BPC November 15 update

Kezar KZR +67% AH Phase 2 data; Tricida TCDA +16% Direct equity financing

Price and Volume Movers

Kezar Life Sciences, Inc. (NASDAQ: KZR) released interim results from its Phase 2 portion of its MISSION trial of KZR-616 to treat lupus nephritis (LN). 3 of 5 patients had a 50% or greater reduction in UPCR, and 4 patients reported a clinically meaningful reduction in proteinuria to less than 0.8 UPCR. Shares are currently trading up 67% at $14.10 during the after-hours.

Tricida, Inc. (NASDAQ: TCDA) announced that it has entered into definitive agreements with certain investors for the purchase and sale of common stock, pre-funded warrants, and common warrants in a registered direct equity financing for gross proceeds of approximately $42 million. Shares closed up 16% at $6.35.

Mesoblast Limited (NASDAQ: MESO) presented new results from its Phase 3 study of rexlemestrocel-L to treat chronic heart failure (CHF) with reduced ejection fraction (HFrEF). The results reported that rexlemestrocel-L on top of standard of care reduced the incidence of heart attacks or strokes by 65% across all treated patients compared with standard of care alone. Shares closed up 13% at $7.09.

BioXcel Therapeutics, Inc. (NASDAQ: BTAI) shares fell after Goldman Sachs downgraded the stock from Buy to Sell and lowered its price target from $55 to $24. Shares closed down 21% at $27.21.

Cassava Sciences (NASDAQ: SAVA) shares traded lower after the company disclosed in a quarterly report that certain government agencies have asked the company to provide corporate information and documents. Shares closed down 12% at $60.51.

Notable earnings releases from today:

UroGen Pharma Ltd. (NASDAQ: URGN)

Trevena, Inc. (NASDAQ: TRVN)

Impel NeuroPharma (NASDAQ: IMPL)

Landos Biopharma, Inc. (NASDAQ: LABP)

Advancers

CompanyPriceChange
BFRI
Biofrontera Inc.
$7.90+2.68  +51.34%
NRXP
NRX Pharmaceuticals Inc.
$6.75+2.25  +50.00%
ADGI
Adagio Therapeutics Inc.
$25.12+6.39  +34.12%
ATNF
180 Life Sciences Corp.
$5.41+0.95  +21.30%
GOVX
GeoVax Labs Inc.
$4.69+0.81  +20.88%
MRNA
Moderna Inc.
$329.63+56.24  +20.57%
ICVX
Icosavax Inc.
$27.55+4.51  +19.57%
VALN
Valneva SE
$59.60+9.7  +19.44%
TYRA
Tyra Biosciences Inc.
$23.87+3.66  +18.11%
PTPI
Petros Pharmaceuticals Inc.
$2.09+0.26  +14.21%

Decliners

CompanyPriceChange
LGVN
Longeveron Inc.
$30.67-11.63  -27.49%
AMTI
Applied Molecular Transport Inc.
$16.33-2.66  -14.01%
NEXI
NexImmune Inc.
$7.94-1.06  -11.78%
IGMS
IGM Biosciences Inc.
$49.63-6.58  -11.71%
CRNX
Crinetics Pharmaceuticals Inc.
$24.40-3.08  -11.21%
AVRO
AVROBIO Inc.
$4.08-0.51  -11.11%
ELYM
Eliem Therapeutics Inc
$13.76-1.72  -11.11%
MRSN
Mersana Therapeutics Inc.
$6.95-0.82  -10.55%
MNOV
MediciNova Inc.
$3.22-0.35  -9.80%
TARS
Tarsus Pharmaceuticals Inc.
$25.00-2.7  -9.75%

Pipeline Database Updates

Drug Price Stage Catalyst Market Cap

ALNY – Alnylam Pharmaceuticals Inc.
Zilebesiran (ALN-AGT) - (KARDIA-1)
Hypertension

$188.53
-0.27  0%
Phase 2 Phase 2 initiation announced June 30, 2021. Phase 2 enrollment expected to be complete by mid-2022, with top-line data due in late 2022.
$22.5 billion

ARWR – Arrowhead Pharmaceuticals Inc.
ARO-APOC3-3001
Familial chylomicronemia syndrome (FCS), Hypertriglyceridemia

$72.02
-2.02  -3%
Phase 3 Phase 3 trial expected to be initiated 4Q 2021. Phase 1/2 date reported APOC3 was reduced by 98% in FCS patients and 96% in MCM patients. Both groups showed similar maximum median reductions in TG of 91% and 90%, respectively, noted November 15, 2021.
$7.5 billion

CBAY – CymaBay Therapeutics Inc.
Seladelpar (RESPONSE)
Primary biliary cholangitis (PBC)

$3.77
-0.03  -1%
Phase 3 Phase 3 trial has been initiated. Continues to enroll as of August 12, 2021. Long term data reported that the composite endpoint was achieved by 64% and 79% of patients after one and two years, respectively. In addition, ALP normalization was achieved in 24% and 42% in patients at one and two years, respectively, noted November 15, 2021.
$319.2 million

CYTK – Cytokinetics Incorporated
Omecamtiv mecarbil - (GALACTIC-HF)
Acute heart failure

