May 10, 2018 6:57pm

While volume is low, the sector stays on stride as Q1 results head home

 

Q1/18 financial results AKA earnings - snapshots: Athersys (ATHX), BioLife Solutions (BLFS), Biostage (BSTG), BioTime (NYSEMKT: BTX), Caladrius Biosciences (CLBS), Fate Therapeutics (FATE), Histogenics (HSGX) Voyager Therapeutics (VYGR), Solid Bioscience (SLDB), Pluristem (PSTI) and Intrexon (XON)

Out and about: 33 of 45 (covered) reporting so far

Pre-open indications: 3 hits and 3 miss (sold into strength for profit)

 

End of day briefing; the “numbers” speak; what I do in a day is reducing the sector’s “happenings” so you, the investor get to know the perspectives of what transpired today in a 15 minute read to understand what you need to do tomorrow!

Henry’omics:

 

From Thursday’s pre-open newsletter, “…an end in sight for quarterly results lends meaning to volatility.”

Today’s moves … estimates for Q1/18 have taken a beating as LPS (loss-per-share) numbers rule! There has not been any improvement in Q1 relative to what was expected.

 

Some sector “names” stocks kept the lights on: SAGE, QURE, BLUE, SLDB (again and with earnings) and AXGN (after an offering)

While losers hid in the open – RENE.L, BOLD, VYGR, EDIT and GBT (again)

 

The advance/decline line scenario of 45 covered companies:   

  • The open started negative with an ADL of 17/24 and 4 flat;
  • The mid-day stayed positive with an A/DL of 35/10 and 0 flats;
  • The close concluded positive with an A/DL of 26/18 and 1 flat;

 

Pre-open indications – 3 hits and 3 miss:

  • Adverum Biotechnologies (ADVM) closed down -$0.10 – hit;
  • Audentes Therapeutics (BOLD) closed down -$3.35 – hit;
  • AxoGen (AXGN) closed up +$2.15 – miss;
  • Biostage (BSTG) closed up +$0.33 – miss;
  • Ionis Pharmaceuticals (IONS) closed flat $0.00 – hit;
  • Regenxbio (RGNX) closed up +$2.10 – miss;

 

 

MY working five (5) trend lines:

… The greatest volume to the downside:  MDXG, SGMO, VCEL, BOLD and VYGR

… Upside volume was weighted to:  BMRN, ONVO, XON, AXGN and CRSP

… Biggest $ downside:  RENE.L (-$4.00), BOLD (-$3.35), VYGR (-$2.27), EDIT (-$0.90) and GBT (-$0.75)

… Best moves to the $ upside:  SAGE (+$3.69), QURE (+$2.50), BLUE (+$2.50), SLDB (+$2.31) and AXGN (+$2.15)

… Flats:  IONS

 

The month of May:

  • Thursday closed POSITIVE with 18 decliners, 26 advancers and 1 flat;
  • Wednesday closed POSITIVE with 12 decliners, 24 advancers and 2 flats;
  • Tuesday closed NEGATIVE with 28 decliners, 16 advancers and 1 flat;
  • Monday (May 7) closed POSITIVE with 15 decliners, 29 advancers and 1 flat;
  • Friday closed POSITIVE with 8 decliners, 34 advancers and 3 flat
  • Thursday closed NEGATIVE with 29 decliners, 11 advancers and 5 flats;
  • Wednesday closed POSITIVE with 15 decliners, 22 advancers and 8 flats;
  • Tuesday (May 1) closed POSITIVE with 15 decliners, 25 advancers and 5 flats;

 

Daily analytics:

U.S. stock benchmarks Thursday finished the day broadly higher,

  • The Dow ended +0.8% higher at 24,739, posting its sixth straight day of gains, following the release of weaker-than-expected U.S. inflation data
  • The S&P 500 gained 0.94% or to 2,723.07,
  • The NASDAQ advanced 0.89% to close at 7,404.97.

 

The CBOE Volatility Index (VIX) widely considered the best gauge of fear in the market,

  • Thursday traded at 13.23, down -1.42%
  • Wednesday traded at 13.41, down -8.84%
  • Tuesday traded at 14.71, down -0.27%
  •  Monday traded at 14.75, down -0.14%
  • Friday traded at 14.77, down -7.11 %
  • Last Thursday traded at 15.90, down -0.44% …

 

The iShares Russell 2000 (IWM) indicated:

  • Thursday closed up +0.53 %
  • Wednesday +0.56%
  • Tuesday +0.78%
  • Monday was up +0.84%
  • Friday +1.22%
  •  Last Thursday -0.55%

 

The iShares NASDAQ Biotechnology (IBB) indication:

