The Daily Biotech Pulse: Bluebird Bio Plunges On Regulatory Filing Delay, Synlogic Soars On Study Initiation, European Label Expansion For Vertex, Merck Goes Shopping

Here's a roundup of top developments in the biotech space over the last 24 hours:

Scaling The Peaks

(Biotech Stocks Hitting 52-week Highs Nov. 4)

Abbott Laboratories ABT
Amicus Therapeutics, Inc. FOLD
Anavex Life Sciences Corp AVXL
Aptevo Therapeutics Inc APVO
Arena Pharmaceuticals, Inc. ARNA
Ascendis Pharma A/S ASND
Athira Pharma Inc ATHA
Bio-Rad Laboratories, Inc. Class A Common Stock BIO
Bio-Rad Laboratories, Inc. Class B Common Stock BIO
Biohaven Pharmaceutical Holding Co Ltd BHVN
BioLife Solutions Inc BLFS
Cardiff Oncology Inc CRDF
Denali Therapeutics Inc DNLI - moved in sympathy with Biogen Inc BIIB
Guardant Health Inc GH
Halozyme Therapeutics, Inc. HALO
Hologic, Inc. HOLX
Idera Pharmaceuticals Inc IDRA
Inspire Medical Systems Inc INSP
Immunovant Inc IMVT
Intellia Therapeutics Inc NTLA
Jazz Pharmaceuticals PLC JAZZ
Kodiak Sciences Inc KOD
Laboratory Corp. of America Holdings LH
Madrigal Pharmaceuticals Inc MDGL
Mirati Therapeutics Inc MRTX
Myokardia Inc MYOK - waiting period with respect to its proposed acquisition by Bristol-Myers Squibb Co BMY ended
Natera Inc NTRA
Oncorus Inc ONCR
PTC Therapeutics, Inc. PTCT
Repligen Corporation RGEN
Replimune Group Inc REPL
ResMed Inc. RMD
Retrophin Inc RTRX
Rocket Pharmaceuticals Inc RCKT (reacted to third-quarter results)
Scholar Rock Holding Corp SRRK
Spero Therapeutics Inc SPRO
SpringWorks Therapeutics Inc SWTX
SQZ Biotechnologies Co SQZ (listed on Nasdaq Friday)
Stoke Therapeutics Inc STOK
Sutro Biopharma Inc STRO
Tcr2 Therapeutics Inc TCRR
Ultragenyx Pharmaceutical Inc RARE
United Therapeutics Corporation UTHR
Wright Medical Group NV WMGI - announced regulatory clearance for its acquisition by Stryker Corporation SYK

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Nov. 4)

Brickell Biotech Inc BBI
FSD Pharma Inc HUGE
Happiness Biotech Group Ltd HAPP
Liminal BioSciences Inc LMNL
Lyra Therapeutics Inc LYRA
Nabriva Therapeutics PLC – ADR NBRV
Neovasc Inc NVCN
Titan Pharmaceuticals, Inc. common stock TTNP
Zosano Pharma Corp ZSAN

Stocks In Focus

Twin Approvals For Merck-AstraZeneca's Lynparza

Merck & Co., Inc. MRK and AstraZeneca plc AZN said Lynparza in combination with Roche Holdings AG's Basel ADR Common Stock RHHBY Avastin has been approved in the European Union for the maintenance treatment of adult patients with advanced high-grade epithelial ovarian, fallopian tube or primary peritoneal cancer who are in response following completion of first-line platinum-based chemotherapy in combination with bevacizumab and whose cancer is associated with homologous recombination deficiency-positive status.

Separately, the companies said the European Union has also approved Lynparza as a monotherapy for the treatment of adult patients with metastatic castration-resistant prostate cancer and BRCA1/2 mutations who have progressed following a prior therapy that included a new hormonal agent.

Merck To Buy Cancer Biopharma VelosBio For $2.75B

Merck announced a definitive agreement to buy VelosBio Inc. — a privately held clinical-stage biopharma developing cancer therapies targeting receptor tyrosine kinase-like orphan receptor 1 — for $2.75 billion in cash, subject to certain customary adjustments.

"VelosBio's lead investigational candidate is VLS-101, an antibody-drug conjugate (ADC) targeting ROR1 that is currently being evaluated in a Phase 1 and a Phase 2 clinical trial for the treatment of patients with hematologic malignancies and solid tumors, respectively," Merck said in a statement.

Novartis Reports Positive Results For Late-Stage Study of Arthritis Drug In Treating Joint Lining Inflammation

Novartis AG NVS announced 12-week results from the Phase 3b ULTIMATE trial of Cosentyx, which demonstrated its significant treatment response on synovitis, or joint lining inflammation, in psoriatic arthritis versus placebo. These data are being presented at the American College of Rheumatology All-Virtual Annual Meeting.

