December 11, 2017 7:04am

American Society of Hematology (ASH) Annual Meeting in Atlanta, Georgia will give the sector a kick …

 

One of the BIG reasons, “Unheard of' responses to bluebird (BLUE) CAR-T therapy seen in myeloma study!”

 

Pre-open indications:  short and non-sweet, 5 BUYs and 1 SELL

Out and about: bluebird bio (BLUE) at ASH - more than half of patients with advanced multiple myeloma who had run out of therapeutic options remained in complete remission after receiving BLUE’s experimental gene-modifying immunotherapy in a small, early stage study, according to updated data released on Sunday. Of 18 patients who received a therapeutic dose of bb2121, all but one responded to the treatment, a 94% response rate, while 56% remained in remission with a median follow-up of 40 weeks after treatment. (Reuters, Bill Berkrot).

BLUE also announced data from two studies of its LentiGlobin gene therapy product candidate in patients with transfusion-dependent β-thalassemia (TDT).

VSTM presentation of the results from the Phase 3 DUO study evaluating the efficacy and safety of duvelisib in patients with relapsed or refractory chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL)

Bellicum Pharmaceuticals, Inc. (BLCM), announced results from a leading European transplant center participating in the BP-004 trial in children with blood cancers and nonmalignant disorders.

Fate Therapeutics, Inc. (FATE), announced the generation of chimeric antigen receptor (CAR)-targeted CD8αβ+ T cells from a clonal engineered master pluripotent cell line (MPCL). The groundbreaking development enables the renewable production of CAR-targeted, TCR-null CD8αβ+ T cells that are not restricted to an individual patient for off-the-shelf administration.

 

Can’t read because of no subscription rights - you’re missing situational awareness and the most trustworthy source of independent insight!

 

Higher open expected

Dow futures are UP +0.19`% (+47 points) and NASDAQ futures are UP +0.11% (+7.25 points)

 

U.S. stock index futures pointed to a higher open on Monday, as investors kept an eye on the latest bitcoin moves.

European stocks were mixed in mid-morning trade as investors focused on two upcoming monetary policy meetings.

Asian markets edged higher as investors focused their attention on the launch of bitcoin futures, and an upcoming Federal Reserve meeting that's slated for later in the week.

 

Issues that will affect the trading day: Bitcoin will be front and center — again — for investors during trade, after the cryptocurrency's futures started trading on the Cboe Futures Exchange on Sunday.

Data docket: the main piece of economic news set to be released is the Job Openings and Labor Turnover Survey (JOLTS) data, due out at 10 a.m. ET.

Issues on the political front: Markets will also keep a close eye on any developments coming out of the U.S. administration, whether that is related to Russia, overhauling the U.S. tax system or President Donald Trump's recent announcement that the U.S. would recognize Jerusalem as the capital of Israel — a move criticized internationally.

Henry’omics:

From Friday night’s closing bell post, “… the newest version of an old normal. Opened up, stayed up yet neutral at the mid-day and closed neutral of our 40 covered companies while the IBB closed up +1.94% after Thursday’s positive +1.53%. Notice the strong got stronger and the weak dived weaker.”

 

 

The RegMed and cell therapy sector closed NEUTRAL on Friday, POSITIVE on Thursday, NEGATIVE on Wednesday, Tuesday and last Monday.

The cell therapy sector’s record over 1 session (of 40 covered companies):

·         Friday closed NEUTRAL with 19 decliners, 19 advancers and 2 flats;

·         Thursday closed POSITIVE with 11 decliners, 24 advancers and 5 flats;

·         Wednesday closed NEGATIVE with 29 decliners, 7 advancers and 4 flats

·         Tuesday closed NEGATIVE with 21 decliners 18 advancers and 1 flat;

·         Last Monday closed NEGATIVE with 32 decliners, 7 advancers and 1 flat;

 

 

You’ve made it to the office, turned on the monitor, having just gotten your coffee and it hits you - what are today’s indications?