  • Thursday closed down -0.06%
  • Wednesday +1.35%
  • Tuesday -0.57%%
  • Monday was up +0.64%
  • Friday +1.06%
  • Last Thursday -1.36%

 

The count in May - decliners versus gainers:

……. look at the differences in decliners:

  • Thursday’s decliners ranged from -0.24% <BLFS -$0.02 > to -12% <KOOL -$0.18 > in 18 equities;
  • Wednesday’s decliners ranged from -0.38% <HSGX -$0.01> to -7.49% <MDXG -$0.60 > in 12 equities;
  • Tuesday’s decliners ranged from -0.18% <BMRN -$0.16 > to -9.85% <SGMO -$1.60 > in 28 equities;
  • Monday’s decliners ranged from -0.25% < RGNX -$0.10> to -5.67% <SLDB -$1.07> in 15 equities;
  • Friday’s decliners ranged from -0.28% <XON -$0.05 > to -16.14% <ADVM -$1.025 > in 8 equities;
  • Last Thursday’s decliners ranged from -0.08% <BMRN -$0.07> to -4.97% <SGMO -$0.85 > in 29 equities;

Versus

… Look at the percentage’s (%) and spreads …

  • Thursday’s gainers ranged from +0.11% <ONCE +$0.08 > to +10.91% <BSTG +$0.33 > in 26 equities;
  • Wednesday’s gainers ranged from +0.24% <VSTM +$0.01> to +16.77% <SLDB +$2.89 > in 34 equities;
  • Tuesday’s gainers ranged from +0.11% <XON +$0.02 > to +7.96% <GBT +$3.60 > in 16 equities;
  • Monday’s gainers ranged from +0.17% <BLUE +$0.30 > to +7.85% <BCLI +$0.27 > in 29 equities;
  • Friday’s gainers ranged from +0.17% <SAGE +$0.25 > to +17.20% <SLDB +$2.77 > in 34 equities;
  • Last Thursday’s gainers ranged from +0.45% <BTX +$0.01 > to +8% <AST +$0.10 > in 11 equities;

                                               

Q1/18 financial results i.e. earning – the LPS (loss-per-share) skirmish:

Pluristem (PSTI) …

Platform development:

  • PLX-PAD (Critical Limb Ischemia has launched a P3 study (N=246) in CLI patients for the US and EU.
  • PLX-PAD (Muscle Regeneration Following Hip Replacement) has been cleared to initiate a P3 in 2018 for muscle recovery following femoral neck fracture surgery.
  • The Acute Radiation Syndrome (ARS) program reported positive results from a p2-equivalent study in non-human primates. The company is now in discussions with the FDA and several U.S. governmental agencies to clear the path for a pivotal study.
  • PLX-R18 (Insufficient HCT Recovery is currently under a P1 trial (N=24) for insufficient hematopoietic cell transplant recovery.

The “numbers”:

Reported a loss of $1.5 million in its Q3 or -$0.01 per share

Cash was reported as $34.1 M

“Runway”: Q1/2020

 

Histogenics (HSGX) …

Platform development:

  • Superiority Data from NeoCart® P3 clinical trial and potential biologics license application submission on track for Q3/18,
  • Initiation of P3 Clinical Trial in Japan Expected in Second Half of 2018,
  • Received a $10 million up-front payment from MEDINET Co., Ltd in a license agreement for the development and commercialization of NeoCart in Japan 

The “numbers”:

Net loss was $14.4 million or $0.52 per share, compared to $5.8 million, or $0.27 per share in Q1/17,

Cash, cash equivalents and marketable securities of $15.5 million, compared to $8.0 million at 12/31/17.

“Runway”:  Q4/18

 

Solid Biosciences (SLDB) …

Platform development:

  • Receipt of a full clinical hold letter from the FDA relating to IGNITE DMD, its P1/2 clinical trial for its investigational gene therapy, SGT-001, for the treatment of Duchenne muscular dystrophy (DMD). The clinical hold is in response to an unexpected Serious Adverse Event (SAE) reported in the first patient dosed with SGT-001. In its clinical hold letter, the FDA requested additional information, including an assessment of the underlying etiology of the event, the patient’s clinical status and laboratory parameters, and any additional measures to address patient safety.

