BPC August 06 update

​Allakos ALLK shares rally for second day +22%; Regeneron REGN Sanofi SNY positive atopic dermatitis data

Price and Volume Movers

Allakos Inc. (Nasdaq: ALLK) shares, which surged Monday to close up 111%, added a further 22% Tuesday to close up at $79.47. The company announced yesterday that it met both primary and secondary endpoints in its Phase 2 trial of AK002 in patients with eosinophilic gastritis and/or eosinophilic gastroenteritis.

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi (NASDAQ: SNY) announced that their pivotal Phase 3 trial evaluating Dupixent (dupilumab) to treat severe atopic dermatitis in children aged 6 to 11 years met its primary and secondary endpoints. 33% of patients who received Dupixent every four weeks and 30% of patients who received Dupixent every two weeks achieved clear or almost clear skin, compared to 11% for placebo (p<0.0001 and p=0.0004, respectively).

Aquinox Pharmaceuticals, Inc. (NASDAQ:AQXP) and Neoleukin Therapeutics, Inc. announced that the two companies will merge and Aquinox will be renamed as Neoleukin Therapeutics, Inc., under the new ticker symbol “NLTX”. Following completion of the merger, former Aquinox stockholders will own approximately 61.42% of the combined company, while former Neoleukin stockholders will own 38.58%. The merger is expected to close on or about August 8.

Akcea Therapeutics, Inc. (NASDAQ: AKCA) and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), announced top-line data from their Phase 3 trial of Waylivra in patients with familial partial lipodystrophy (FPL). The study met its primary endpoint demonstrating a statistically significant reduction in triglyceride levels in patients with FPL compared to placebo.

The FDA released a statement regarding the approval in May of Zolgensma, a gene therapy product marketed by Novartis (NYSE: NVS) intended to treat children less than two years of age with spinal muscular atrophy (SMA). The FDA announced that in late-June, following approval of the product, the agency was informed by AveXis Inc. (acquired by Novartis), the product’s manufacturer, about a data manipulation issue that impacts the accuracy of certain data submitted in the biologics license application (BLA). Furthermore, the FDA noted that they were aware that AveXis became aware of the data manipulation before the FDA approved the product, yet did not inform the FDA until after the product was approved. The FDA is assessing this situation and remains confident that Zolgensma should remain on the market.

-

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

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

ADVANCERS:

Aeterna Zentaris Inc. (AEZS): $2.47; +18%.

Stemline Therapeutics, Inc. (STML): $16.80; +17%.

Liquidia Technologies Inc. (NASDAQ: LQDA): $6.76; +14%.

DURECT Corporation (DRRX): $1.44; +13%.

ImmunoGen, Inc. (IMGN): $2.80; +12%.

DECLINERS:

Miragen Therapeutics, Inc. (MGEN): $1.33; -29%.

Endo International plc (ENDP): $2.25; -21%.

Leap Therapeutics, Inc. (LPTX): $1.77; -15%.

Mallinckrodt Public Limited Company (MNK): $5.70; -12%.

Provention Bio, Inc. (PRVB): $8.91; -10%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AKCA – Akcea Therapeutics Inc.
Volanesorsen
Familial partial lipodystrophy (FPL)

Phase 3 Phase 3 data met primary endpoint - August 6, 2019.
$2 billion

ALNY – Alnylam Pharmaceuticals Inc.
Lumasiran (ALN-GO1) ILLUMINATE-A
Primary Hyperoxaluria Type 1 (PH1)

Phase 3 Phase 3 data due late-2019.
$9 billion

BCRX – BioCryst Pharmaceuticals Inc.
BCX7353 - APeX-2
Reduce or eliminate attacks in HAE patients

Phase 3 NDA filing due 4Q 2019.
$286 million

BLRX – BioLineRx Ltd.
BL-8040 in combination with KEYTRUDA - COMBAT trial
Pancreatic cancer

Phase 2 Phase 2 top-line data due 2H 2019 with PFS data due mid-2020.
$30.6 million

DOVA – Dova Pharmaceuticals Inc.
Avatrombopag
Chemotherapy-induced thrombocytopenia (CIT)

Phase 3 Phase 3 data due 1H 2020.
$418.2 million

EVLO – Evelo Biosciences Inc.
EDP1815
Psoriasis and Atopic Dermatitis

Phase 1b Phase 2 trial to be initiated early 2020 with initial data due late 2020.
$195.9 million

GMDA – Gamida Cell Ltd.
NiCord (omidubicel)
Acute myeloid leukemia

Phase 3 Phase 3 top-line data due 1H 2020.
$80.4 million

KALA – Kala Pharmaceuticals Inc.
KPI‑121 0.25% STRIDE 3
Dry eye disease

Phase 3 Phase 3 data due 4Q 2019.
$136 million

KMDA – Kamada Ltd.
Alpha-1 antitrypsin (AAT)
Prevention of lung transplant rejection

Phase 1/2 Phase 2 top-line data due YE 2019.
$214 million

KPTI – Karyopharm Therapeutics Inc.
Selinexor - BOSTON
Multiple myeloma

Phase 3 Phase 3 data due end of 2019 or into 2020.
$561.6 million

LPTX – Leap Therapeutics Inc.
DKN-01 + KEYTRUDA
Esophagogastric adenocarcinoma

Phase 1/2 Phase 1/2 data August 6, 2019 noted 50% ORR.
$35.1 million

MCRB – Seres Therapeutics Inc.
SER-109 - ECOSPOR III
Recurrent C. Difficile infection

Phase 3 Phase 3 data due early 2020.
$207.6 million

MNK – Mallinckrodt plc
StrataGraft skin tissue
Deep partial thickness severe burns - skin defects

Phase 3 Phase 3 data due late 2Q or early 3Q 2019.
$342.8 million

REGN – Regeneron Pharmaceuticals Inc.
Dupixent (dupilumab)
Atopic dermatitis 6-11 year-olds

Phase 3 Phase 3 data met endpoints - August 6, 2019.
$32.6 billion