$39.03
-1.87  -5%
Phase 3 Phase 3 trial additional data demonstrated that treatment was associated with greater reductions in stroke for patients with a history of stroke, reducing the risk by 77%, as well as in patients with a history of atrial fibrillation, reducing the risk by 51%, noted November 15, 2021.
$3.3 billion

EYPT – EyePoint Pharmaceuticals Inc.
EYP-1901 (DAVIO)
Wet age-related macular degeneration (AMD)

$16.53
-0.18  -1%
Phase 1 Phase 1 preliminary safety 3-month data continues to demonstrate that dosage is well-tolerated in eyes with wet AMD, presented October 12, 2021. Phase 1 top-line data reported 76% and 53% of patients did not require rescue following a single dose, with stable and sustained BCVA (-2.5 letters) and CST (-2.7 μm), a 79% reduction in treatment burden at six months, and a median time to rescue of six months across all patients, noted November 13, 2021. Phase 2 trial due in 2022 with Type C meeting on December 1, 2021.
$560.4 million

IFRX – InflaRx N.V.
Vilobelimab (IFX-1) - (IXCHANGE)
ANCA-associated Vasculitis (AAV)

$5.08
+0.06  +1%
Phase 2 Phase 2 data released November 15, 2021. Clinical response as well as clinical remission were achieved in comparably high rates in all three arms: Clinical response at week 16 in evaluable patients was observed in 16 out of 18 (88.9%) patients in the treatment group receiving vilobelimab alone; in 22 out of 23 (95.7%) patients receiving SDGC; and in 10 out of 13 (76.9%) patients in the vilobelimab + RDGC group.
$224.5 million

MIST – Milestone Pharmaceuticals Inc.
Etripamil - NODE-301
Paroxysmal supraventricular tachycardia (PSVT)

$6.03
+0.01  +0%
Phase 3 Phase 3 top-line data showed that the nasal dose significantly decreased heart rate during supraventricular tachycardia (SVT) episodes and independent of conversion to sinus rhythm (SR). Heart rate changes occurred within 5 minutes and were sustained for 60 minutes, noted November 15, 2021.
$180.1 million

MRNA – Moderna Inc.
VEGF-A(AZD8601)
Heart Failure

$329.63
+56.24  +21%
Phase 2a Phase 2a study met the primary endpoint of safety and tolerability; numerical trends observed in endpoints in the heart failure efficacy domains compared with placebo, including increase in LVEF and patient reported outcomes, noted November 15, 2021.
$133.6 billion

MRNA – Moderna Inc.
mRNA-2752
Solid tumors, lymphoma

$329.63
+56.24  +21%
Phase 1 Phase 1 data presented at SITC reported plasma levels maintained at < 1ng/mL with dose ranges up to 8mg supports the therapeutic goal of ITu therapy to limit systemic exposure and toxicity. Two durable partial responses noted November 12, 2021. Enrollment in dose expansion part of Arm B and Arm C is currently ongoing.
$133.6 billion

ONCT – Oncternal Therapeutics Inc.
ONCT-216 (formerly TK216)
Relapsed or refractory Ewing sarcoma

$3.04
-0.11  -3%
Phase 1/2 Phase 1/2 interim data showed that the drug remains generally well tolerated. 2 CR in heavily pretreated patients. As of the October 1, 2021 data cutoff date, the most common drug-related adverse events included myelosuppression, fatigue, alopecia, nausea, pyrexia, and decreased appetite. No unexpected off-target toxicities have been observed, noted November 15, 2021.
$150.3 million

PHGE – BiomX Inc.
BX001
Acne

$2.04
+0.14  +7%
Phase 2 Program to be discontinued to focus resources on CF and AD programs, noted November 15, 2021.
$58.3 million

PTGX – Protagonist Therapeutics Inc.
Rusfertide (PTG-300)
Hereditary hemochromatosis

$33.26
-1.45  -4%
Phase 2 Phase 2 data released November 15, 2021. Phase 2 trial met primary endpoint, rusfertide-treated patients had 0.009 phlebotomies per month during the study compared to 0.28 phlebotomies per month pre-study. The average TSAT during treatment was 31.4% compared to 45.0% pre-study and the serum iron was 101 mcg/dl compared to 137 mcg/dl.
$1.6 billion

RCKT – Rocket Pharmaceuticals Inc.
RP-A501
Danon disease

$23.67
-1.46  -6%
Phase 1 Phase 1 clinical hold lifted August 16, 2021. Phase 1 low-dose pediatric patient cohort initiated, noted November 3, 2021. Phase 1 results reported a sustained benefit across clinical, functional and biomarker endpoints in all four patients with long-term follow up. NYHA class improvement (from II to I) was noted in all three patients with closely monitored immunosuppressive regimen and decreased cardiac wall thickness with improved or stabilized ejection fraction on echocardiogram, noted November 15, 2021.
$1.5 billion

RYTM – Rhythm Pharmaceuticals Inc.
IMCIVREE (setmelanotide)
Alstrom Syndrome and Bardet-Biedl Syndrome

$9.24
-0.90  -9%
PDUFA PDUFA date March 16, 2022.
$464.5 million