Synlogic Begins Early Stage Study Of Metabolic Disorder Drug

Synlogic Inc SYBX said it has treated the first healthy volunteer in its Phase 1 study of the investigational Synthetic Biotic medicine SYNB8802 for the treatment of enteric hyperoxaluria, an acquired metabolic disorder caused by increased absorption of dietary oxalate.

The stock was up 11.7% premarket at $2.10.

Bluebird Bio Pushes Back BLA Filing Timeline For Sickle Cell Disease Gene Therapy

bluebird bio Inc BLUE said it is adjusting its submission timing from the second half of 2021 to late 2022 for its LentiGlobin gene therapy for sickle cell disease.

The decision comes after the FDA's feedback regarding BLA submission for LentiGlobin, alongside COVID-19 related shifts and contract manufacturing organization COVID-19 impacts, the company said.

The company also reported third-quarter results, showing revenue increasing from $8.9 million in 2019 to $19.3 million in 2020. The loss per share narrowed from $3.73 per share from $2.94, while analysts estimated a loss of $3.01 per share.

The stock was down 14.43% premarket at $50.10.

Stealth Receives Development Funding For Elamipretide

Stealth BioTherapeutics Corp MITO said it has received $20 million from Morningside Ventures contingent on near-term clinical milestones associated with its geographic atrophy and Barth syndrome development programs.

The company said it expects to receive up to an additional $15 million from the same investor.

In addition, the agreement provides for Stealth receiving up to an additional $35 million upon meeting certain pre-defined future milestones.

The company noted that the funds are intended to support the continued clinical development of elamipretide.

The stock was up 11.35% premarket at $1.57.

Vertex Announces Label Expansion For Cystic Fibrosis Drug In Europe

Vertex Pharmaceuticals Incorporated VRTX said the European Commission has granted approval of the label extension for Kalydeco granules to include the treatment of infants with cystic fibrosis.

The approval is for infants ages 4 months and older and weighing at least 5 kg who have the R117H mutation or a specific gating mutations in the cystic fibrosis transmembrane conductance regulator gene.

The stock was up 1.59% at $218.52 premarket Thursday.

Rhythm Pharma Announces Positive Midstage Results For Weekly Formulation For Weight Loss Drug

Rhythm Pharmaceuticals Inc RYTM announced at the Obesity Society's ObesityWeek 2020 interim results from its Phase 2 study evaluating a once-weekly formulation of setmelanotide in healthy obese volunteers.

The results showed that healthy obese people treated with the weekly formulation of setmelanotide achieved comparable weight loss to those treated with the daily formulation.

As of the data cutoff of April 17, weekly setmelanotide administration was generally well-tolerated, with no serious treatment-emergent adverse events, the company said. The safety results were similar to the daily administration and consistent with prior clinical experience.

The stock gained 4.52% to $24.49 in after-hours trading.

Earnings

ACADIA Pharmaceuticals Inc. ACAD reported a wider-than-expected third-quarter net loss of 54 cents per share. Revenues, consisting solely of Nuplazid product sales, increased from $90.59 million to $120.58 million.

The stock was down 0.16% at $49.05 premarket Thursday.

T2 Biosystems Inc's TTOO third-quarter revenues increased from $3.8 million in 2019 to $5.2 million in 2020. The net loss per share narrowed from 31 cents to 8 cents. The company raised its full-year revenue guidance to $19 million to $20 million.

The stock was down 6.62% at $1.41 premarket.

Cytosorbents Corp's CTSO third-quarter revenues climbed 73% year-over-year to $10.5 million. The net loss per share narrowed from 21 cents to 2 cents, which compared favorably to the consensus loss estimate of 9 cents per share.

The company said it expects that fourth quarter will be one of its strongest quarters to date in terms of product sales.

In after-hours trading, the stock moved up 6.08% to $8.90.

G1 Therapeutics Inc GTHX reported license revenue of $26.6 million for the third quarter compared to no revenue in the year-ago period. The loss per share narrowed from 86 cents to 31 cents.

In after-hours trading, the stock ended up 5.04% to $12.50.

Protagonist Therapeutics Inc PTGX reported higher revenues and a narrower loss for the third quarter.

The stock rose 5.56% to $22.40 in after-hours trading.

On The Radar

Clinical Readouts

Bellerophon Therapeutics Inc BLPH will present at the American Thoracic Society Interstitial Lung Disease Mini Symposia as well as the 2020 Pulmonary Fibrosis Foundation Meeting, data from its Phase 2 study of INOpulse for the treatment of fibrotic interstitial lung disease patients at risk for pulmonary hypertension. (Thursday)