The Daily Biotech Pulse: ASH Meeting Wraps, Fast Track Designation For Equillium, Pfizer To Buy $3M In ContraFect Shares

The following are top developments in the biotech space over the last 24 hours.

Scaling The Peaks

(Biotech stocks that hit 52-week highs Dec. 9.)

• Acceleron Pharma Inc XLRN
• Aimmune Therapeutics Inc AIMT
• Aptose Biosciences Inc APTO (moved on ASH presentation)
• argenx SE – ADR ARGX
• ArQule, Inc. ARQL, which agreed to be bought by Merck & Co., Inc. MRK 
• Arvinas Inc ARVN 
• Avadel Pharmaceuticals PLC AVDL
• Bausch Health Companies Inc BHC
• BioNTech SE – ADR BNTX
• BridgeBio Pharma Inc BBIO (appointed oncology scientist Eli Wallace as chief scientific officer for its oncology programs)
• Bristol-Myers Squibb Co BMY (reacted to ASH presentation)
• Constellation Pharmaceuticals Inc CNST
• Deciphera Pharmaceuticals Inc DCPH
• Eidos Therapeutics Inc EIDX
• Epizyme Inc EPZM (reacted to ASH presentation)
• Flexion Therapeutics Inc FLXN
• Forty Seven Inc FTSV (reacted to ASH presentation)
• GALAPAGOS NV/S ADR GLPG
• Global Blood Therapeutics Inc GBT
• Globus Medical Inc GMED
• Immunomedics, Inc. IMMU
• Inspire Medical Systems Inc INSP
• IVERIC bio Inc ISEE
• Kadmon Holdings Inc KDMN
• Krystal Biotech Inc KRYS
• Momenta Pharmaceuticals, Inc. MNTA
• Morphosys Ag MOR
• Neoleukin Therapeutics Inc NLTX
• Precision BioSciences Inc DTIL (moved on ASH presentation)
• Principia Biopharma Inc PRNB (reacted to ASH presentation)
• Repro-Med Systems, Inc. KRMD
• Rocket Pharmaceuticals Inc RCKT (presented positive preliminary results for Phase 1/2 study of RP-L201, its lentiviral vector--based gene therapy, for the treatment of Leukocyte Adhesion Deficiency-I)
• SpringWorks Therapeutics Inc SWTX (moved on ASH presentation)
• Synthorx Inc THOR, which announced a deal to be acquired by Sanofi SA SNY
• TG Therapeutics Inc common stock TGTX (moved on ASCO presentations)
• Vertex Pharmaceuticals Incorporated VRTX
• XBiotech Inc XBIT (announced an agreement to sell true human antibody bermekimab targeting IL-1a to Johnson & Johnson JNJ's Janssen)
• Zai Lab Ltd ZLAB

Down In The Dumps

(Biotech stocks that hit 52-week lows Dec. 9.)

• AEterna Zentaris Inc. AEZS
• Assertio Therapeutics Inc ASRT
• Aytu Bioscience Inc AYTU
• Biocept Inc BIOC (priced its $10-million underwritten public offering of stock and warrants)
• Centogene NV CNTG
• Cerus Corporation CERS
• Endologix, Inc. ELGX
• Evelo Biosciences Inc EVLO
• Galera Therapeutics Inc GRTX
• HTG Molecular Diagnostics Inc HTGM
• Jaguar Health Inc JAGX
• KemPharm Inc KMPH
• KITOV PHARMA LT/S ADR KTOV
• Midatech Pharma PLC-ADR MTP
• Midatech Pharma PLC-ADR MTP
• Orgenesis Inc ORGS
• RA Medical Systems Inc RMED (received notice from the NYSE regarding non-compliance of continued listing standard)
• Syros Pharmaceuticals Inc SYRS
• Tonix Pharmaceuticals Holding Corp TNXP
• TRACON Pharmaceuticals Inc TCON
• Viveve Medical Inc VIVE

Stocks In Focus

Equillium's Lupus Nephritis Treatment Gets Fast Track Designation

Equillium Inc EQ, which develops therapies for severe autoimmune and inflammatory disorders, said the FDA has granted Fast Track designation for EQ001, or itolizumab, for the treatment of lupus nephritis.

The stock was trading 23.46% higher to $5 in Tuesday's premarket session. 

See also: 5 Stocks Moving On ASH Presentations

Supernus Says New Analysis Of Late-Stage Study Of ADHD Drug Shows Positive Results

Supernus Pharmaceuticals Inc SUPN announced updated results from the first Phase 3 study of SPN-810, a treatment of impulsive aggression in patients with attention deficit hyperactivity disorder.

