Rhythm Pharmaceuticals Inc (RYTM)

PANTHERx® Rare Selected by Rhythm Pharmaceuticals Inc. as the Exclusive U.S. Specialty Pharmacy for the Expanded Indication for IMCIVREE® (setmelanotide)March 20, 2026 12:58 PM
PR Newswire (US)

PITTSBURGH, March 20, 2026 /PRNewswire/ -- PANTHERx® Rare Pharmacy, the trusted leader and innovator in patient access and support services for personalized rare disease care, announced that it was selected by Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), as the exclusive U.S. Pharmacy distribution partner for the expanded indication of IMCIVREE® (setmelanotide) for adults and children 4 years of age and older living with acquired hypothalamic obesity.1







Acquired hypothalamic obesity is a rare disease characterized by accelerated and sustained weight gain caused by an injury to the hypothalamus. Acquired hypothalamic obesity most frequently follows the growth or treatment of craniopharyngioma, astrocytoma or other hypothalamic-pituitary tumors. Additional causes of injury may include traumatic brain injury, stroke or inflammation.2, 3PANTHERx will leverage its proprietary and industry-leading RxARECARE® model to support patients, caregivers, and prescribers with therapy-specific clinical expertise. Through individualized case management, proactive education, and comprehensive financial and insurance navigation, PANTHERx makes complex rare disease therapies more personal, less overwhelming, and easier to access."This new indication for IMCIVREE marks an important scientific milestone for individuals and families affected by acquired hypothalamic obesity, a highly complex disease with historically limited therapeutic options," said Bansi Nagji, CEO of PANTHERx Rare. "We're honored to deepen our collaboration with Rhythm Pharmaceuticals, and to help patients receive the personalized, high-touch clinical support necessary to navigate treatment."PANTHERx has been the exclusive pharmacy distribution partner for IMCIVREE for chronic weight management in adult and pediatric patients 2 years of age and older with obesity due to Bardet-Biedl Syndrome and proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency confirmed by genetic testing since 2020.1About PANTHERx Rare
PANTHERx Rare makes rare disease care more hyper-personalized and less overwhelming by focusing relentlessly on each patient and each therapy. PANTHERx experts develop deep personal relationships with patients, prescribers, and pharmaceutical partners, serving as trusted advocates to ensure seamless collaboration and exceptional care. Since its founding in a garage in Pittsburgh, PA in 2011, PANTHERx has grown into the largest independent rare pharmacy in the U.S., leveraging established-company resources while maintaining small-company responsiveness, innovation, and attention to detail.PANTHERx is licensed in all 50 states and was the first national pharmacy to achieve dual accreditations in rare disease from the Accreditation Commission for Health Care (ACHC) and Utilization Review Accreditation Commission (URAC). PANTHERx is also the eight-time winner of the prestigious MMIT Patient Choice Award for patient satisfaction, including the 2025 honor.About Acquired Hypothalamic Obesity
Acquired hypothalamic obesity is a rare disease characterized by accelerated and sustained weight gain caused by an injury to the hypothalamus. Hypothalamic injury may lead to decreased alpha-melanocyte-stimulating hormone (a-MSH) production and impairment of MC4R pathway signaling. The MC4R pathway is responsible for regulating energy balance and body weight. Acquired hypothalamic obesity most frequently follows the growth or treatment of craniopharyngioma, astrocytoma or other hypothalamic-pituitary tumors. Additional causes of injury may include traumatic brain injury, stroke or inflammation. Due to impairment of the MC4R pathway, patients experience accelerated and sustained weight gain, often accompanied by hyperphagia and/or decreased energy expenditure.For more information, please email TheRareSP@pantherxrare.com or visit www.pantherxrare.com.IMCIVREE (setmelanotide) Prescribing Information. https://rhythmtx.com/wp-content/uploads/2026/03/IMCIVREE-prescribing-information.pdfKim JH, Choi J-H. Pathophysiology and clinical characteristics of hypothalamic obesity in children and adolescents. Annals of Pediatric Endocrinology & Metabolism. 2013;18(4):161. doi:10.6065/apem.2013.18.4.161Acquired hypothalamic obesity. Rhythm Pharmaceuticals. Accessed March 3, 2026. Our Focus—Acquired Hypothalamic Obesity | Rhythm Pharmaceuticals*RxARECARE® is a registered trademark of PANTHERx Specialty, LLCPANTHERx Rare Media Contact:
Jennifer May, Senior Manager, Marketing Communications
jmay@pantherxrare.com



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Original: PANTHERx® Rare Selected by Rhythm Pharmaceuticals Inc. as the Exclusive U.S. Specialty Pharmacy for the Expanded Indication for IMCIVREE® (setmelanotide)

RYTM 10Q 11/5

RYTM approaching a 52=week high

RYTM new 52 week high

RYTM new 52 week high

RYTM new 52 week high

RYTM new 52 week high

In my view ORMP is better than RYTM from investment perspective.

Sigh, how wrong can one be!
(including me - of course)

Why am I the only one posting here? This thing has been steady up over100% since I started posting here. Even on a day like today when most others are red. Another green day here!! $RYTM

When $20 breaks and holds this will fly! IMO

Looking like it’s ready to move up again here. Possible bounce off of support.

