• The US FDA has just approved the most expensive drug
  • CSL Behring’s hemophilia B gene therapy costs US$3.5m a dose
  • A quick look at the best and worst performing stocks on the ASX

 

The US FDA has just approved the world’s most expensive drug.

At US$3.5m a pop, CSL Behring’s hemophilia B gene therapy is a one-off infusion that frees patients from regular treatments.

The drug has been proven to cut the number of bleeding events caused by hemophilia over the course of a year by 54%.

It frees 94% of patients from the time-consuming infusions of blood proteins, which is also a very expensive process.

Hemophilia B is a rare genetic bleeding disorder in which affected individuals have insufficient levels of Factor IX, a clotting protein.

These clotting factors are what the body needs to form clots and stop bleeding, and Hemgenix works by delivering a gene that can produce the missing clotting factors into the liver, where it starts working to make the Factor IX protein.

Although it is usually passed down from parents to children, about one third of cases of hemophilia are caused by a spontaneous mutation or a change in a gene.

While there have been advances in treatments, exisiting drugs mostly erode a patient’s quality of life.

Experts believe that CSL Behring’s hemophilia B gene therapy may be well received because those existing drugs are also very expensive, and hemophilia patients constantly live in fear of bleeds.

CSL Behring says the drug will be manufactured in Massachusetts by uniQure NV, which sold the commercialisation rights for Hemgenix to CSL Behring in 2020.

CSL Behring’s parent company, CSL Limited (ASX:CSL) is headquartered in Melbourne.

Other expensive drugs in the world

In general, gene therapies could be very expensive for the average patient.

The groundbreaking Biogen’s Alzheimer’s drug Aduhelm has a yearly maintenance dose (10mg/kg) cost of US$28,200 for those without private insurance.

Bluebird Bio’s blood disorder gene therapy Zynteglo, which was approved by the FDA in August, costs US$2.8m a patient.

Zynteglo is a one-time treatment for a blood disorder known as beta thalassaemia in patients 12 years and older who require regular blood transfusions.

Meanwhile Novartis’ Zolgensma for babies with spinal muscular atrophy had a price tag of $US2.1 million when it was approved in 2019.

Zolgensma is currently the only approved gene therapy for the treatment of spinal muscular atrophy.

 

NOW TO THE ASX….

