BPC March 15 update

Biotech Stock events slated for the final two weeks of March; Biotech week in Review

Weekly watchlist

The latest week continued to see a steady flow of clinical readouts, while speculative price action among nano-cap stocks continued. Head of the list was Atossa Genetics Inc. (Nasdaq: ATOS), which issued a press release headlined “Atossa Genetics Announces FDA Approval of Oral Endoxifen for “Expanded Access” as Post-Mastectomy Treatment for a U.S. Breast Cancer Patient”.

A number of investors trading on headline-only news, no doubt would have assumed that the company had been issued marketing approval, yet towards the end of its press release the company noted under the FDA expanded access IND program, the use of Atossa's proprietary oral Endoxifen is restricted solely to this (one) patient.”

Many nano-cap companies are taking advantage of recent enthusiasm for nano-cap biotechs by releasing minor news but including specific keywords, such as "FDA" and "approval" to attract traders, knowing that nano-caps (sub $50m) with low share floats can be moved relatively easily.

Let’s review some of the other news from the week that was:

Karyopharm Therapeutics Inc. (Nasdaq: KPTI) announced the PDUFA date for its New Drug Application (NDA) for selinexor, for the treatment of patients with relapsed refractory multiple myeloma, has been extended by three months to July 6, 2019. Previously, an FDA Advisory Committee (ODAC) voted 8 to 5 recommending that the FDA wait for the results from its Phase 3 BOSTON trial, due late-2019/early-2020, before making a final decision regarding approval. Shares closed Friday up 7% to $4.71.

Aerie Pharmaceuticals, Inc. (NASDAQ:AERI) announced after-hours Tuesday that the FDA approved Rocklatan 0.02%/0.005% to reduce elevated intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension. Shares closed Wednesday up 5% to $47.97.

Zafgen, Inc. (Nasdaq:ZFGN) shares closed the week down 36% to $3.01 following news late-Monday that it will suspend plans to file an investigational new drug (IND) application for ZGN-1258, its pipeline candidate for Prader-Willi syndrome (PWS), based on findings in muscle tissue in rodent toxicology studies.

Seres Therapeutics, Inc. (Nasdaq: MCRB) shares closed Monday up 25% to $6.38 on news of a research collaboration with AstraZeneca, focusing on cancer immunotherapy. It will receive $20m in three equal installments over two years, with the first payment due at the start of the agreement.

Spectrum Pharmaceuticals, Inc. (NASDAQ-GS: SPPI) announced Friday that it has withdrawn its Biologics License Application (BLA) for Rolontis due to the FDA’s request for additional manufacturing-related information.

Updated list of biotech stock events slated for final two weeks of March:

Drug Stage Catalyst Market Cap

ALDX – Aldeyra Therapeutics Inc.
Reproxalap (ADX-102) - INVIGORATE
Allergic conjunctivitis

Phase 3 Phase 3 trial to be initiated 1H 2020.
$132.2 million

AMRN – Amarin Corporation plc
High Triglycerides With Mixed Dyslipidemia

PDUFA priority review PDUFA date December 28, 2019. Advisory Committee meeting November 14, 2019 voted 16-0 in favor.
$8.1 billion

ARPO – Aerpio Pharmaceuticals Inc.
AKB-9778 - TIME 2B
Non-Proliferative Diabetic Retinopathy

Phase 2 Phase 2 data released March 18, 2019 did not meet primary endpoint.
$22.7 million

DERM – Dermira Inc.
Atopic Dermatitis

Phase 3 Phase 3 trial initiation announced October 9, 2019. Top-line data due 1H 2021.
$429.6 million

GLPG – Galapagos NV
Rheumatoid arthritis (RA)

NDA Filing NDA filing due 4Q 2019.
$13.8 billion

LPTX – Leap Therapeutics Inc.
DKN-01 and Paclitaxel
Endometrial Cancer

Phase 2 Phase 2 updated data September 20, 2019 at IGCS.
$19.2 million

LXRX – Lexicon Pharmaceuticals Inc.
Type 1 Diabetes

CRL CRL issued March 22, 2019. Appeal rejected by FDA - December 2, 2019.
$517.5 million

PRQR – ProQR Therapeutics N.V.
Epidermolysis bullosa

Phase 1/2 Phase 1/2 interim analysis announced March 26, 2019. Future development to be conducted by Wings Therapeutics.
$323.4 million

PRTO – Proteon Therapeutics Inc.
Vonapanitase (PRT-201) PATENCY-2
Chronic kidney disease (CKD) patients undergoing surgical placement of an arteriovenous fistula (AVF)

Phase 3 Phase 3 data released Match 28, 2019 did not meet primary endpoint.
$6.6 million

PTI – Proteostasis Therapeutics Inc.
PTI-428 + PTI-801 + PTI-808
Cystic fibrosis

Phase 2 Phase 2 data due 4Q 2019.
$181.5 million

REPH – Recro Pharma Inc.
Intravenous (IV) meloxicam
Acute pain following bunionectomy surgery

CRL First CRL announced May 24, 2018. Second CRL issued March 22, 2019. Intends to appeal decision.
$375.1 million

SAGE – Sage Therapeutics Inc.
Brexanolone - SAGE-547 (202C)
Postpartum Depression - moderate

Approved FDA approval announced March 19, 2018.
$3.3 billion

SGEN – Seattle Genetics Inc.
Enfortumab vedotin
Urothelial cancer

PDUFA priority review PDUFA date under priority review is March 15, 2020.
$20.1 billion

UROV – Urovant Sciences Ltd.
Overactive bladder (OAB)

NDA Filing NDA filing early 2020.
$387.7 million

UTHR – United Therapeutics Corporation
Esuberaprost - BEAT
Pulmonary arterial hypertension (PAH)

Phase 3 Phase 3 data released April 8, 2019 did not meet primary endpoint.
$4 billion