is a human mAb, against VEGFR2 to target angiogenesis.

Cyramza (Ramucirumab) is developed by LLY for treating various solid tumors.

Cyramza is approved for treating advanced gastric cancer after prior chemotherapy.

Phase 3 study of Cyramza in Non-small cell lung cancer has been done and this indication is ready for filing BLA at the end of 2014

LLY and DYAX_2: Necitumumab

Similar story as Cyramza.

Necitumumab is a mAb binding to EGFR.

Necitumumab is developed by LLY for clinical trials.

Phase 3 study of Necitumumab in Non-small cell lung cancer has been done and this indication is ready for filing BLA at the end of 2014

Roche and EXEL: Cobimetinib + Vemurafenib

Vemurafenib is marketed drug, developed by Genentech.

Cobimetinib is first discovered by EXEL (Exelixis) and now under co-development with Genentech (Exelixis is responsible for phase I, and Genentech is responsible for later phases; both companies share the profits and losses if marketed).

Vemurafenib and Cobimetinib are both MEK (as known as MAPKK) inhibitors.

Positived top-line results for phase 3 study of Cobimetinib + Vemurafenib in treating metastatic melanoma.

Ready to file NDA at the end of 2014.