The FDA has approved the most expensive drug, CSL Behring’s hemophilia B gene therapy. Picture Getty
Health & Biotech
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.
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….
| 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 |
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.
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 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 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.
Health & Biotech
Health & Biotech
Health & Biotech
