FDA Approves First Two Sickle Cell Gene Therapies

The Food and Drug Administration on Friday approved two gene therapies to treat patients with sickle cell disease, a serious genetic blood disorder most common in African-American people in the U.S.

• The new treatments, one developed by Vertex Pharmaceuticals (VRTX) and Crispr Therapeutics (CRSP)  and the other by bluebird bio (BLUE)  are the first two gene therapies available in the U.S. for sickle cell, which the FDA says affects 100,000 people in the U.S.
• Bluebird said it would price Lyfgenia at $3.1 million. Vertex, in a securities filing, said it would price Casgevy at $2.2 million.

Both Casgevy and Lyfgenia are made with a patient’s own blood stem cells, which must be removed and then modified. Casgevy edits the DNA of the patient’s stem cells, which are then returned to the patient, where they improve the body’s production of a particular form of hemoglobin. Lyfgenia modifies the stem cells using a viral vector approach. For both treatments, the process for patients is intensive and requires lengthy hospital stays and chemotherapy.

By midafternoon, shares of bluebird had dropped 33.9%. Nasdaq halted trading because of volatility four times between 1:30 p.m. and 1:55 p.m.

The FDA’s label for bluebird’s drug included a warning that some patients who received the treatment had developed blood cancer. The label for the Vertex and Crispr drug included no such warning.

In a related financial setback, bluebird said Friday that the FDA hadn’t awarded the company a priority review voucher along with the approval of its gene therapy. Those vouchers entitle a company to speedier regulatory review of a drug, and are awarded by the agency along with certain approvals. Bluebird had already agreed to sell any priority review voucher it received along with the gene therapy approval to Novartis for $103 million; that sale won’t happen now.

The Vertex and Crispr gene therapy, which will be marketed under the name Casgevy, is also the first FDA-approved drug that uses a cutting-edge genome editing technology called Crispr/Cas9. That technology, described as “genetic scissors,” won a Nobel Prize in Chemistry in 2020 but had been out of reach for the patients it could benefit.

U.K. regulators approved Casgevy in mid-November. The approval of the bluebird bio gene therapy, Lyfgenia, came as a surprise; an FDA decision on the treatment wasn’t expected until Dec. 20.

 

 

 

 

This week's biggest % winners & losers: Aug 9 - 13, 2021 (wk 32)

This week's biggest % gainers/losers The following are this week's top percentage gainers and losers, categorized by sectors (over $300 mln market cap and 100K average daily volume).

This week's top % gainers

• Healthcare: NVAX (256.36 +35%), DVAX (13.66 +27.14%), ARRY (17.23 +26.47%), GRTS (8.05 +17.35%), CASI (1.36 +14.29%), ADAP (3.98 +14.04%), EDIT (62.97 +13.28%), RUBY (24.34 +12.53%)
• Materials: NUE (126.05 +20.84%), LTHM (25.86 +16.89%), TMST (16.63 +15.89%), STLD (72.79 +15.83%), CMC (36.21 +12.37%)
• Consumer Discretionary: LRN (35.42 +17.95%), PLCE (103.75 +14.7%)
• Information Technology: CYBR (158.18 +13.54%), TDC (54.75 +13.38%)
• Financials: AEG (4.93 +12.3%)
• Consumer Staples: PPC (27.38 +28.67%), TSN (82.29 +15.71%)

This week's top % losers

• Healthcare: BLUE (18.3 -26.89%), DRNA (20.48 -24.84%), RDNT (31.1 -18.48%), GBT (27.71 -17.51%), QTNT (3.06 -16.16%), PRGO (41.08 -16.11%), EPZM (5.44 -15.92%), RETA (105.59 -15.05%), SIBN (20.99 -14.74%), NVRO (102.45 -14.43%), NTRA (100.63 -14.21%), CDNA (69.96 -14.21%)
• Materials: NGD (1.21 -22.76%), PQG (12.9 -18.9%)
• Consumer Discretionary: GRPN (25.05 -18.16%), GOOS (36.54 -16.08%)
• Information Technology: JKS (46.18 -16.31%)
• Financials: EHTH (43.66 -14.64%)
• Energy: NGL (1.52 -19.15%), NINE (1.83 -18.67%)

Bluebirdbio (BLUE) reported earnings on Mon 9 Aug 21 (b/o)

** charts after earnings **

 

 

Bluebirdbio also provides eli-cel safety update

• The company received a reported Suspected Unexpected Serious Adverse Reaction (SUSAR) ofmyelodysplastic syndrome (MDS), that is likely mediated by Lenti-D lentiviral vector (LVV)insertion, in a patient who was treated with eli-cel, or Lenti-D drug product for CALD over oneyear ago in the Phase 3 ALD-104 study. Evidence currently available suggests that specificdesign features of Lenti-D LVV likely contributed to this event. The company has shared thisinformation with the independent data monitoring committee of the study and the FDA hasplaced the eli-cel program on a clinical hold.
• The company does not anticipate the clinical hold to impact its programs in sickle cell disease(SCD), -thalassemia or oncology. Subject to resolution of the clinical hold, the companyanticipates completing the submission of the rolling BLA for eli-cel in 2021.