The “numbers”:

Net loss of $15.9 million or -$0.54 per share as compared to $13.9 million for Q1/17,

  • Completed its initial public offering, issued and sold 8,984,375 shares of common stock, including 1,171,875 shares of common stock pursuant to the underwriters’ overallotment, at a public offering price of $16.00 per share; SLDB received total net proceeds of approximately $129.1 million from the offering after discounts, commissions and offering expenses,

Ended with $182.4 million in cash, cash equivalents and available-for-sale securities as compared to $69.1 million as of 12/31/17,

“Runway”: 2021

 

Biostage (BSTG) …

Platform development:

  • "Preliminary" - (we've heard that before) findings related to the first successful in-human use of a Cellspan™ implant to regenerate a patient's esophagus

The “numbers”:

Reported a net loss of $1.5 million, or $0.56 per diluted share, compared to $3.8 million, or $2.83 per diluted share in Q1/17;

Reported $2.8 million in cash

Runway”: Q1/19

 

Voyager therapeutics (VYGR) …

Platform development:

  • Clearance of the IND application by the FDA for VY-AADC in January 2018 to begin activating clinical trial sites for its pivotal P2/3 program for Parkinson’s disease,
  • Exclusive strategic collaboration and option agreement with AbbVie to develop and commercialize vectorized antibodies directed against tau for the treatment of Alzheimer’s disease and other neurodegenerative diseases, combining VYGR’s gene therapy platform with AbbVie’s monoclonal antibody expertise, global clinical development and commercial capabilities.

The “numbers”:

Net loss of $19.9 million, or $0.63 per share compared to a GAAP net loss of $16.6 million, or $0.65 per share, for Q1/17,

Cash, cash equivalents were $218.2 million.

 “Runway”: early 2020

 

Athersys (ATHX) …

Platform development:

  • Advanced preparations for the MASTERS-2 P3 registration study for ischemic stroke to enable initiation of this important study and supported the continued enrollment of Healios’ TREASURE study;
  • Progressed P1/2 study evaluating MultiStem therapy in acute respiratory distress syndrome (ARDS) patients;
  • Announced plans with The University of Texas Health Science Center at Houston to conduct a P2 clinical trial evaluating MultiStem cell therapy for early treatment and prevention of complications after severe traumatic injury;

The “numbers”:

Net loss of $10.2 million, or $(0.08) per share compared to $5.6 million or -$0.06 per share in Q1/17

  • Completed a $21.1 million equity investment and plans to expand the HEALIOS K.K. collaboration by June 1, 2018; proposed expansion includes a $10 million license fee funded in an escrow account and a potential additional $25 million of committed payments over time, as well as additional possible payments including milestones and royalties, if the collaboration is fully expanded in accordance with the letter of intent disclosed in March 2018;

Cash and cash equivalents of $49.78 million, excluding the $10 million in escrow from Healios related to the proposed collaboration expansion,

Established a new equity facility with Aspire Capital Fund LLC, as a follow-on to a prior facility, providing access to additional capital as needed;

“Runway”: Q1/2020

 

Caladrius Biosciences (CLBS)

Platform development:

  • Dosed the first patient in a P2 clinical trial in Japan with Caladrius’ proprietary CD34 cell therapy (CLBS12) for the treatment of no-option critical limb ischemia (“CLI”);
  • Received SAKIGAKE designation from the Japan Ministry of Health, Labour and Welfare (the “MHLW”) for CLBS12 for the treatment of CLI, which reflects MHLW’s expectation of “prominent effectiveness” based on data of mechanism of action from non-clinical and early phase of clinical trials and provides an expedited path to potential conditional approval in Japan for products that show sufficient safety evidence and signals of efficacy in a P2 study;
  • Acquired an exclusive worldwide license to data and regulatory filings from Shire plc (SHP.L) (SHPG) for a late-stage CD34 cell therapy program for the treatment of chronic myocardial ischemia targeting refractory angina;
  • Reactivated the investigational new drug application (“IND”) with the U.S. Food and Drug Administration (“FDA”) for the program licensed from Shire treating chronic myocardial ischemia targeting refractory angina;
  • Enrolled the first patient in the study of CD34 cell therapy (CLBS14-CMD) for the treatment of coronary microvascular dysfunction.

The “numbers”:

The net loss was $5 million, or $0.52 per share, compared with $6.6 million, or $0.78 per share, for Q1/17,

Cash, cash equivalents, restricted cash and marketable securities of $53.6 million

 “Runway”: Q1/2020

 

BioLife Solutions (BLFS) …

Platform:

  • 61% revenue growth at $3.8 Million
  • Operating profitability achieved
  • Gained 19 new direct customers, including several T cell therapy startups,

The “numbers”:

  • Product revenue: $2.1 million; 55% of total revenue, with 100% growth over Q1/17,

Net loss was $103,000, or $0.01 per share, compared to $870,000, or $0.07 per share in Q1/17,

