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.

Related Link: The Week Ahead In Biotech (Nov. 1-7): Adcom Test For Biogen's Aducanumab, Alzheimer's Conference, SMID-cap Earnings

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)

Earnings

• Kura Oncology Inc KURA (before the market open)
• Synlogic Inc SYBX (before the market open)
• Regeneron (before the market open)
• Bristol-Myers Squibb Co BMY (before the market open)
• Arbutus Biopharma Corp ABUS (before the market open)
• Alnylam Pharmaceuticals, Inc. ALNY (before the market open)
• Athenex Inc ATNX (before the market open)
• Adaptimmune Therapeutics PLC – ADR ADAP (before the market open)
• ANI Pharmaceuticals Inc Common Stock ANIP (before the market open)
• Antares Pharma Inc ATRS (before the market open)
• Agios Pharmaceuticals Inc AGIO (before the market open)
• X4 Pharmaceuticals Inc XFOR (before the market open)
• SAGE Therapeutics Inc SAGE (before the market open)
• Intellia Therapeutics Inc NTLA (before the market open)
• Kala Pharmaceuticals Inc KALA (before the market open)
• OptiNose Inc OPTN (before the market open)
• Orthofix Medical Inc OFIX (before the market open)
• Radius Health Inc RDUS (before the market open)
• Repligen Corporation RGEN (before the market open)
• Incyte Corporation INCY (before the market open)
• Eyepoint Pharmaceuticals Inc EYPT (before the market open)
• Concert Pharmaceuticals Inc CNCE (before the market open)
• Clovis Oncology Inc CLVS (before the market open)
• Vericel Corp VCEL (before the market open)
• Provention Bio Inc PRVB (before the market open)
• Harvard Bioscience, Inc. HBIO (before the market open)
• BioCryst Pharmaceuticals, Inc. BCRX (before the market open)
• BioDelivery Sciences International, Inc. BDSI (before the market open)
• Amarin Corporation plc AMRN (before the market open)
• Y-mAbs Therapeutics, Inc YMAB (after the close)
• AcelRx Pharmaceuticals Inc ACRX (after the close)
• Aerie Pharmaceuticals Inc AERI (after the close)
• Adamas Pharmaceuticals Inc ADMS (after the close)
• Transenterix Inc TRXC (after the close)
• Paratek Pharmaceuticals Inc PRTK (after the close)
• Nektar Therapeutics NKTR (after the close)
• Spero Therapeutics Inc SPRO (after the close)
• Misonix Inc MSON (after the close)
• TFF Pharmaceuticals Inc TFFP (after the close)
• Xencor Inc XNCR (after the close)
• Oyster Point Pharma Inc OYST (after the close)
• Sarepta Therapeutics Inc SRPT (after the close)
• Natera Inc NTRA (after the close)
• ICU Medical Inc ICUI (after the close)
• Rigel Pharmaceuticals, Inc. RIGL (after the close)
• Guardant Health Inc GH (after the close)
• Retrophin Inc RTRX (after the close)
• Tandem Diabetes Care Inc TNDM (after the close)
• Chembio Diagnostics Inc CEMI (after the close)
• Dicerna Pharmaceuticals Inc DRNA (after the close)
• Exelixis, Inc. EXEL (after the close)
• Endo International PLC ENDP (after the close)
• FibroGen Inc FGEN (after the close)
• Fluidigm Corporation FLDM (after the close)
• Nevro Corp NVRO (after the close)
• CytomX Therapeutics Inc CTMX (after the close)
• Dynavax Technologies Corporation DVAX (after the close)
• Geron Corporation GERN (after the close)
• CorMedix Inc. CRMD (after the close)
• Celcuity Inc CELC (after the close)
• BioLife Solutions Inc BLFS (after the close)
• Caladrius Biosciences Inc CLBS (after the close)
• Theravance Biopharma Inc TBPH (after the close)
• Global Blood Therapeutics Inc GBT (after the close)
• Coherus Biosciences Inc CHRS (after the close)
• Iovance Biotherapeutics Inc IOVA
• AtriCure Inc. ATRC
• Acceleron Pharma Inc XLRN
• ADMA Biologics Inc ADMA
• Ocular Therapeutix Inc OCUL
• Ironwood Pharmaceuticals, Inc. IRWD
• Syros Pharmaceuticals Inc SYRS
• ZIOPHARM Oncology Inc. ZIOP
• Sensus Healthcare Inc SRTS
• Deciphera Pharmaceuticals Inc DCPH
• Eloxx Pharmaceuticals Inc ELOX
• Chiasma Inc CHMA
• Emergent Biosolutions Inc EBS
• INmune Bio Inc INMB
• Calithera Biosciences Inc CALA

Related Link: A Pfizer Analyst's 3 Possible Coronavirus Vaccine Readout Scenarios