Some you win some you lose - I had a lot of conviction but later lost faith after that time and sold completely.

Sorry

Thanks for posting. Breakout mode! GL

RYTM Chart

I’m liking the chart on this one.

Two year slide continues.

I lost money on RYTM and booked losses already.

In my view ORMP is better than
RYTM from investment perspective.

Spot on!

In my view ORMP is better than RYTM from investment perspective.

The current market capon $255 million allows it to be a 10 bagger ($2.55 B) - speciall that it has first in class product and huge unmet market.

RTYM also has first in class product with unmet needs but it is rare disease and not many outside US can afford

Agree - it is good company
Will take time to make money but proof of product is there.

The valuation is fair at this point - upside maybe 50-70% by end of the year based on how they price and are able show sale.

The payment cycle with insurance will be key as the product is very high priced -

I started building a major position. And already a little green!
Quite the biotech soon last couple weeks= opportunity!

For some reason it is tanking now -sale the news

RYTM is not cheap as it has market cap of $832 million for pre-revenue biotech - so it is richly valued.


Are you kidding? You know what the mc is today?

Market Cap 1.737B

Since you introduced me to this company it has doubled
in price, if it was richly valued then, what is it now?

But this is besides my previous point - namely i 'missed'
the entry point.

You can actually wait for some time and buy - this is a long term play

I missed out this one!

Just amazing results - they are starting new basket trials.

The product is approved for one indication so it is now label expansion - the pricing power is amazing. This should really take off. Is it next Alexion in terms of rare disease treatment and pricing power?

Head the conf call - It was good call.

The results are much better that what PR said. Slide 14-15 shows correct numbers which were clarified by them.

Real numbers
Total patients - 31
Total BBS patients - 28
No of patients dropped - 4
Total no of patients relevant - 24

No of patients with 5%+ weight reduction - 16 = 67% that is double the number compared to combined number of 34.5%

No of patients who lost more than 10% weight - 11 out of 24 (45.8%)
Mean reduction - 14.5%

Compared to 1st approval which had few hundred population, BBS has 1000-1500 target population in US alone

This is large population for rare disease - @ $200k per year - this is a big market.

Also this is not a gene therapy - so drug is needed forever and it keeps the weight down.

Last point - All genes targeted in basket trial are in BBS category (strong or very strong relationship) so they think the basket trial results may be positive.

Cash enough till 2022. I think if stock goes down further, it will be good opportunity to buy - stock could give 50% returns from here in 6 months based on basket trial results.

Phase 3 results - BBS stat significant -AL not

Market reaction is not good. Conf call will give more info

https://finance.yahoo.com/news/rhythm-pharmaceuticals-announces-positive-topline-120000038.html

Finally - Approval

“Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE™(setmelanotide) as First-ever Therapy for Chronic Weight Management in Patients with Obesity Due to POMC, PCSK1 or LEPR Deficiency”

Another good news - voucher that they can sell or use for next approval

“With this approval, the FDA issued a Rare Pediatric Disease Priority Review Voucher (PRV) to Rhythm. The PRV can be redeemed to receive priority review for any subsequent marketing application or sold or transferred to other companies for their programs.”


https://finance.yahoo.com/news/rhythm-pharmaceuticals-announces-fda-approval-123000486.html

That’s a big run with PDUFA being in November. I took some profit but still in the game and will buy 100% back if reversal. Outstanding shares only @ 44M and float 30M. PT @ $33.

Good call!

What is happening? Looks good

Not so sure the last few years.

I am no US citizen, i would not like to comment
about US politics. We have enough political
unrest in my part of the globe, in Israel.

I’ve done enough DD on PSTI and RDHL

I was a heavy investor in PSTI, sold it all a few
weeks ago at a big loss, and not looking back.

RDHL, otoh is a serious company, i am sure it will
go places, high places that is.

As for RYTM, I have no funds at the moment, but
will start a position soon.

GLTY

RYTM is not cheap as it has market cap of $832 million for pre-revenue biotech - so it is richly valued.

It has 70 employees - more than many biotech at this stage but not sufficient for marketing the drug. So they have to find a partner or go with buyout.

The good news is - the co has $228 milllion plus cash and it will allow them to run till end of 2021.

They are in niche area and there is no other competition nearby. If the drug can address genetic obesity - they have 80,000 target audience in US alone.

If the drug can be tested on genetic obesity/weight loss, it will be gold mine.

Not so sure the last few years. But what the heck.
I’m new on RYTM. I’ve done enough DD on PSTI and RDHL to hold a small 500 share stake in both. I’ll do some extra digging here because the stakes are much higher. The last 3 months haven’t been bad on the other two.
GLTUA
Pr

Sure. A free country, isn't it?

Can anyone play???

Thanks for the heads up Amatuer17,
i'll dig into it over the weekend.

Welcome to the board. Hope we all make money

This is a PDUFA based play

PDUFA date - 27th Nov

Disease - rare disease - obesity based on genetics

MOA - impacts the MC4PA gene that controls obesity and hunger. They have 4 more trials for other sub areas also in advance stage.

As per conf call - they are looking at pricing from $200k to $400k per year Patient population for this approval - around 1000

https://ir.rhythmtx.com/static-files/fd4e0919-4d82-47e0-afe3-8cd9b5151490