Best and worst performing ASX biotechs over the past month

CODE COMPANY PRICE 1 MONTH RETURN % 1 YEAR RETURN % MARKET CAP
MAP Microba Life Sciences 0.34 90% -3% $77,110,000.00
JTL Jayex Technology Ltd 0.008 60% -1% $1,993,828.31
CU6 Clarity Pharma 0.98 46% 24% $179,686,992.87
NSB Neuroscientific 0.12 45% -22% $17,216,597.16
IMM Immutep Ltd 0.375 34% -12% $320,867,650.35
PAB Patrys Limited 0.025 32% -1% $57,595,993.20
BPH BPH Energy Ltd 0.022 29% -1% $17,261,294.00
GSS Genetic Signatures 0.905 28% -89% $129,065,396.40
ILA Island Pharma 0.205 28% -5% $8,865,497.24
LCT Living Cell Tech. 0.0165 27% 1% $21,208,501.43
EZZ EZZ Life Science 0.37 21% -14% $4,721,200.00
ALC Alcidion Group Ltd 0.155 19% -12% $196,550,703.22
EXL Elixinol Wellness 0.031 19% -4% $10,753,029.41
M7T Mach7 Tech Limited 0.655 19% -17% $155,440,597.65
ARX Aroa Biosurgery 1.02 19% 0% $368,428,523.60
NC6 Nanollose Limited 0.071 18% -3% $10,570,932.13
EYE Nova EYE Medical Ltd 0.3 18% -3% $43,765,034.70
GTG Genetic Technologies 0.0035 17% 0% $27,701,895.43
NEU Neuren Pharmaceutical 8.38 16% 461% $1,032,918,543.20
PCK Painchek Ltd 0.038 15% -1% $49,263,404.55
OCC Orthocell Limited 0.465 15% -5% $91,699,428.02
VBS Vectus Biosystems 1.05 14% -36% $49,613,872.35
VTI Vision Tech Inc 0.3 13% -58% $9,398,730.90
CTE Cryosite Limited 0.75 13% 29% $36,607,172.25
MDR Medadvisor Limited 0.2 11% -20% $108,769,683.40
PSQ Pacific Smiles Grp 1.6 10% -133% $252,139,462.04
CYP Cynata Therapeutics 0.35 9% -20% $50,146,807.90
ONE Oneview Healthcare 0.13 8% -14% $69,278,911.52
IMU Imugene Limited 0.1775 8% -22% $1,149,381,272.88
PBP Probiotec Limited 2.34 7% 14% $190,296,770.04
RGS Regeneus Ltd 0.044 7% -4% $13,483,224.22
PYC PYC Therapeutics 0.074 6% -6% $235,388,531.62
IMC Immuron Limited 0.078 5% -2% $17,768,270.99
BNO Bionomics Limited 0.062 5% -5% $91,061,596.29
AMT Allegra Orthopaedics 0.105 5% -5% $10,968,216.32
SDI SDI Limited 0.865 5% -15% $102,818,683.45
ANP Antisense Therapeut. 0.088 5% -11% $60,213,958.02
IPD Impedimed Limited 0.091 5% -8% $162,270,213.11
AHC Austco Healthcare 0.115 5% -3% $33,328,994.45
PGC Paragon Care Limited 0.36 4% 0% $233,002,804.80
MVF Monash IVF Group Ltd 1.005 4% -5% $389,634,840.00
TLX Telix Pharmaceutical 7.115 4% -64% $2,286,687,232.72
ICR Intelicare Holdings 0.026 3% -6% $3,132,123.98
CDX Cardiex Limited 0.31 3% -38% $39,224,862.65
CGS Cogstate Ltd 1.89 3% -53% $326,057,796.24
DXB Dimerix Ltd 0.165 3% -8% $52,944,154.89
PAR Paradigm Bio. 1.375 3% -50% $391,970,527.20
CMP Compumedics Limited 0.23 2% -14% $40,747,478.04
ATX Amplia Therapeutics 0.099 2% -7% $19,012,542.53
ADO Anteotech Ltd 0.056 2% -25% $109,337,042.65
PXS Pharmaxis Ltd 0.063 2% -6% $39,979,927.89
HGV Hygrovest Limited 0.07 1% 1% $16,096,778.95
CHM Chimeric Therapeutic 0.085 1% -17% $28,344,556.51
PNV Polynovo Limited 1.97 1% 45% $1,314,306,384.92
TD1 Tali Digital Limited 0.0025 0% -2% $3,394,076.31
ALT Analytica Limited 0.001 0% 0% $4,613,801.13
BXN Bioxyne Ltd 0.024 0% -1% $15,975,489.55
RCE Recce Pharmaceutical 0.68 0% -35% $120,203,534.25
TSN The Sust Nutri Grp 0.14 0% -6% $16,884,893.90
SCU Stemcell United Ltd 0.013167 0% 0% $14,995,836.55
MEB Medibio Limited 0.001 0% 0% $3,320,593.79
ICS ICSGlobal Limited 0.575313 0% 0% $6,054,604.94
AC8 Auscann Grp Hlgs Ltd 0.04 0% -4% $17,621,884.40
BWX BWX Limited 0.63 0% -368% $125,992,126.26
HCT Holista CollTech Ltd 0.03 0% -2% $8,364,002.01
MX1 Micro-X Limited 0.135 0% -12% $65,583,725.63
RSH Respiri Limited 0.04 0% -2% $32,850,690.19
OVN Oventus Medical Ltd 0.02 0% -7% $4,834,530.70
1ST 1St Group Ltd 0.009 0% 0% $11,593,564.34
SHG Singular Health 0.125 0% -16% $8,161,133.63
CAN Cann Group Ltd 0.2325 -1% -6% $82,138,421.53
PIQ Proteomics Int Lab 0.88 -1% -34% $101,140,123.88
BOT Botanix Pharma Ltd 0.062 -2% 1% $71,044,203.45
VLS Vita Life Sciences.. 1.5 -2% -40% $79,511,418.00
IIQ Inoviq Ltd 0.56 -2% -64% $52,450,660.14
MVP Medical Developments 1.6 -2% -332% $138,951,331.75
LDX Lumos Diagnostics 0.049 -2% -88% $10,285,418.35
OPT Opthea Limited 0.97 -2% -33% $451,455,906.20
OSL Oncosil Medical 0.048 -2% 0% $47,723,628.58
1AD Adalta Limited 0.048 -2% -3% $15,080,867.81
CBL Control Bionics 0.165 -3% -26% $8,304,599.49
NOX Noxopharm Limited 0.16 -3% -23% $48,219,261.75
CYC Cyclopharm Limited 1.3 -4% -34% $120,969,973.80
RNO Rhinomed Ltd 0.12 -4% -19% $32,857,764.81
RAD Radiopharm 0.115 -4% -27% $25,138,817.55
RAD Radiopharm 0.115 -4% -27% $25,138,817.55
CAJ Capitol Health 0.315 -5% -8% $334,817,350.56
IVX Invion Ltd 0.0105 -5% -1% $67,410,966.85
RHT Resonance Health 0.061 -6% -8% $28,111,970.84
PTX Prescient Ltd 0.15 -6% -8% $107,971,431.90
AT1 Atomo Diagnostics 0.057 -7% -24% $32,540,786.49
DVL Dorsavi Ltd 0.012 -8% -1% $5,220,074.95
RAC Race Oncology Ltd 2.2 -8% -140% $351,400,360.68
LBT LBT Innovations 0.055 -8% -6% $18,184,891.29
ACR Acrux Limited 0.075 -9% -3% $21,464,361.30
UBI Universal Biosensors 0.235 -10% -76% $50,842,664.40
IHL Incannex Healthcare 0.23 -10% -40% $358,044,994.67
HXL Hexima 0.018 -10% -40% $2,875,297.52
AVE Avecho Biotech Ltd 0.009 -10% -1% $16,540,824.10
AHK Ark Mines Limited 0.265 -10% 3% $9,145,177.30
IBX Imagion Biosys Ltd 0.026 -10% -5% $29,714,941.15
BDX Bcaldiagnostics 0.059 -11% -7% $8,029,587.89
VHT Volpara Health Tech 0.605 -11% -44% $151,234,145.40
OSX Osteopore Limited 0.18 -12% -5% $21,108,282.84
RHY Rhythm Biosciences 1.105 -12% -45% $241,056,023.79
SOM SomnoMed Limited 1.36 -13% -99% $112,552,668.40
TRP Tissue Repair 0.235 -13% -31% $11,695,088.75
NXS Next Science Limited 0.62 -13% -63% $133,169,883.08
NYR Nyrada Inc. 0.13 -13% -11% $19,501,087.50
EPN Epsilon Healthcare 0.025 -14% -5% $7,809,204.29
PAA Pharmaust Limited 0.067 -14% -3% $21,233,129.66
ACW Actinogen Medical 0.11 -15% -5% $188,726,350.79
DOC Doctor Care Anywhere 0.059 -16% -58% $13,178,766.38
ZLD Zelira Therapeutics 0.99 -16% -479% $9,577,116.00
IDT IDT Australia Ltd 0.09 -16% -17% $21,691,961.73
MXC Mgc Pharmaceuticals 0.0125 -17% -2% $35,823,091.51
ATH Alterity Therap Ltd 0.01 -17% -1% $24,164,184.18
IRX Inhalerx Limited 0.054 -17% -4% $10,112,415.68
AN1 Anagenics Limited 0.029 -17% -3% $6,630,639.09
GLH Global Health Ltd 0.205 -18% -15% $11,890,693.11
TRU Truscreen 0.035 -19% -4% $12,700,318.86
MEM Memphasys Ltd 0.013 -19% -8% $12,482,589.98
BIT Biotron Limited 0.029 -19% -6% $26,156,048.68
ADR Adherium Ltd 0.004 -20% -1% $19,100,782.14
AGH Althea Group 0.065 -22% -16% $22,816,727.71
ALA Arovella Therapeutic 0.024 -23% -2% $16,771,270.20
NTI Neurotech Intl 0.083 -23% 2% $69,389,712.84
OIL Optiscan Imaging 0.105 -25% -8% $65,058,588.21
MDC Medlab Clinical Ltd 7.95 -25% -1455% $18,153,840.90
S66 Star Combo 0.13 -26% -13% $17,560,787.53
4DX 4Dmedical Limited 0.465 -28% -89% $138,411,163.39
ZNO Zoono Group Ltd 0.1 -31% -28% $16,692,522.20
CPH Creso Pharma Ltd 0.0195 -37% -6% $33,047,228.81
KZA Kazia Therapeutics 0.09 -40% -117% $14,870,216.82
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Microba Life Scienes (ASX:MAP)