Watch list:

  • The iShares Nasdaq Biotechnology (IBB) is NOT indicating in Monday’s pre-market;
  • The SPDR S&P Biotech ETF (XBI) is NOT indicating in Monday’s pre-market;
  • The Health Care Select Sector SPDR ETF (XLV) is NOT indicating in Monday’s pre-open;
  • The iShares Russell 2000 (IWM) is indicating a POSITIVE +0.06% in Monday’s pre-open

 

 

Out and about:

Researchers, who reported the bluebird (BLUE) data at the American Society of Hematology meeting in Atlanta, said the initial response to the treatment was very quick and that many of the patients continued to improve over time.

·         Patients in the PI dose-escalation study had received seven prior treatment regimens; including regimens with the newest multiple myeloma drugs, such as Johnson & Johnson's Darzalex, and had undergone at least one stem cell transplant before receiving bb2121, which is being co-developed with Celgene Corp.

·         "Some of these patients were going to hospice until they got this," said Dr. Jesus Berdeja, the study's lead investigator.

"This is unheard of, something that we haven't seen with any drugs approved for myeloma in this type of population. The excitement among all the myeloma providers is crazy," said Berdeja, director of myeloma research at the Sarah Cannon Center for Blood Cancer in Nashville.

·         bb2121 belongs to a potentially revolutionary new type of one-time treatment called CAR-T therapy that involves genetic manipulation of a patient's immune system. A patient's own disease-fighting T-cells are harvested and genetically reengineered to target specific proteins on cancer cells before being replaced so they can circulate seeking out and attacking the cancer, possibly for years.

·         The first two CAR-T therapies from Novartis and Gilead Sciences, through its acquisition of Kite Pharma, were approved earlier this year for other blood cancers.

·         Three patients who received what proved to be sub-optimal doses of cells early in the bb2121 study died. Of the 18 who received higher doses, four patients have now experienced disease progression, while 14 continue to respond, researchers reported.

·         The treatment was very well tolerated for a CAR-T product, Berdeja said.

Only two patients experienced serious cytokine release syndrome with no reports of serious brain toxicity, both common side effects of CAR-T treatment. The most serious side effect was neutropenia, or very low white blood cell count, researchers said.

·         While this was a small Phase I study, the results were considered so impressive that Celgene plans to begin enrolling patients this month for a larger, potentially pivotal trial that could position bb2121 to become the third approved CAR-T.

·         "They're hitting it so far out of the park that they're not wasting any time," Berdeja said.

 

Companies in my headlights:

Bellicum Pharmaceuticals (BLCM) closed up +$0.32 to $9.53 with 329.5 K shares traded <3 month average =454.3 K shares>.  BLCM announced (Saturday) results from a leading European transplant center participating in the BP-004 trial in children with blood cancers and nonmalignant disorders. Patients were treated with BPX-501 following an alpha/beta T cell and CD19+ B cell depleted haploidentical hematopoietic stem cell transplant (haplo-HSCT). Results demonstrated that donor BPX-501 cells infused after transplant expanded in vivo and persisted over time, contributing to improved immune recovery for patients in the study as compared to historical controls from the same transplant center. The data were reviewed in an oral presentation today during the 59th Annual Meeting of the American Society of Hematology (ASH) – BUY;

bluebird bio (BLUE) closed up +$3.25 to $171.15 with 727.2 K shares traded <3 month average = 777.4 K shares>. Celgene Corporation (CELG) and BLUE announced that updated results from the ongoing CRB-401 P1 clinical study of bb2121, an investigational anti-B-cell maturation antigen (BCMA) CAR T cell therapy, in 21 patients with late-stage relapsed/refractory multiple myeloma will be presented in an oral presentation at the American Society of Hematology (ASH) Annual Meeting in Atlanta, Georgia. “The growing body of bb2121 clinical data are building a compelling story, further supporting the importance of the therapy’s unique features,” said Dave Davidson, M.D., chief medical officer, bluebird bio. “The responses achieved in this relapsed and refractory patient population, combined with the generally tolerable safety profile, reinforce the potential role of bb2121 as a groundbreaking CAR T therapy in multiple myeloma.” One set of initials – ASH spells appreciation. The aftermarket indication is a positive +$2.85 or +1.67% - BUY;