A new analysis conducted by excluding patients with mild IA from both the placebo and control group showed that the study produced positive results, the company said, in contrast to top-line results revealed in early November that showed a statistically insignificant median percentage reduction in the average weekly frequency of impulsive aggressive episodes from baseline.

The stock was up 10.14% at $24.99 in Tuesday's premarket session. 

Pfizer to Invest $3M In ContraFect

ContraFect Corp CFRX said it has commenced an underwritten public offering of shares of its common stock. The company also entered into a stock purchase agreement with Pfizer Inc. PFE, under which the latter has agreed to buy up to $3 million worth of ContraFect shares in a separate private placement at a price equal to the price per share of the proposed common stock offering.

ContraFect shares were soaring 21.28% to 29 cents in Tuesday's premarket session.

Sanofi Targets 30% Operating Income Margin By 2020 

Sanofi disclosed a new strategy to drive growth and innovation at its capital markets day, with the French pharma company singling out Dupixent and vaccines as its growth drivers.

The company said it will accelerate its R&D focus on six transformative medicines: the RNAi therapeutic Fitusiran, the factor VIII therapy BIVV001, the lysosomal storage disorder treatment candidate venglustat, the HR-positive breast cancer treatment SERD, the preventative RSV treatment option nirsevimab and the MS treatment BTKi.

The company expects to expand its operating income margin to 30% by 2020, with a 32% target set for 2025.

ASH Roundup

◘ Gamida Cell Ltd GMDA announced updated results from a Phase 1 study of GDA-201, an investigational, natural killer cell-based cancer immunotherapy for treating non-Hodgkin lymphoma and multiple myeloma.

The results showed that GDA-201 in combination with monoclonal antibodies was generally well-tolerated and demonstrated early evidence of clinical activity in heavily pretreated patients.

The stock was 4% higher at $4.94 in the premarket session. 

◘ Bristol-Myers Squibb and Acceleron presented initial Phase 2 data from a study evaluating luspatercept-aamt in myelofibrosis-associated anemia, showing promising clinical activity. The companies said they will initiate a pivotal Phase 3 study dubbed "INDEPENDENCE" in 2020.

The companies also presented longer-term follow-up data from the pivotal Phase 3 studies: MEDALIST in MDS-associated anemia and BELIEVE in beta-thalassemia-associated anemia. Both studies showed that patients experienced sustained clinical benefit.

◘ Bristol-Myers Squibb and bluebird bio Inc BLUE announced updated Phase 1 results from the study of bb21217, an investigational BCMA-targeted chimeric antigen receptor T cell therapy being studied in relapsed/refractory multiple myeloma. It showed the potential for durable responses in heavily pretreated patients.
The median duration of response was 11.1 months at the 150x 106 CAR+ T cell dose level, the companies said.

Bluebird shares were edging higher in Tuesday's premarket session. 

◘ Editas Medicine Inc EDIT, a gene editing company, announced in vivo proof-of-concept data supporting the development of EDIT-301 as a treatment option for sickle cell disease and beta-thalassemia.

The shares were adding 4.22% to $31.60 in Tuesday's premarket session. 

Offerings

◘ Constellation Pharma said it has commenced an underwritten public offering of 4.75 million shares of its common stock. All the shares are being offered by the company.

The stock was down 3.02% at $35.05 at the time of publication.

◘ SCYNEXIS Inc SCYX said it has commenced an underwritten public offering of its common stock and warrants. All the shares and warrants are to be sold by the company.

The stock was plummeting 22.78% to 74 cents in Tuesday's premarket session. 

On The Radar

Adcom Meeting

FDA's Cardiovascular and Renal Drugs Advisory Committee will discuss Correvio Pharma Corp's CORV for Brinavess, or vernakalant HCl solution, ahead of the Dec. 24 PDUFA action date.

Clinical Readouts

ASH Presentations

• Amgen, Inc. AMGN & GENMAB A/S/S ADR GMAB: Phase 3 data for krypolis in multiple myeloma (late-breaker abstract).
• Sanofi: Phase 3 data for sutimlimab in cardinal cold agglutinin disease.
• Bristol-Myers Squibb: Phase 3 data for oral azacitidine for acute myeloid leukemia maintenance.

Earnings

Enzo Biochem, Inc. ENZ (after the close)

Related Link: The Week Ahead In Biotech: ASH Presentations Pick Up Steam, Correvio Awaits Adcom Ruling