Giant healthcare stock Sonic Healthcare (ASX:SHL) has bought a stake in small cap Microba, sending the MAP share price 90%+ higher for the month.

Sonic took a 19.9% stake at 26c a share as it seeks to acquire options for a further 5% stake.

Microba focuses on gut health with a revenue-generating business in microbiome testing. The company sells these testing products to healthcare practitioners and gets paid on a fee-for-service basis.

Microba and Sonic have agreed on the terms to deliver the microbiome testing technology into Australia, New Zealand, Germany, United Kingdom, Belgium, Switzerland and the United States.
 

Jayex Technology (ASX:JTL)

The e-health SaaS healthcare platform has completed a thorough review of its operations by newly appointed CEO Rob Hadley.

The review will result in significant cost savings across all aspects of the business.

Total savings are expected to be in the order of $195,000 per month or $2.34 million per year.

Jayex said the restructure was necessary to capitalise on growth opportunities in the UK, where Jayex serves approximately 3,000 GP clinics.

Hadley was appointed in September as the company looks to expand the business in the UK amid changes in the country’s NHS health system.
 

Immutep (ASX:IMM)

Immutep  has entered into a second clinical trial collaboration agreement with Merck (Germany) and Pfizer to study Immutep’s LAG-3 candidate, Eftilagimod Alpha, combined with Avelumab to treat urothelial cancer.

The new Phase I clinical study called INSIGHT-005 will examine patients with urothelial cancer, and builds on Immutep’s strategy to increase target indications for its drug candidate when combined with standard of care drugs.

The collaboration with Merck and Pfizer also builds on the encouraging clinical data previously reported from the INSIGHT-004 trial.
 

Mach7 Tech (ASX:M7T)

Mach7 received a signed agreement from St Paul’s Hospital in Hong Kong to replace its Picture Archiving and Communication System (PACS).

The one-year licence agreement is valued at $1.52 million, and will involve Mach7 installing the entire Enterprise Imaging Platform including its Vendor Neutral Archive (VNA), eUnity Diagnostic Viewer, and Universal Worklist.

St Paul’s Hospital is a large scale, long established private hospital located in Causeway Bay on Hong Kong Island.

The hospital has approximately 500 beds and more than 20 departments.

 

 

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