May 8, 2018 6:47pm

LPS (loss-per-share) numbers were on display with a net income by Regenxbio (RGNX)

With seven (7) reporting today, all but, one is down as investors are hitting the SELL button

 

Q1/18 AKA earnings: CRISPR Therapeutics (CSPR -$0.62 per share), Vericel (VCEL -$0.21 per share), Sangamo Therapeutics (SGMO -$0.23 per share), Spark Therapeutics (ONCE -$1.25 per share), Applied Genetic Technologies (AGTC -$0.45 per share), Bellicum Pharmaceuticals (BLCM - $0.68 per share) and Regenxbio (RGNX –net income of +$3.30 per basic share) … read more of each platform’s development, the “numbers” breakdown and “runway” extension …

 

Pre-open indications: 2 miss

 

End of day briefing; the “numbers” speak; what I do in 8-10 hours is reducing the content so you, the investor get to know what happened today in a 15 minute read to understand what you need to do tomorrow!

Henry’omics:

Today’s moves … the overbought got trampled and sold; the algorithms only let you thrive for so long …

Retail (investing) activity in the market has also been rising and accounted for the bulk of equity ownership, but their share of trading has surged in early 2018. < Goldman Sachs>

  • That surge in trading occurred at a time when composite trading volumes were otherwise low.

Those factors meant that retail trading had a bigger impact on overall trading, which contributed to the market’s volatility, in Goldman’s estimation.

  • Retail investors apparently have been reducing positions as well as broad equity market exposure.

SO, sector executive had BETTER get-on-the-stick and start RE-COURTING investors to SPUR momentum!

 

Some sector “names” stocks kept the lights on: GBT, SAGE (again), VCEL (earnings), NTLA and CRSP (again and after being down -$1.50 pops +$0.25)

While losers hid in the open – IONS, ONCE (earnings), QURE, BLUE and SGMO (earnings)

 

Indexes saw some rough swings with concerns over geopolitical uncertainty – Iran and North Korean, never forgetting Russia and predominately Q1 financial results AKA earnings season!

Retail investors individually control small amounts of money relative to portfolio managers or other professional market participants; however, their collective influence in the market is sizable! <MarketWatch>

 

The advance/decline line scenario of 45 covered companies:   

  • The open started negative with an ADL of 18/26 and 1 flat;
  • The mid-day stayed negative with an A/DL of 13/28 and 4 flats;
  • The close concluded negative with an A/DL of 16/28 and 1 flat;

 

From Tuesday’s pre-open newsletter, “… pick a word, any term for weakness; my word is short-coming in regard to quarterly results. Watch total operating expense and SG&A expense as conditions of net loss.”

I also stated, “Consensus is about to be a smack to pricing as LPS (loss-per-share) of the smaller-caps. I believe there is more to cash positions than ever before. Could be a flip side day in the sector; Q1 results are going to drag down, shove or be pulled but, you’re still going down!”

 

Pre-open indications:

  • AxoGen (AXGN) closed up +$0.20 – miss;
  • Ultragenyx Pharmaceuticals (RARE) closed down -$0.1 – miss;

 

MY working five (5) trend lines:

… The greatest volume to the downside:  IONS, SGMO, CRSP, ALNY and ONCE

… Upside volume was weighted to:  VCEL, GBT, MDXG, BMRN and NTLA

… Biggest $ downside:  IONS (-$3.81), ONCE (-$3.44), QURE (-$2.14), BLUE (-$1.95) and SGMO (-$1.60),

… Best moves to the $ upside:  GBT (+$2.50), VCEL (+$0.52), AXGN (+$0.48), SAGE (+$1.13) and NTLA (+$0.34)

… Flats:  BSTG and RENE.L

 

The month of May:

  • Tuesday closed NEGATIVE with 28 decliners, 16 advancers and 1 flats;
  • 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;

 

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

Spark Therapeutics (ONCE) …

Platform development;

  • Launched LUXTURNA in U.S., with three patients treated at different treatment centers – Massachusetts Eye and Ear in Boston, Bascom Palmer Eye Institute in Miami and The Vision Center at Children’s Hospital of Los Angeles,
  • Progressed Marketing Authorization Application with European Medicines Agency (EMA) for investigational voretigene neparvovec; on track for regulatory action in Q3/18,
  • Advanced two investigational hemophilia programs with no reported serious adverse events, thrombotic events or inhibitors to date

The “numbers”:

Net loss applicable was $46.4 million, or ($1.25) per share, as compared to $52.3 million, or ($1.70) for Q1/17,

$587.5 million in cash, cash equivalents,

  • Entered into agreement to sell rare pediatric disease priority review voucher for $110 million

 

CRISPR Therapeutics (CRSP) …

  • Remains on track for CTX001 in β-thalassemia and sickle cell disease (SCD), after announcing the acceptance of the first Clinical Trial Application (CTA), CRISPR, together with its partner, Vertex, remains on track to initiate a P1/2 trial to assess the safety and efficacy of CTX001 in patients with transfusion dependent β-thalassemia in the second half of 2018,
  • Remains on track to begin clinical studies for CTX001 in SCD in 2018, CTX001 is an autologous gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies,
  • Advances wholly-owned allogeneic CRISPR-based CAR-T cell therapies,
  • Remains on track to file an IND for CTX101, its lead allogeneic CAR-T cell therapy targeted toward CD19+ malignancies, by year-end 2018.

