BPC December 10 update

Biotech stock events to watch for rest of December; Biotech week in Review

Weekly watchlist

The latest week saw an embarrassment of price-moving news releases, a level not seen for at least this year. A sample of the main price movers are listed below, followed by a list of upcoming catalysts to watch over the next three weeks.

REGULATORY:

Pfizer Inc. (NYSE:PFE) and BioNTech SE (NASDAQ:BNTX) announced late-Friday the FDA issued an Emergency Use Authorization (EUA) for their COVID-19 vaccine, BNT162b2.

Lipocine Inc. (NASDAQ:LPCN) made a remarkable website gaffe in regard to its New Drug Application for Tlando. Published on their website was a post that noted Tlando had been approved by the FDA as an oral testosterone replacement therapy (TRT). This initially saw shares climb over 20% in Tuesday’s after hours’ session, only for the company to later issue a press release that an independent contractor had mistakenly posted the information to the website without authorization and as a result the company deleted the posting. Lipocine later announced that the FDA had issued "tentative" approval but is not eligible for final approval until the expiration of the exclusivity period previously granted to Clarus Therapeutics, Inc. with respect to Jatenzo, which expires on March 27, 2022. Shares closed Wednesday down 21% to $1.32.

Chimerix (NASDAQ:CMRX) shares rallied to close Monday up 25% to $4.54 following news the FDA accepted the filing of its New Drug Application (NDA) for brincidofovir (BCV) as a medical countermeasure for smallpox and set a PDUFA date under Priority Review of April 7, 2021. Further gains were made throughout the week with shares closing Friday up at $4.93.

scPharmaceuticals Inc. (NASDAQ:SCPH) announced Monday it received a Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA) for Furoscix. Shares fell by 36% to $5.62 on the news.

CLINICAL DATA:

TG Therapeutics, Inc. (NASDAQ:TGTX) shares closed Thursday up 41% to $41.72 on news that it met the primary endpoints in its two Phase 3 trials evaluating ublituximab, compared to teriflunomide in patients with relapsing forms of multiple sclerosis (RMS).

Curis, Inc. (NASDAQ: CRIS) announced promising data from its Phase 1 trial of CA-4948 in patients with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS). Marrow blast reductions were observed in all evaluable patients (6 patients). Two patients experienced a marrow complete response. Shares surged to close the week up 320% to $7.89.

Pluristem Therapeutics Inc. (NASDAQ:PSTI) shares fell Wednesday 39% to $7.00 on news it will be discontinuing its Phase 3 trial for the treatment of critical limb ischemia (CLI) following interim analysis from an independent Data Monitoring Committee (DMC) that the trial is unlikely to meet the primary endpoint by the time of the final analysis.

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) announced the release of promising data from its Phase 1 trial in patients with Danon disease and followed the release with a public offering of $260m shares to close the week up 86% to $59.57.

Gamida Cell Ltd. (NASDAQ:GMDA) shares closed the week up 42% to $9.92 following the release of data from its Phase 1 trial of GDA-201 for the treatment of patients with non-Hodgkin lymphoma (NHL) and multiple myeloma (MM). In 19 patients with NHL, an overall response rate of 74% and a complete response rate of 68% were recorded.

Oncternal Therapeutics, Inc. (Nasdaq: ONCT) announced updated interim clinical data from the ongoing Phase 1/2 trial in patients with mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL). The company noted a best objective response rate of 87% reported for 15 patients with relapsed/refractory mantle cell lymphoma, with a median follow-up of 12.1 months. Shares closed the week up 95% to $5.20.


Biotech stock events to watch for rest of December:

Drug Stage Catalyst Market Cap

APRE – Aprea Therapeutics Inc.
APR-246 (eprenetapopt) and azacitidine
TP53 mutant Myelodysplastic syndromes (MDS)

Phase 3 Phase 3 top-line data did not meet primary endpoint - December 28, 2020.
$124.4 million

ARPO – Aerpio Pharmaceuticals Inc.
Razuprotafib (AKB-9778)
Open-angle glaucoma (OAG)

Phase 2 Phase 2 data met primary endpoint with twice-daily dose group but not with once-daily group.
$61.7 million

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

Phase 2 Phase 2 data released December 16, 2020. ORR 21%; PFS 4 months; OS 6.5 months.
$60 million

CALA – Calithera Biosciences Inc.
CB-839 + Cabozantinib (CANTATA)
Renal cell carcinoma

Phase 2 Phase 2 top-line data did not meet primary endpoint - January 4, 2020.
$206.7 million

FGEN – FibroGen Inc
Roxadustat
Anaemia in Chronic Kidney Disease

PDUFA PDUFA date extended by three months to March 20, 2021.
$4.1 billion

GOSS – Gossamer Bio Inc.
GB002
Pulmonary arterial hypertension (PAH)

Phase 1/2 Phase 2 enrollment to commence 4Q 2020 with top-line data due 1H 2022.
$771.4 million

MRNA – Moderna Inc.
mRNA-1273
COVID-19 vaccine

Approved FDA approval (EUA) announced December 18, 2020.
$52.6 billion

MYOV – Myovant Sciences Ltd.
Relugolix
Advanced prostate cancer

Approved FDA approval announced December 18, 2020.
$1.9 billion

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
Alstrom Syndrome and Bardet-Biedl Syndrome

Phase 3 Phase 3 top-line data met primary endpoint - December 22, 2020. All primary endpoint responders were patients with BBS. Alstrom syndrome patients did not meet primary endpoint.
$1.6 billion

SIOX – Sio Gene Therapies Inc.
AXO-AAV-GM1
GM1 gangliosidosis

Phase 1 Phase 1 positive six-month data from low dose cohort released December 15, 2020.
$139.6 million

SNGX – Soligenix Inc.
SGX942 (dusquetide)
Oral Mucositis in Patients with Head & Neck Cancer

Phase 3 Phase 3 trial did not meet primary endpoint - December 22, 2020.
$50.4 million

VTVT – vTv Therapeutics Inc.
Azeliragon
Mild Alzheimer’s disease with type 2 diabetes

Phase 2 Phase 2 top-line data did not meet primary endpoint - December 15, 2020.
$165.4 million