BPC April 28 update

​Protalix PLX issued CRL -33%; Adverum ADVM gene therapy adverse reaction -54% AH

Price and Volume Movers

Protalix BioTherapeutics, Inc. (NYSE American:PLX) announced that the FDA issued a Complete Response Letter (CRL) regarding the Biologics License Application (BLA) seeking approval of pegunigalsidase alfa (PRX‑102) for the proposed treatment of adult patients with Fabry disease. Shares closed down 33% to $3.93. In its initial press release no mention was made for the CRL. However, the company followed up with an afternoon press release where it detailed the CRL did not report any concerns relating to the potential safety or efficacy of PRX–102. However, it mentioned the FDA needs to inspect its manufacturing facility in Israel prior to its decision but was unable to do so due to travel restrictions. Issues were also noted regarding a third-party facility in Europe. Furthermore, the FDA noted that the recent full approval of Fabrazyme by Sanofi must be addressed in the context of any potential resubmission seeking accelerated approval of PRX‑102.

Nymox Pharmaceutical Corporation (NASDAQ:NYMX) announced a private placement of 3,669,724 shares of common stock and warrants to purchase 1,834,862 shares of common stock at a combined purchase price of $2.18 per share for gross proceeds of $8 million. Shares fell 15% to $2.09.

Adverum Biotechnologies, Inc. (NASDAQ:ADVM) shares have slumped after hours, currently trading down 54% to $4.69. The company announced a Suspected Unexpected Serious Adverse Reaction (SUSAR) of hypotony (decrease in ocular pressure) in its Phase 2 Infinity trial evaluating ADVM-022 gene therapy for the treatment of diabetic macular edema (DME).

Amgen Inc. (NASDAQ:AMGN) shares fell 7% to $236.71, initially on the back of Tuesday’s first-quarter earnings report but also following an update of its KRAS G12C inhibitor sotorasib, currently assigned a PDUFA date of August 16, 2021. The dose under review is 960mg but the company announced that the FDA has advised to test the dose against a lower 240 mg daily dose. Data from this trial are due in late-2022. However, the company notes that this will not have any impact on the August 16 timeline.

Revolution Medicines, Inc. (Nasdaq: RVMD), which is developing RMC-4630 with sotorasib, saw its shares fall 21% to $33.80 on the news.

A number of companies announced they will be presenting data at the upcoming American Society for Clinical Oncology (ASCO) conference, of which a selection is noted below in the pipeline updates section. Full abstracts will be released on Wednesday, May 19. The meeting will take place virtually on June 4 - 8, 2021.

-

Major price movers (stocks priced > $1.00, volume > 50k listed on NASDAQ/NYSE):

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

ADVANCERS:

Brooklyn ImmunoTherapeutics, Inc. (BTX): $43.73; +76%.

Galectin Therapeutics Inc. (GALT): $5.10; +23%.

Millendo Therapeutics, Inc. (MLND): $1.17; +19%.

VistaGen Therapeutics, Inc. (VTGN): $2.44; +15%.

Akouos, Inc. (AKUS): $15.96; +14%.

DECLINERS:

Atea Pharmaceuticals, Inc. (AVIR): $6.65; -21%.

Benitec Biopharma Inc. (BNTC): $4.35; -21%.

Reviva Pharmaceuticals Holdings, Inc. (RVPH): $6.39; -12%.

Kintara Therapeutics, Inc. (KTRA): $1.36; -8%

COMPASS Pathways plc (CMPS): $35.92; -8%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ADVM – Adverum Biotechnologies Inc.
ADVM-022 - INFINITY
Diabetic macular edema

Phase 2 Phase 2 update announced Suspected Unexpected Serious Adverse Reaction (SUSAR) of hypotony - April 28, 2021.
$350.6 million

AGEN – Agenus Inc.
AGEN2373
Solid tumors

Phase 1 Phase 1 initial data to be presented at ASCO June 4, 2021.
$725.2 million

APLS – Apellis Pharmaceuticals Inc.
APL-2 PRINCE
Paroxysmal Nocturnal Hemoglobinuria (PNH)

Phase 3 Phase 3 top-line data due 2Q 2021.
$4.3 billion

CALT – Calliditas Therapeutics AB
Nefecon (NefIgArd)
IgA nephropathy

PDUFA priority review PDUFA date under priority review September 15, 2021.
$727.1 million

CLLS – Cellectis S.A.
ALLO-501 - ALPHA
Non-Hodgkin lymphoma (NHL)

Phase 1 Phase 1 further data due at ASCO June 4, 2021.
$669.3 million

IOVA – Iovance Biotherapeutics Inc.
Lifileucel (LN-144)
Refractory metastatic melanoma

Phase 2 Phase 2 updated data from cohort 2 to be presented at ASCO June 6, 2021.
$4 billion

MRUS – Merus N.V.
Zenocutuzumab (MCLA-128)
Solid tumors - NRG1

Phase 1/2 Phase 1/2 interim data to be presented at ASCO June 4, 2021.
$864.4 million

NBTX – Nanobiotix S.A.
NBTXR3 (Study 1100)
Advanced Cancers

Phase 1 Phase 1 updated data due at ASCO June 4, 2021.
$545 million

ONCT – Oncternal Therapeutics Inc.
Cirmtuzumab and Imbruvica (ibrutinib)
Chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL)

Phase 1/2 Phase 1/2 poster to be presented at ASCO June 7, 2021.
$261.7 million

ORIC – Oric Pharmaceuticals Inc.
ORIC-101 and Abraxane (nab-paclitaxel)
Solid tumors

Phase 1b Phase 1b data to be presented at ASCO June 4, 2021.
$917.4 million

PLX – Protalix BioTherapeutics Inc. (DE)
Pegunigalsidase alfa (PRX-102)
Fabry disease

CRL CRL announced April 28, 2021.
$131.2 million

SIOX – Sio Gene Therapies Inc.
AXO-Lenti-PD - SUNRISE-PD
Parkinson's disease

Phase 1/2 Phase 1/2 efficacy, safety, and tolerability data from the second cohort at 6 months post administration presented at ASGCT meeting May 13, 2021.
$125.7 million

SURF – Surface Oncology Inc.
SRF388
Solid tumors

Phase 2 Phase 1 data due at ASCO June 4, 2021.
$337.6 million

TRIL – Trillium Therapeutics Inc.
TTI-622
Refractory multiple myeloma

Phase 1b Phase 1b TTI-622 monotherapy showed 33% objective response rate (ORR) in relapsed/refractory (R/R) lymphomas.
$778.6 million