The “numbers”:

Net loss was $28.3 million or -$0.62 compared to a loss of $21.5 million or -$0.54 per share for Q1/17 driven predominantly by increased R&D expense,

  • Collaboration revenues were $1.4 million compared to $2.7 million for Q1/17,
  • R&D expenses were $19.5 million compared to $14.8 million for Q1/17,
  • Completed successful follow-on offering in January 2018, completing  a follow-on offering, including full exercise of the overallotment, totaling 5.75 M shares of its common stock at a price of $22.75 for net proceeds of $122.6 million,

Cash as of March 31, 2018 was $341.8 million, compared to $239.8 million as of 12/ 31/17,

“Runway”:

 

Sangamo Therapeutics (SGMO) …

Platform development:

  • Treated the fourth patient in the SB-525 P1/2 Alta Study for hemophilia A
  • Expects potential clinical data readouts from 7 studies in 2018 and 2019, beginning in late summer of this year with anticipated data from our hemophilia A gene therapy and MPS II genome editing programs,
  • Treated the fourth patient in the SB-913 P1/2 CHAMPIONS Study for MPS II
  • Received Clinical Trial Authorization (CTA) from the MHRA of the U.K. for enrollment of subjects in the ongoing P1/2 clinical trial of SB-FIX for hemophilia B. (The CTA allows enrollment of adolescent patients, ages 12-17, once preliminary safety and efficacy have been demonstrated in adults),
  • Awarded an $8 million grant from the California Institute of Regenerative Medicine (CIRM) to evaluate ST-400, a gene-edited cell therapy candidate, for the treatment of transfusion-dependent beta-thalassemia. ST-400 is being developed in collaboration with Bioverativ, a Sanofi Company,
  • After demonstrating safety at the first dose cohort in the SB-913 MPS II clinical trial, amended P1/2 study protocol for SB-318 MPS I trial to begin enrolling patients directly into the second dose cohort,

The “numbers”:

Net loss of $20.2 million or $0.23 per share compared to a net loss of $16.6 million or $0.23 per share, for Q1/17.

  • Revenues were $12.6 million, compared to $3.4 million 2017.

Cash, cash equivalents, marketable securities and interest receivable of $234.9 million; this balance does not include the $150 million upfront payment from the collaboration agreement with KITE, effective April 5th, or the approximately $216 million in net proceeds from the recent offering (4/30). Total: $600.9 M

“Runway”: early 2021

 

Vericel (VCEL) …

Platform development:

  • Deployed the expanded MACI (autologous cultured chondrocytes on porcine collagen membrane) sales force, which increased from 28 to 40 sales representatives;

The “numbers”:

Net loss of $7.7 million, or $0.21 loss per share, which included warrant-related expense of $2.9 million, compared to net loss of $9.8 million, or $0.31 per share, in Q1/17 which included $100 K of warrant income;

  • Total net revenues of $18 million compared to $9.4 million in Q1/17; which also included a $2.8 million revenue reserve for Carticel® and MACI® related to a contractual dispute between one pharmacy providers and a third-party payer; 
  • Gross margins of 57% compared to gross margins of 24% in Q1/17;
  • Total operating expenses were $14.7 million compared to $11.9 million in Q1/17,

In October 2016, VCEL entered an ATM with Cowen to raise up to $25M with NO mention of long-term debt of $15.46 M or is it part of ATM usage?

Cash of $29.8 million in cash compared to $26.9 million in cash at 12/311/17,

 

Applied Genetic Technologies (AGTC) … Q3

Platform development:

  • AGTC completed enrollment of the P1/2 clinical study of investigational gene therapy in patients with X-linked retinoschisis (XLRS), in collaboration with Biogen.
  • AGTC dosed the first patient in the P1/2 clinical trial evaluating the safety and biologic activity of an investigational AAV-based gene therapy for the treatment of X-linked retinitis pigmentosa caused by mutations in the RPGR gene (XLRP) and, as a result, received a $2.5M milestone payment under its collaboration agreement with Biogen.

The “numbers”:

  • Revenue was $3.6 million and $18.8 million or -$0.81 per share for the nine months ended March 31, 2018, compared to $8.4 million or -$0.07 per share and $31.1 million or -$0.31 per share in the comparable periods in 2017.

The net loss was $8.1 million or -$0.45 per share

Cash, cash equivalents and investments amounted to $111.8 million.

