BPC September 03 update

Akebia AKBA shares plunge on failed Phase 3 trial -74%; ​Applied Genetic AGTC adds +13% ahead of updated data

Price and Volume Movers

Applied Genetic Technologies Corporation (NASDAQ:AGTC) shares closed up 13% to $5.82. The company is due to release updated data from its Phase 1/2 trial of AGTC-501 in patients with X-linked Retinitis Pigmentosa (XLRP) during its earnings call on September 9.

Akebia Therapeutics, Inc. (Nasdaq: AKBA) shares plunged to close down 74% to $2.65 following the release of data from its Phase 3 PRO2TECT trial, the second of its two global Phase 3 cardiovascular outcomes programs evaluating vadadustat for the treatment of anemia due to chronic kidney disease (CKD) in adult patients not on dialysis. Vadadustat did not meet the primary safety endpoint which measured vadadustat versus darbepoetin alfa in time to first occurrence of major adverse cardiovascular events (MACE).

Auris Medical Holding Ltd. (NASDAQ: EARS) shares closed up 22% to $1.01 following the release of top-line data from its Phase 2 TRAVERS trial of AM-125 in patients with vertigo. The treatment demonstrated a dose-dependent improvement in balance as well as good safety and tolerability of ascending doses.

Amarin Corporation plc (NASDAQ:AMRN) shares, which closed Wednesday down 31% to $5.04 following the commencement of its court appeal to review its patent for Vascepa against two generic drugmakers, added to losses Thursday, closing down a further 10% to $4.56. The company announced today the court upheld a previous ruling in favor of the two generic companies.

Savara Inc. (NASDAQ:SVRA) announced it will discontinue its Phase 2a ENCORE trial of Molgradex for the treatment of nontuberculous mycobacterial (NTM) lung infection in people living with cystic fibrosis (CF). The company noted factors such as the impact of COVID-19 on patient recruitment for its decision. Shares are trading down 15% to $1.17 after hours.

ObsEva SA (NASDAQ:OBSV) announced the pricing of an underwritten equity offering and concurrent private placement transaction. The offering of 6,448,240 units at an effective price of $2.869 per unit, has warrants attached. Gross proceeds of $18.5m are expected in addition to a private placement of $1.5m.

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Major price movers (stocks priced > $1.00, volume > 50k):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume:

ADVANCERS:

TRACON Pharmaceuticals, Inc. (TCON): $2.83; +16%.

Sutro Biopharma, Inc. (STRO): $13.00; +15%.

CNS Pharmaceuticals, Inc. (CNSP): $2.24; +13%.

Inhibrx, Inc. (INBX): $18.39; +10%.

Rexahn Pharmaceuticals, Inc. (REXN): $1.93; +8%.

DECLINERS:

Cyclerion Therapeutics, Inc. (CYCN): $6.38; -17%.

Sunesis Pharmaceuticals, Inc. (SNSS): $1.43; -15%.

Recro Pharma, Inc. (REPH): $2.32; -14%.

Syros Pharmaceuticals, Inc. (SYRS): $11.96; -14%.

Unum Therapeutics Inc. (UMRX): $2.16; -14%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AKBA – Akebia Therapeutics Inc.
Vadadustat - PRO2TECT
Non-dialysis patients with anemia related to CKD (NDD-CKD)

Phase 3 Phase 3 trial met efficacy primary endpoint but did not meet primary safety MACE endpoint.
$401.3 million

BYSI – BeyondSpring Inc.
Plinabulin (Trial 103) - DUBLIN-3
Non small cell lung cancer - refractory

Phase 3 Phase 3 final data due 1H 2021.
$464.4 million

BYSI – BeyondSpring Inc.
Plinabulin (Trial 105)
Chemotherapy-induced neutropenia (CIN)

Phase 3 Phase 3 final data and NDA filing due 4Q 2020.
$464.4 million

EARS – Auris Medical Holding Ltd.
AM-125
Vertigo

Phase 2 Phase 2 interim data released September 3, 2020 noting that improvement from baseline was statistically significant.
$5.6 million

ETNB – 89bio Inc.
BIO89-100
Severe hypertriglyceridemia

Phase 2 Phase 2 trial initiation announced September 3, 2020.
$430.3 million

INZY – Inozyme Pharma Inc.
INZ-701
ENPP1 deficiency

Phase 1/2 Phase 1/2 trial planned for early 2021.
$560.1 million

MDGL – Madrigal Pharmaceuticals Inc.
Resmetirom - MAESTRO-NAFLD-1
Non-Alcoholic Fatty Liver Disease

Phase 3 Phase 3 52-week top-line data due by end of 2021. Open label data to be presented by end of 2020.
$1.9 billion

PRTA – Prothena Corporation plc
PRX002 (PASADENA)
Parkinson's disease

Phase 2 Phase 2 (Part 1) trial did not meet primary endpoint - April 22, 2020. Data presented September 11, 2020.
$425.5 million

SVRA – Savara Inc.
Molgradex - ENCORE
Nontuberculous mycobacterial (NTM) lung infection with cystic fibrosis (CF)

Phase 2a Phase 2a trial to be discontinued - noted September 3, 2020.
$68.7 million

TNXP – Tonix Pharmaceuticals Holding Corp.
TNX-102 SL (Tonmya)
Fibromyalgia

Phase 3 Phase 3 interim analysis due September 2020. Top-line data due 4Q 2020. Initiation of second Phase 3 trial announced September 3, 2020.
$120.8 million