Fate Therapeutics (FATE) closed down -$0.02 to $4.17 and jumped +$0.28 or +6.71% to $4.45 after closing on news of the first subject has been treated in the APOLLO study of FATE-NK100 in women with ovarian cancer resistant to, or recurrent on, platinum-based treatment. FATE also announced the generation of chimeric antigen receptor (CAR)-targeted CD8αβ+ T cells from a clonal engineered master pluripotent cell line (MPCL). The clonal engineered MPCL was created from an induced pluripotent stem cell (iPSC), which was modified in a one-time engineering event using CRISPR/Cas9 to both insert a CAR into the T-cell receptor α constant (TRAC) locus and eliminate T-cell receptor (TCR) expression. The groundbreaking development enables the renewable production of CAR-targeted, TCR-null CD8αβ+ T cells that are not restricted to an individual patient for off-the-shelf administration. – BUY;

Juno Therapeutics (JUNO) closed up +$1.92 to $58.65 with 2.37 M shares traded <3 month average = 2.20 M shares> after Thursday’s +$2.17 to $56.73 with 1.18 M shares traded after Wednesday’s +$1.36 to $54.56 after Tuesday’s -$1.46 to $53.20 with 1.45 M shares trading after last Monday’s -$1.44 to $68.64 with 417.8 K shares traded. November started at $78.56 had a high of $82.06 and a low of $68.25 on 11/14 while October ended at $80.90 having started at $89.37. The aftermarket indication is a POSITIVE +$0.27 or +0.48% – Maintaining BUY;

Regenxbio (RGNX) closed up +$1.45 to $31.75 with 325.7 K shares traded <3 month average = 366 K shares> after Thursday’s +$0.85 to $30.30 with 391.8 K shares traded after Wednesday’s +$1.25 to $29.45 with 198.9 k shares traded <3 month average = 362.9 K shares>. December started out at $28.90 following Novembers end at $28.10 and start at $28.00 with a low of $26.00 and a high of $29.70. On Thursday, There is $3.95 or a 12.4% premium in the share, in the “greedy” environment of five sessions – BUY to SELL;

Verastem (VSTM) closed up +$0.36 to $4.33 with 1.29 M shares traded <3 month average = 752.2 K shares>. ASH news, “In the P3 DUO study, oral duvelisib monotherapy achieved a statistically significant improvement in Progression-Free Survival (PFS) versus the approved standard of care treatment ofatumumab, along with a well characterized and manageable safety profile, in patients with previously treated CLL/SLL,” said Ian Flinn, MD, PhD, Director of the Blood Cancer Research Program at Sarah Cannon Research Institute and lead investigator of the DUO study. “Similar PFS advantages were also observed across all analyzed patient subgroups, including patients with 17p deletion, a genotype that historically correlates with poorer clinical outcomes. Duvelisib also achieved a statistically significant improvement in Overall Response Rate (ORR) and significantly reduced lymph node burden in the vast majority of patients. These data are encouraging for patients with CLL/SLL who progress or relapse following initial treatment.” – BUY;

 

 

Opinions expressed are those of the author and are subject to change, and not intended to be a forecast of future events, a guarantee of future results, nor investment advice.

Whether information or intelligence is good, bad or somewhere in between; I put into context what is relevant and useful for investors.  All investments are subject to risks. Investors should consider investment objectives.

Henry McCusker, the editor and publisher of RegMed Investors does not hold or have positions in securities referred to in this publication.