Cash balance was $7 million compared with $2.3 million in Q1/17,

  • BLFS received $4.4 million in cash proceeds from the exercise of outstanding warrants

 “Runway”: N/A

 

BioTime (NYSEMKT: BTX) …

Platform development:

  • Submitted Renevia® for CE Marking in Europe,
  • U.S. investigator-initiated study in facial aesthetics is continuing to enroll patients.
  • OpRegen® (dry-AMD): the independent Data Safety Monitoring Board approved initiation of the fourth cohort for the OpRegen® clinical trial in patients in the advanced stage of the dry form of age-related macular degeneration.
  • Positive OpRegen® clinical trial data from the first nine patients from the first three cohorts were presented at the Annual Meeting of the Association for Research in Vision and Ophthalmology in Honolulu, Hawaii,
  • Ascendance Biotechnology was sold in March 2018. AgeX received approximately $3.2 million for its interest in Ascendance.

The “numbers”:

Net loss was $63.5 million, or ($0.50) per share, compared to net income attributable to BioTime of $49.3 million, or $0.46 per share, for Q1/17,

Cash, cash equivalents were $31.4 million, compared to $38.2 million as of 12/31/17,

 “Runway”: 2020

 

Intrexon (XON) …

Platform development:

  • ActoBio Therapeutics and collaborator Intrexon T1D Partners, LLC, have been granted allowance by the FDA for their IND application to initiate a PIb/2a study for the treatment of early onset type 1 diabetes with AG019, an innovative disease-modifying approach to induce immune tolerance;
  • Intrexon's Energy team demonstrated successful third party catalytic conversion of 2, 3 BDO to 1, 3 butadiene;
  • Exemplar Genetics, a wholly owned subsidiary of Intrexon, announced the FDA exercised enforcement discretion clearing for commercial use as a research model the ExeGen® ATM MiniSwine, which is genetically engineered to model ataxia telangiectasia (AT), a rare, inherited, predominantly neurological human disease. Following Exemplar's previous approval of its ExeGen® LDLR MiniSwine model for use in cardiovascular disease research, the ExeGen® ATM model is the second engineered MiniSwine model reviewed and cleared by the FDA;
  • Okanagan Specialty Fruits (OSF), a wholly owned subsidiary of Intrexon, launched the sales of dried Arctic® Goldens – Arctic ApBitz™ apple snacks – via Amazon;
  • Intrexon's Industrial Products Division has demonstrated microbial production of cannabinoids that has potential to provide >20-fold reduction in Cost of Goods with reduced environmental impacts for THC and CBD versus current synthetic and extraction-based routes;
  • Collaborator Fibrocell Science, Inc. (FCSC) obtained allowance from the FDA to begin clinical trials for FCX-013, its gene therapy candidate for the treatment of moderate to severe localized scleroderma; and

The “numbers”:

Net loss of $42.0 million attributable to Intrexon, or $(0.33) per basic share, including non-cash charges of $26.3 million;

Cash, cash equivalents, and short-term investments totaled $120.2 million

“Runway”: 2021

 

Fate Therapeutics (FATE) …

Platform development:

  • Reported new ProTmune™ clinical data showing NO events of cancer relapse: presented additional P1 PROTECT data from seven subjects administered ProTmune, the Company’s next-generation hematopoietic cell graft for patients with hematologic malignancies. As of a February 26, 2018 data cut-off, with a median time on study of 228 days, no serious adverse events related to ProTmune and no events of cancer relapse had been reported by investigators. The randomized, controlled and double-blinded Phase 2 PROTECT study is currently open for enrollment at 15 U.S. centers.
  • Advancing FATE-NK100 in Multiple Phase 1 Studies; the first subject was administered NK100, the Company’s first-in-class, and donor-derived adaptive memory natural killer (NK) cell cancer immunotherapy, in the DIMENSION study.
  • Universal Off-the-Shelf Cancer Immunotherapy preclinical programs – Highlights & Updates
  • Completed key activities to support FT500 IND submission,
  • Presented breakthrough preclinical data demonstrating the dual-targeted anti-tumor activity of FT819, a universal, off-the-shelf CAR19 T-cell product

The “numbers”:

  • Secured $4M from CIRM to Advance FT516 into a First-in-Human Clinical Trial. The award from the California Institute for Regenerative Medicine (CIRM) is being used to support ongoing IND-enabling activities,

Net loss of $14.1 M or -$0.27 per share as compared to $10.15 M or -$0.24 in Q1/17,

Cash, cash equivalents and short-term investments as of 3/31/18 were $88.6 million compared to $100.9 million as of 12/31/17;

 “Runway”: Q1/2020

 

                                                                                                                                                                               

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.