“Runway”: 2020

 

Bellicum Pharmaceuticals (BLCM ) …

Platform development:

Clinical Hold Lifted by FDA on U.S. Studies of BPX-501 and is now working with U.S. clinical sites to resume patient recruitment based on amended study protocols, including guidance on monitoring and management of neurologic adverse events. The FDA clinical hold did not affect the BP-004 registration trial in Europe.

The “numbers”:

Net loss of $22.8 million or -$0.68 per share as compared to $22 million or -$0.80 per share  in Q1/17; results included non-cash, share-based compensation charges and depreciation of $5 million in Q1 versus $4.1 million in Q1/17.

  • In April completed an offering of 9.2 million shares, including 1.2 million shares sold under the underwriters’ option to purchase additional shares, at $7.50 per share. The aggregate offering size was $69.0 million before deducting the underwriting discounts and commissions and other offering expenses,

Cash, restricted cash and investments totaling $88 million, compared to $106.5 million at the end of Q4/17,

 “Runway”: the end of 2019

 

Regenxbio (RGNX) …

Platform development:

In January 2018, RGNX and AveXis, Inc. amended their license agreement for the development and commercialization of treatments for spinal muscular atrophy (SMA). Under the terms of the amended agreement, REGENXBIO could receive up to $260 million, of which $80 million was received in January 2018. In addition to the $80 million, RGNX will receive payments of $30 million on the first and second anniversaries of the agreement and is eligible to receive potential commercial milestone payments of up to $120 million,

  • Completed dosing of third cohort in RGX-314 PI clinical trial for wet AMD,
  • Completed dosing of third patient in second cohort in RGX-501 P1/2 clinical trial for HoFH,
  • Continue to plan to present topline data from RGX-314 and RGX-501 clinical trials in late 2018,
  • Expect to initiate dosing in clinical trials for RGX-111 for MPS I and RGX-121 for MPS II in mid-2018
  • In April 2018, Ultragenyx Pharmaceutical (RARE) announced that the investigational new drug application is active for DTX401 for the treatment of glycogen storage disease type Ia. DTX401 uses RGNX’s NAV AAV8 vector.

The “numbers”:

Net income was $104.2 million, or $3.30 basic and $3.04 diluted net income per share, for Q1, compared to a net loss of $22 million, or $0.82 basic and diluted net loss per share, for Q1/17. Net income for the three months ended March 31, 2018 was primarily driven by the non-recurring license revenue recognized upon the amendment of the license agreement with AveXis.

  • Revenues were $132.4 million forQ1/18, compared to $500 K in Q1/17,

Cash, cash equivalents and marketable securities were $235.8 million as of 3/31/18, compared to $176.4 million as of 12/31/17; which include the $80 million received from AveXis in January 2018,

“Runway”: 2021

 

Daily analytics:

Stocks closed little changed on Tuesday after President Donald Trump announced he was withdrawing the U.S. from the Iran nuclear deal signed three years ago.

  • The Dow finished 2.89 points (+0.01%) higher at 24,360.21,
  • The S&P 500 closed -0.71 (-0.03%) just below breakeven at 2,671.92.
  • The NASDAQ eked out a small gain of +1.69 (+0.02%) to close at 7,266.90.

 

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

  • Tuesday traded at 14.71, down -0.27%
  • Monday traded at 14.75, down -0.14%
  • Friday traded at 14.77, down -7.11 %
  • Thursday traded at 15.90 , down -0.44% …
  • Wednesday traded at 15.97, up +3.10 %
  • Last Tuesday traded at 15.49, down -2.76%

 

The iShares Russell 2000 (IWM) indicated:

  • Tuesday was up +0.78%
  • Monday was up +0.84%
  • Friday +1.22%
  • Thursday -0.55%
  • Wednesday +0.36%
  • Last Tuesday +0.46%

 

The iShares NASDAQ Biotechnology (IBB) indication:

  • Tuesday was down -0.57%%
  • Monday was up +0.64%
  • Friday +1.06%
  • Thursday -1.36%
  • Wednesday -1.32%
  • Last Tuesday +0.39%

 

The count in May - decliners versus gainers:

……. look at the differences in decliners:

  • 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;
  • Thursday’s decliners ranged from -0.08% <BMRN -$0.07> to -4.97% <SGMO -$0.85 > in 29 equities;
  • Wednesday’s decliners ranged from -0.11% <XON -$0.02 > to -4.40% < BMRN -$3.79> in 15 equities;
  • Last Tuesday’s decliners ranged from -0.75% <VCEL -$0.10> to -17.33% <RENE.L> in 15 equities;

Versus

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

  • 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;
  • Thursday’s gainers ranged from +0.45% <BTX +$0.01 > to +8% <AST +$0.10 > in 11 equities;
  • Wednesday’s gainers ranged from +0.26% <IONS +$0.11 > to +5.66% <SLDB +$0.83 > in 22 equities;
  • Last Tuesday’s gainers ranged from +0.45% <FIXX +0.09> to +5.30% <PSTI +$0.07> in 25 equities;

                                                                                                                                                                               

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.