Bluebirdbio misses by $0.32, misses on revs

• Reports Q2 (Jun) loss of $3.58 per share, $0.32 worse than the S&P Capital IQ Consensus of($3.26); revenues fell 96.2% year/year to $7.5 mln vs the $13.31 mln S&P Capital IQConsensus.
• Cash, cash equivalents and marketable securities as of June 30, 2021 and December 31, 2020were $941.6 million and $1.27 billion, respectively.
• On track to complete planned business separation in 4Q21; each company launching withapproximately 24 months of runway following separation.

Earnings this week : Aug 9 - 13, 2021 (wk 32)

 Monday (Aug 9)

• Morning:  APD AMRX AVYA AXSM BALY GOLD BHVN BNTX BLUE CRNC CEVA DISH EBIX ELAN ENR EPZM GTES RDNT SGMS SWAV SYNH TGNA TTD TSN USFD VTRS
• Afternoon: DDD ACM AMC ATCO CABO CBT ELY CMBM CF CHGG CCXI COMP CXW DHT APPS ESE ETH EVBG FGEN FLGT HALO HLIO HBM INO IPAR DOOR LOTZ  MESA MODN NCMI NHI NTR OSH PEN PLNT PRPL QLYS RETA RGNX RPAY RCKT SAIL SDC STE REAL TPTX VAPO VUZI WES ZIOP

Tuesday (Aug 10)

• Morning: ARMK BSY CSPR IMOS DFH IIVI KRNT EDU NOVT SYY TDG VSH
• Afternoon:  EPAY COIN DAR FLYW FUBO GO NARI JAMF MRVI MCFE OLO ONTF LPRO PAAS POSH PUBM SGFY STEP LRN SMCI TASK U UPST WW

Wednesday (Aug 11)

• Morning:  APG ATC BLI CAE GOOS HAE PRGO RPRX TUFN VERX WEN WIX
• Afternoon:  DIBS AMWL APP ARRY AZPN AVT BLNK BMBL CACI CPNG LFST MSP EBAY ENS FNV GOCO HIMS BEKE RIDE MQ NIO OPEN RXT ROOT RGLD SONO VRM

Thursday (Aug 12)

• Morning: AZEK AZUL BIDU BR CSIQ CELH CYBR DCBO GDRX GRWG IQ KELYA MDP MIDD EYE OGN PLTR PGTI SDGR SHC TAL THRY UTZ
• Afternoon:  ABNB AQN CRCT DASH FIGS FLO GLOB MAX PVG RKT RSI SEER SOFI SXI VIAV DIS WPM WISH ZIP

Friday (Aug 13)  

• Morning:  DSEY ERJ HNST

=Incyte (INCY) and Merck (MRK) : failed key Phase 3 melanoma study

• Biotech stocks (XBI) lower after important immune-oncology combo data (Incyte's IDO inhibitor plus Merck's Keytruda) fails in key Phase 3 melanoma study:  NLNK (IDO inhibitor peer) -37.57%,  INCY -18.23%,  NKTR -9.84%,  JNCE -10.20%,  LABU -5.14%,  FPRX -3.84%,  ONCS -3.72%,  SAGE -2.21%,  SGEN -1.81%,  BLUE -4.85%,  PBYI-1.85%,  SPPI -2.46%,  CRVS -9.17%,  TSRO +0.37%,  BMRN -2.01%, GLYC-2.03%, MRK-1.19% 
• IBB -1.99%,  XBI -1.78%

Incyte announces that the External Data Monitoring Committee determined that the Phase 3 ECHO-301/KEYNOTE-252 study did not meet the primary endpoint 

The co and Merck (MRK) announced that an external Data Monitoring Committee review of the pivotal Phase 3 ECHO-301/KEYNOTE-252 study results evaluating Incyte's epacadostat in combination with Merck's KEYTRUDA in patients with unresectable or metastatic melanoma determined that the study did not meet the primary endpoint of improving progression-free survival in the overall population compared to KEYTRUDA monotherapy.

• The study's second primary endpoint of overall survival also is not expected to reach statistical significance. Based on these results, and at the recommendation of the eDMC, the study will be stopped. The safety profile observed in ECHO-301/KEYNOTE-252 was consistent with that observed in previously reported studies of epacadostat in combination with KEYTRUDA.

Incyte and Merck will inform investigators of the results and work with investigators to appropriately conclude the study in a manner consistent with the best interests of each patient. Data from this study will be analyzed and submitted for presentation at an upcoming scientific congress.

=Bluebirdbio (BLUE) reported earnings on Wed 1 Nov 2017 (b/o)

Bluebirdbio misses by $0.02, misses on revs 

• Reports Q3 (Sep) loss of $1.73 per share, $0.02 worse than the Capital IQ Consensus of ($1.71); revenues rose 397.4% year/year to $7.71 mln vs the $8.47 mln Capital IQ Consensus.
• Today, bluebird bio announced that the company will present clinical and pre-clinical data in 11 abstracts spanning the company's research and development portfolio.
• Cash, cash equivalents and marketable securities as of September 30, 2017 were $1.1 billion, compared to $884.8 million as of December 31, 2016, an increase of $257.8 million.
• "Coming into ASH, we have an early, but promising, indication that the changes we made in the HGB-206 study have improved engraftment and that mobilization and collection of stem cells using plerixafor may be a safe and viable option for patients with severe sickle cell disease. At ASH, we look forward to sharing additional data across our clinical programs as well as preclinical data that supports our future pipeline."